As patients, we normally rely on our doctors to tell us which tests and medications to take for the betterment of our health. Rarely do we question them since they know a whole lot more than most of us when it comes to medical ailments and overall health. However, that doesn’t mean you can’t find out more about the various suggestions doctors make.
If you have an ailment in the body and your doctor finds it hard to determine exactly what it is, they will likely ask you to get either a CT scan or MRI done. The tests are used to provide a detailed view of your internal body to help determine the ailment. We breakdown the two for your better understanding:
CT scans provide imaging using x-rays at different angles. This scan is more in-depth as compared to an x-ray. X-ray tests use a beam of radiation from a set angle and display the image. Since a CT scan uses a series of radiation beams at different angles, it slices the same image up, giving a 3D view so doctors can understand the ailment better. With the help of a computer, an image is produced. CT scans can help determine ailments such as cancer, bone injuries, and chest and lung ailments.
Magnetic resonance imaging (MRI) uses a magnetic field instead of radiation and provides a more detailed image of the body which also includes soft tissues along with the internal body. It is used to help diagnose the following:
Damaged blood vessels
Spinal cord injury
It can also be used to ensure that various organs are healthy.
Both methods are noninvasive and rely on heavy technology. But when it comes to CT scans, more and more hospitals are opting for mobile CT scanners, which make it easier for them to manage.
Getting Ready for the Test
Preparing for CT Scan and MRI is slightly different. With CT scans, your doctor may recommend you take a contest dye. The dye helps highlight the scanned region more and is generally consumed when scanning the abdomen. It is important to notify your doctor if you have any allergies because you may react to the dye. If you’ve previously had reactions to prednisone (a steroid), iodine, or seafood then the doctor should be immediately notified. Other than that, the doctor may ask you not to drink or eat several hours before the test.
For an MRI, the one thing you need to make sure is that you are not wearing anything that can be detected by magnets. This means, no jewelry, watches, hearing aids, glasses, and other items that may have a metal can be worn during the test. In some cases, a gadolinium dye may be recommended which is injected into the hand or arm. The dye highlights certain details in the imaging and rarely results in any type of reaction. The test can be lengthy for some as it takes anywhere from 30-45 minutes, so if you are claustrophobic, you may want to discuss that with your doctor since you are required to stay in a closed space for that period.
CT Scan: You will be asked to put on a robe and remove jewelry and other metal objects so they don’t have any impact on the image produced. The scanner itself is a doughnut-shaped machine and you lie on a flat table in the middle. The table starts to move back and forth and x-ray tubes fitted into the scanner send out beams and different angles. They pass through your body to the other end of the scanner. The test is painless but make sure you are comfortable because you will be asked to stay still as the scan is going on.
MRI: The MRI machine is a long narrow tube that is open at both ends. Like in a CT scan, you lie down on a flat movable table that slides into the tube. As you slide in, the table stops at the specific part of the body being examined and a magnetic field is created and radio waves are directed to the body. The machine does make tapping and thumping noises, so the technician will likely offer earplugs to block it out.
Understanding the Test Result
After getting either a CT scan or MRI done, you will need to consult your doctor. Unless you are a trained doctor, the images will make little to no sense to you. You will need to consult a radiologist that can explain the results to you. In case of an ailment, they will usually recommend you consult a specialist, depending on the ailment, that can assist you further.
As a patient, it is important for you to understand the tests and treatment doctors recommend. Most of the time, you can consult your doctor and they will be more than willing to give you the information you need. Knowing makes it easier for you to undergo the tests and treatments with a little more ease.
Scott has been working in the radiology field for over 30 years. He finds the biological phenomenons found in humankind fascinating and appreciates the incredible use that diagnostic imagery has to save lives. Other than acting as the President for Catalina Imaging, Scott enjoys spreading the word on new insights and breakthroughs in the radiology field, specifically the impact that mobile imaging has for patient care.
https://powerfulpatients.org/pen/wp-content/uploads/What-Patients-Should-Know-About-CT-Scans-and-MRIs.png600600Scott Ronkhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngScott Ronk2019-10-28 13:18:052019-10-28 13:18:05What Patients Should Know About CT Scans and MRIs
As soon as the first golden leaves of autumn appear on the trees a feeling of sadness starts to descend over me. I’m catapulted back over the years to a late September day. A day that’s etched forever on my mind. The day I was diagnosed with breast cancer.
In the weeks that followed my diagnosis, I became enveloped in a sea of pink as Breast Cancer Awareness Month took place. But when October came to an end and the pink ribbon wearers disappeared, I was left wearing the everyday reality of the disease. Over a decade has passed since then and yet I still feel a sense of sadness when I think of that time and all I had yet to learn and go through. I can’t help wondering how much my experience might have been different if I had known then what I know now.
One thing I now know is that we owe it to those who come after us to share our hard-earned wisdom. So in that spirit, here are ten things I wish I had known back when I was a newly diagnosed patient. I hope sharing these lessons may make the path towards recovery that little bit smoother for others who are new to this journey.
1. Everything in your life is about to change
Once you’ve been baptized in the fire of cancer your life as you knew it will be irrevocably changed. The apparent randomness of a cancer diagnosis can shake your sense of identity to its very core and afterwards nothing will ever feel certain again. Cancer invades not only your body but every other area of your life, including your relationships, family life, friendships, finances, career, and even your sense of self. You may be surprised to find the people you thought you could count on disappear from your life. At the same time, you will be surprised to find support can also come from unexpected sources.
2. Online support is real
Among those unexpected sources will be the people you meet online. Online communities may be virtual but they are no less real in terms of support and advice. On Facebook and Twitter you can find patient communities which will be an invaluable source of information and support to you. Check out the #BCSM Twitter chat – a weekly chat for breast cancer patients and their caregivers.
3. You will feel fearful and anxious
One of the most common emotional and psychological responses to the experience of cancer is anxiety. Cancer is a stressful experience and normal anxiety reactions present at different points along the cancer pathway. Anxiety is a natural human response that serves a biological purpose. It’s the body’s physical “fight or flight” (also known as the stress response) reaction to a perceived threat. You may experience a racing or pounding heart, tightness in the chest, shortness of breath, dizziness, headaches, upset tummy, sweating or tense muscles. All of these signs indicate that sympathetic arousal of your nervous system has been activated, preparing you to stand your ground and fight or take flight and run away from danger.
Try this coping tip. When we are fearful and anxious we tend to breathe more shallowly. Shallow breathing, which doesn’t allow enough oxygen to enter our bodies, can make us even more anxious. Practice taking some slow deep abdominal breaths. Deep abdominal breathing slows the heart down and lowers blood pressure. The advantage of focussing on the breath is that it is always there with us. We can turn to it anytime we are feeling anxious.
4. You are your own best health advocate
Although you may be reeling from the news of a cancer diagnosis, it’s important that you learn as much as you can about your diagnosis and what treatment options are available. In this article, you will learn which questions you should ask your healthcare team and where to find reliable and trustworthy information to become better informed about your health condition.
5. Your will experience brain fog
Cognitive impairment, to a lesser or greater degree, can affect you both during and after your treatment. You may have the feeling that your cognitive abilities are slower and less acute than before – almost as if your brain is shrouded in a fog. We call this the “chemobrain” effect and the effect may persist for months or even years after treatment ends. A more formal term – post-cancer cognitive impairment (PCCI) – is used by researchers to describe a group of symptoms, which include slow mental processing, difficulty concentrating, organizing, and multitasking.
PCCI symptoms can also include:
memory loss – forgetting things that you normally remember
struggling to think of the right word for a familiar object
difficulty following the flow of a conversation
confusing dates and appointments
misplacing everyday objects like keys and glasses
It’s still not clear how many people with cancer get chemobrain or which drugs cause it. However, there are several things that you can do to help you cope with its symptoms. Read this article for more information.
6. You will experience bone-crushing tiredness
We all know what it’s like to feel tired – physically, mentally, and emotionally, but usually after some relaxation and a good night’s sleep, we are ready to take on the world again. When you have cancer, though, rest often isn’t enough. You experience a persistent, whole-body exhaustion. Even after adequate sleep or rest, you will still feel tired and unable to do the normal, everyday activities you did before with ease.
A lot of cancer patients don’t report fatigue to their doctors because they think that nothing can be done for it. In fact, there are things that can be done to alleviate the debilitating effects of cancer-related fatigue. If left untreated, fatigue may lead to depression and profoundly diminish your quality of life, so it’s important that you speak to your doctor if fatigue is an issue for you. Read How To Cope With Cancer-Related Fatigue for more tips and information.
7. You may be surprised by feelings of guilt
Cancer-related guilt is a complex emotion. You may feel guilty and worry that your lifestyle choices somehow contributed to a cancer diagnosis. If you learn that you carry the BRCA1/2 gene, you may feel guilty that you could pass this gene mutation on to your children. Or you may feel guilty that your cancer was diagnosed at an earlier stage than a friend or family member who has a worse prognosis. These feelings of guilt may surprise you, but it’s a perfectly normal reaction to a traumatic life event like cancer. Read How Do You Deal With Cancer Guilt to learn more about this topic.
8. You will feel pressured to stay in a positive frame of mind
I admit I caved in at the beginning to pressure to stay strong and positive when I was first diagnosed with breast cancer. I did it because it reassured the people around me. While I accept that for some people maintaining a positive attitude is a valid coping mechanism, for myself and many others, the pressure to always show our sunny side is a denial of our pain, anger, grief, and suffering. I now believe by promoting this attitude in the face of cancer, we create unfair expectations and deprive patients of an outlet for their darker fears. This is my personal viewpoint, and it’s one that I don’t expect everyone to share. However, I mention it here so that those who are newly diagnosed don’t feel they have to always present a smiling face to the world. It really is ok to express your fears, your sadness, your anger, and your grief too.
9. You will need time to grieve
While many people think of grief only as a reaction to bereavement, we can feel grief after any kind of loss. When we step back and look at the cancer experience we see that grief and loss are a fundamental part of the experience. Some of our losses are tangible, for example losing our hair, and some are more intangible, such as the loss of trust in our bodies.
Coping with the losses associated with cancer is challenging. Grief brings many emotions with it. Patients, as well as caregivers and family members, may go through emotions of anger, denial, and sadness. While there is no right or wrong way to grieve, there are healthy ways to cope with the pain and sadness that, in time, can help you come to terms with your loss, find new meaning, and move on with your life.
10. Cancer doesn’t end when treatment does
I wish I had been better prepared for how I would feel when cancer treatment ended. I just assumed I would pick up where I had left off and get on with my life as if cancer was no more than a blip. I wish someone had told me that cancer doesn’t end when treatment does. Sometimes, there can be a code of silence surrounding the aftermath of cancer treatment. There is an expectation that when you walk out of hospital on that last day of treatment, your cancer story has ended. But in many ways it’s only just beginning.
I now know that cancer is more complicated than simply being disease free and that a physical cure doesn’t mark the end of the healing process. Adapting to changes in energy and activity levels, adjusting to altered relationships at work and in your personal relationships, coming to terms with a changed body image, and managing pain and treatment side effects are some of the things you will face in the post-treatment phase of recovery. Be compassionate and gentle with yourself as you move through this stage of your cancer journey. Don’t judge yourself or feel pressured by others to try to hurry this stage along.
Being diagnosed with cancer is a life-changing event. You will go through many emotions and experiences throughout the roller-coaster ride of diagnosis, treatment, and beyond. Each person will experience it in their own way. While there’s no right or wrong way to go through the experience, it’s important that you don’t ever feel as if you have to go through it alone. Reach out at each step of the way and find someone who understands what you are going through and can offer you the support you need.
A Stanford Medicine X e-Patient scholar, Marie Ennis O’Connor is an internationally recognized keynote speaker, writer, and consultant on global trends in patient engagement, digital health and participatory medicine. A board member of the Patient Empowerment Foundation, a network of people, foundations, organizations and medical institutions dedicated to empowering patients worldwide, Marie’s work is informed by her passion for embedding the patient voice at the heart of healthcare values. She writes about the experience of transitioning from breast cancer patient to advocate on her award-winning blog Journeying Beyond Breast Cancer.
https://powerfulpatients.org/pen/wp-content/uploads/Ten-Things-I-Wish-I-Had-Known-When-I-was-Diagnosed-with-Breast-Cancer.png600600Marie Ennis-O'Connorhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngMarie Ennis-O'Connor2019-10-21 12:43:262019-10-21 12:43:26Ten Things I Wish I Had Known When I was Diagnosed with Breast Cancer
So, another issue that is really important for young women is discussing fertility preservation. And this really needs to happen at the time of their diagnosis. So, we know that the cytotoxic agents that we can give females just through chemotherapy can decrease the ovary and the ability for these women to have menstrual periods after chemotherapy. So, the ability for them to get pregnant naturally.
As well as some of the medications. So, somebody who has a breast cancer that is estrogen positive, the recommendation is for these women to be on hormone suppressant medicine for five to 10 years after their breast cancer diagnosis and treatment, therefore not being able to be pregnant while on these medications. So, talking with young women when they get diagnosed about their family planning and their fertility options up front before they have surgery or chemotherapy is really beneficial.
And whether or not they need to see a fertility preservation specialist. If they want to consider IVF. Or if they have a gene, looking at genetic testing for their future offspring. So, these are all conversations that really need to happen before these women begin chemotherapy if they need it.
And the good thing is that at the young women’s clinic, these fertility specialists are embedded in the clinic. So, they are able to get an appointment with them right away. And a lot of times if these women do want to undergo fertility preservation, that can happen within 10 days of seeing the specialist. So, it really doesn’t delay their care. And we do know that it is safe even with the breast cancer diagnosis.
The other thing is that we do offer a medicine which is a GRNH agonist which will kind of essentially shut down the ovaries during chemotherapy to help protect them so that when a young woman is done with chemotherapy, it helps the ovary kind of get back to normal a little bit sooner.
So, it sounds good in theory. Unfortunately, it’s not something that is covered by insurance companies right now. And so, fertility preservation is expensive. And so, the good thing is there are a lot of groups that put together packages and stuff for these young women to be able to afford it. But it is pretty pricey. So, for those that can afford it, it is a great option. And a lot of them do take advantage of it. I think there are a lot of misconceptions about it. Number one is that patients don’t really know if it’s safe.
Number two, they are scared about their overall diagnosis and a potential delay and 10 days might make some of them afraid that doing that is a good option. Another thing is when these women come in with a diagnosis of breast cancer, they see a surgeon, a medical oncologist, a radiation oncologist, a plastic surgeon.
And so a lot of times an extra appointment at that point in time is just really overwhelming for these women. So, our goal is to kind of refocus and say, “Hey, the good news is that with our modern therapies you’re going to be here for a long time. So, let’s plan for the future now so that in the future you’ve got options.”
https://powerfulpatients.org/pen/wp-content/uploads/Breast-Cancer-Before-40_-How-Can-I-Preserve-My-Fertility_.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2019-10-08 14:44:212019-10-09 08:50:56Breast Cancer Before 40: How Can I Preserve My Fertility?
Sure. So, when we talk about breast cancer in young women, usually we are referring to women under the age of 40. And the prevalence for breast cancer in general is one out of eight women. For women under the age of 40, it’s about a seven percent incidence of breast cancer. So, it’s about a seven percent incidence in the general population in the United States. So, that’s about 12,500 women per year are diagnosed under the age of 40 with breast cancer.
So, the prevalence has pretty much been the same over the years. We used to think that women diagnosed at a younger age had a more aggressive breast cancer. But the more we look at things, the more we realize that women under the age of 40 usually are diagnosed at a later stage because it’s not something that somebody in their 20s or 30s is thinking that a breast lump equates to cancer. So, these women unfortunately present at a later stage.
Many times, it’s because they are pregnant, breastfeeding or just not having any family history where the first thing they think of when they get a lump is that they actually have breast cancer.
So, the first thing is women under the age of 40 usually present at a later stage, meaning that they have larger cancers, and the cancers because they are larger have had more time to spread to the lymph nodes. So, these women they don’t necessarily have more aggressive breast cancers than older women, it’s just that it’s found at a later stage.
So, the treatment options are the same for young women with breast cancer. So, depending on the size of the tumor and the size of her breast, women are given the option for a lumpectomy which usually is follow by radiation versus a mastectomy. And studies have shown that either surgical choice is a good option for women. And that one surgery doesn’t make a young woman, or an older woman live any longer.
That the survival for breast cancer is based on stage not the choice of surgery that they pick.
So, one of the first things is that women under the age of 50 – the average age for breast cancer in the United States is between 64 and 68.
So, if you are under the age of 50 or under the age of 40 there’s a higher chance that maybe these women carry a gene that would increase the risk of getting breast cancer. And so nowadays, we test over 21 genes. But these genes can increase their risk of getting breast cancer not only in the breast they have the breast cancer in, but in their other breast as well. It also increases the risk for other types of cancers such as ovarian cancer. And they could potentially pass this gene on to their kids.
So, sometimes women – and this is the great thing about academic medicine. Everything is changing so quickly with the modern research. So, a lot of times a woman who is triple negative, which is an estrogen receptor negative breast cancer.
If they are genetic positive for the BRCA gene, they qualify for certain medications or chemotherapy that we know targets specifically that type of cancer and their gene. So, that’s why it is important for some of these women to get genetic testing to see if the certain chemotherapy regimens or medicines that we have would benefit their type of cancer.
So, one of the things for any woman diagnosed with breast cancer at a young age is to offer those women genetic testing. And sometimes it can be a relief or sometimes it can be very challenging for these women to think, “Oh my gosh. I have this gene. I’m at increased risk for more cancers.” And that potentially they could pass that on to their children.
So, having the women who come in meet with a genetics counselor to go over the risks. And the reality is that of all the women under the age of 50 that test for the gene, only 10 percent actually carry the gene.
So, the good thing is that 90 percent most likely don’t have the gene. But it still is an anxiety provoking thing for these women to go through.
Another thing is that these women a lot of times are younger. So, they have either two things – young children to take care of which is extremely difficult to mange an already stressful motherhood. You throw in a diagnosis of cancer, whether or not there is a dad involved or a father that needs to help out with these kids. A lot of times their families need to help out. So, we have a psych oncologist that’s part of our team. And it’s really great. How do you tell your kids you have cancer? And how do you manage kind of day-to-day life with going through this? So, that’s another great program that is offered. And importantly for a woman who doesn’t have children but maybe desires to have children or even to have more children than the ones that she has,
looking at fertility options for young women is huge.
So, we know that some of the chemotherapy that we give breast cancer patients decreases their ability to have children in the future. So, the chemotherapy can shut down the ovaries. So, sometimes women – and this is the great thing about academic
medicine. Everything is changing so quickly with the modern research. So, a lot of times a woman who is triple negative, which is an estrogen receptor negative breast cancer.
If they are genetic positive for the BRCA gene, they qualify for certain medications or chemotherapy that we know targets specifically that type of cancer and their gene. So, that’s why it is important for some of these women to get genetic testing to see if the certain chemotherapy regimens or medicines that we have would benefit their type of cancer.
So, the one thing is that I tell these women that breast cancer takes a good year between chemotherapy if they need it, surgery, radiation of they need it and the whole process of recovery. That is really takes a good solid year before they are kind of done going to their doctor’s appointments. But the reality is that for an early stage breast cancer, studies have show for young women the overall survival is over 92 percent. So, I say, “This is going to be a tough year. We are going to get through this together. And the good thing is that you are going to be alive in five, 10, 15, 20 years. And so, our goal is to get you the best quality of life not only now but in the future.”
As far as hope for young women with breast cancer, things are changing so fast. The medicine that’s out there – we’re doing these studies where women are getting chemotherapy and by the time they get to surgery about 40 to 60 percent of the tissue that I take out has no residual cancer. That’s phenomenal. That means that the medications that these women are getting are working really well for their cancer. And so, the hope is that in 10 years I don’t have a job because the medicine that they are getting works so well that the cancer can be removed without needing surgery. And so, I think in our lifetime we will find either a cure or a complete resolution of breast cancer.
https://powerfulpatients.org/pen/wp-content/uploads/Breast-Cancer-Before-40_-What-You-Should-Know-1.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2019-10-08 14:42:132019-10-09 08:51:49Breast Cancer Before 40: What You Should Know
This blog was originally published by Cancer Treatments Centers of America on August 21, 2019, here.
When faced with opposition, it’s beneficial to learn as much as possible about the opponent. A pitcher reads a scouting report before facing a lineup. An army consults intelligence before engaging the enemy in battle.
The same principles apply to the treatment of some cancers. When treating a tumor, it’s important for a doctor to know as much as possible about that cancer—specifically, what is driving the tumor’s growth.
To get the inside information on a tumor, doctors are increasingly relying on biomarkers, short for biological markers, measurable signs or substances in the body that may indicate a patient’s overall health and/or the presence or progression of disease.
The discovery of biomarkers in cancer drastically changed the course of cancer treatment. For decades, many cancers were treated similarly, with surgery, radiation therapy or chemotherapy. Identifying biomarkers in cancer cells has led to the development of new precision medicine drugs, such as targeted therapy and immunotherapy, designed to target specific features in cancer cells, potentially reducing the damage to healthy cells. “The routine use of a variety of biomarkers has substantially changed the way in which cancer medicine is practiced,” says Maurie Markman, MD, President of Medicine & Science at Cancer Treatment Centers of America® (CTCA), “from providing more accurate prognostic information to assisting in the prediction of specific therapeutic strategies that are more likely to result in a favorable outcome for an individual patient.”
What are biomarkers?
A biomarker is any measurable indicator of a person’s health. Blood pressure is a biomarker, as are body temperature, blood sugar and cholesterol measurements. In cancer, biomarkers also include proteins, hormones, gene aberrations, such as mutations or rearranged genes, and other molecules found in or on cancer cells. Cancer biomarkers may be found in routine blood, urine or stool tests. Others may require a biopsy and/or advanced genomic testing to uncover. “Genomics has made it so much easier to find gene mutations,” says Arturo Loaiza-Bonilla, MD, MSEd, FACP, Vice Chair for the CTCA® Department of Medical Oncology. “Now we may be able to target a mutation and potentially get the cancer to stop growing.”
Biomarkers play multiple roles in the treatment of diseases, such as cancer, including:
Diagnostic: Helping confirm the presence of disease, sometimes before symptoms develop
Prognostic: Helping forecast the progression and aggressiveness of the disease and the risk of recurrence
Predictive: Helping doctors identify how patients may respond to certain drugs
Biomarkers may play any or all these roles and more. Some biomarkers may be used to assess a patient’s risk of developing disease, the effectiveness of a treatment or whether a treatment is safe or toxic.
PSA: Prostate specific antigen may indicate prostate cancer. This biomarker may be used not just to diagnose the disease, but to measure its progression and how the treatment is performing.
HER2: Human epidermal growth factor receptor 2 is found in many cancers, especially breast cancer. The targeted therapy drug trastuzumab and other similar monoclonal antibodies may be a treatment option for patients with HER2-positive cancers.
BCR-ABL: This gene, known as the Philadelphia chromosome, is found in patients with chronic myelogenous leukemia. Presence of the gene may indicate the patient may respond well to treatment with a tyrosine kinase inhibitor drug such as imatinib.
PD-L1: Programmed death ligand 1 is the companion receptor to PD-1. It may indicate a cancer’s ability to evade the immune system. Immunotherapy drugs called checkpoint inhibitors may be an option to treat cancers high in PD-L1.
CA-125: High levels of cancer antigen-125 are found in many cancers as well as other diseases. Treatment options for cancers with CA-125 vary depending on where the cancer originated.
MSI-H: Microsatellite instability-high is a mutation in the DNA of cells found in many cancers, especially colorectal cancer. Checkpoint inhibitor drugs have been approved for cancers with MSI-H.
Biomarkers don’t always tell the full story. Discovery of a biomarker that might indicate an increased cancer risk doesn’t mean a patient will get cancer. Not all cancers have identifiable biomarkers. And identifying a driving biomarker in a cancer does not necessarily lead to a treatment option. Some biomarkers for cancer have no corresponding targeted therapy or immunotherapy drug. For example:
TP53: Tumor protein 53 is a tumor suppressor gene designed to help stop cancer cells from growing. TP53 mutations are the most common found in cancer cells and may be found in most types of cancer.
RAS: About 30 percent of all cancers, including 95 percent of all pancreatic cancers, have known mutations in the RAS family of genes that control cell death and growth.
No targeted therapy drugs have been approved specifically to treat cancers with these mutations. “A number of recognized critical signaling pathways in cancer development, progression and resistance remain very difficult to ‘target’ to influence clinical outcomes,” Dr. Markman says. “The ability to successfully and safely target either or both of these pathways has the potential to be an important advancement in cancer management.”
Many cancers, especially solid tumors, have multiple biomarkers, any one of which may be able to drive a cancer’s growth. Target one biomarker, and another may take over as the driving mutation. And not all the same biomarkers are found in every cancer cell. “As cancer cells grow, they start to develop new abnormalities, mistakes made while the cells are multiplying,” Dr. Bonilla says. These new mutations may make the cancer more resistant to treatment.
Also, doctors need to take steps to prevent the patient from being harmed by the process of targeting a specific biomarker. For instance, patients on a checkpoint inhibitor that targets cancers high in PD-L1 may develop symptoms of autoimmune diseases, such as colitis. “The goal is to find the specific biomarker that every single cell expresses without compromising the normal cells,” Dr. Bonilla says, “because once you tell the immune system to kill a population of cells, it is going to kill all those cells, whether they are good or bad. But if you are able to find the specific biomarker that is the hallmark of this disease and needs to be eliminated, then it’s much easier to find a therapy.”
The discovery of biomarkers has led to game-changing developments in the cancer treatment. Women who learn they have BRCA mutations are now empowered to make potentially life-saving decisions to prevent breast and ovarian cancer. Men with slow-developing prostate cancer can now actively monitor their disease, in part, because their PSA levels can be measured. And research is ongoing to find new biomarkers to help in the treatment of other cancers and diseases, such as diabetes, Parkinson’s disease and heart disease.
“Biomarkers offer an opportunity to apply genomics to population health and see what diseases or conditions people may be predisposed to,” says Pamela Crilley, DO, Chair of the CTCA Department of Medical Oncology. “Am I going to get diabetes? Am I going to get elevated cholesterol? Is there anything I can do about it? Look at hereditary breast and ovarian cancers. The science has led to being able to prevent disease in patients with BRCA1 and BRCA2 mutations. Now we may be able to significantly reduce your risk of disease.”
https://powerfulpatients.org/pen/wp-content/uploads/1-10.png600600PEN Editorial Staffhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngPEN Editorial Staff2019-10-01 08:00:492020-01-29 10:55:08Identifying Biomarkers Gives Doctors Known Targets to Treat Many Cancers
Three experts discuss the clinical trial process and the difficulty in participating in a trial. Our expert panel includes:
Ken Getz, MBA – Founder and Board Chair, CISCRP
Andy Lee – Senior Vice President and Head, Global Clinical Trial Operations, Merck
T.J. Sharpe – Melanoma Survivor and Patient Advocate
And greetings from Southern California. I’m Andrew Schorr from Patient Power. And welcome to this Patient Empowerment Network program, another in our series of Clinical Trials MythBusters. Our goal, of course, is to help you get the treatment for you or a loved one that you need and deserve. I want to thank the financial supporters for this program to the Patient Empowerment Network; AbbVie, Inc., Celgene Corporation. Daiichi Sankyo and Novartis for their support. They have no editorial control and we’re going to have a very freewheeling discussion today. And really what it’s about is how can a clinical trial be made easier for you to participate? Are there barriers? We’ve talked about it in previous programs. But specifically, what are the companies—the pharmaceutical industry mostly, who sponsor trials all around the world, what are they doing to make trial participation easier? For you to know about trials. For the people at your clinic to know about it and what to say and how to administer it. For you to have documents that are understandable for you and your family to know whether you want to participate. To keep you informed. And also related to the requirements of trials. How can they be relaxed a little so that there may be a trial that would benefit you, that you and your doctor agree on, and the requirements of it allow you to be in the trial. Okay, and the logistics of it are not so tough either. All right, I’ve been in two clinical trials, and I believe I’m alive today because of that. So, I’m very grateful. We have some wonderful panelists with us over the next hour. Now as you have questions, send them to email@example.com. And some of you have. So, you’ll be able to interact with us as we go along. First, I want to go to Ft. Lauderdale, Florida, and T.J Sharpe. And T.J. has been on programs with me over the years. Stage four melanoma patient having been in trials. And T. J., you would agree, you’re alive today because you were in trials, right?
Absolutely, Andrew. I think both of us are very fortunate that we found a trial that was the right treatment for us and gave us the ability to combat our disease in areas may not have been available to us if we just waited for standard of care therapies.
Right. And here you are—we should say that you were diagnosed a number of years ago with melanoma, went through trials. And now you’ve had two years without treatment, right?
Yes. It’s been five years of treatment followed by now two good years of a clean bill of health.
Well, great. And I should mention for our audience, many people are familiar with T.J. T.J. goes around the country, gives speeches. He’s been at many events, consults with industry that are developing trials to try to bring the patient perspective forward. So, T.J., thank you for all you do. We really appreciate it.
You’re welcome. It’s my honor to be able to represent all these patients.
Well, most every family—certainly most have been touched by cancer. But our other guests are not cancer patients but are in national leadership programs. And so, let’s go up to Medford, Massachusetts at Tufts University outside Boston, Ken Getz. Ken, welcome to the program. Ken, ladies and gentlemen, is a true national leader when it comes to clinical trials and really helping us move forward with better processes, better understanding. Ken, tell us a little bit about your organization there, CISCRP. What does that stand for?
Thank you. And I have to say your pronunciation was nearly perfect. It’s hard to pronounce it. It’s an acronym and it stands for The Center for Information and Study on Clinical Research Participation. It’s a non-profit organization. It was founded 18 years ago. And it’s really there to help patients and their families navigate the whole clinical research progress which for many is completely unfamiliar terrain until they’ve been diagnosed with an illness or when they have exhausted all other treatment options. So, CISCRP really helps people become more educated and informed so that they can really think of the clinical research process with more confidence. And they can navigate this unknown terrain.
All right. I’m going to come back to you in a minute because you have such an overview, and you’re also an Associate Professor at Tufts. And so, you study all this, and you’ve written books. But I want to introduce the third guest. And that is a leader from the pharmaceutical industry and one of our most respected and venerable companies in the field, and this is Merck. So, joining us in a senior vice president of clinical operations there around the world. And that’s Andy Lee. Andy, welcome. Thank you so much for being with us.
Andrew, thank you. And pleasure to be with some prestigious panelists, both of whom I know. And I’ve met you over the last two weeks. And thank you to T.J. and yourself who have been trial participants and who are representing that part of the organization.
Okay, and we should mention that both T.J. and Andy are working on a couple of levels. And Ken sounds off on this too. There is a group called TransCelerate where pharmaceutical industry is working together on some of the issues they face in having the proliferation of trials. More trials sites, more accessibility, procedures for that. And then, of course, Andy has helped lead that effort at Merck related to breakthrough therapies that they have been trying to develop there in supporting patients who might be in Merck trials. So, we are going to come back to that. But I want to go to you for a second, Ken. Ken, how low is the participation among adults in clinical trials, at least in the U.S. Now, I’ve heard really low percentages. Where are we now with that?
Right, it’s a great myth for us to start with, this notion that only three to five percent of patients—eligible patients, participate in clinical research. That’s actually a statistic that was published by the National Cancer Institute in the early 1990’s. The latest research really shows that it varies widely. For example, when we look at pediatric cancers, the participation rates are extremely high, 80 to 90 percent in same cases—pediatric leukemia. In part because those communities have very engaged healthcare providers, very engaged families that really share their information. It’s just an enabled community where all of the stakeholders support participation. And then there are other areas of course. Some cancers where we do see relatively low participation rates. But I want to point out that low participation is driven by so many factors, Andy, including the strict eligibility criteria. And the demanding protocol designs which are a real burden for some people, and they choose not to participate. As well as low awareness, very low accessibility to trials among minorities and underserved communities. So, there are many factors that contribute to this variation in the participation rates.
Yeah, you’ve ticked off some now. T.J., in your own experience, one of the breakthrough trials you were in you had to go from Ft. Lauderdale in South Florida and move your whole family to Tampa in central Florida, right. I mean that was a big deal.
Absolutely. When you have a young family and a stage four cancer diagnosis, relocating simply across the state during the holidays especially, is no big deal. We were fortunate because we had the means to be able to move there with work situation, with family. But too many people can barely go across the county, much less the state or the country to find a trial that might be the best match for them.
Andy, so we’ve ticked off some of the obstacles, and Ken touched on some about even the proliferation of trials. Is that a lot of what you do is how can we have trials be more accessible, be more widely distributed to a clinic near you?
Yes, let me just explain. When we look at a new cancer therapy, we look at the various cancers that may be affected. And what we do is we go for high probabilities of success. And the challenge is if you bring a new cancer agent. You normally start off in very advanced disease. So, patients would have failed multiple lines of therapy, and often it is a last gasp. And you have to show some sort of clinical efficacy. And then you move sort of backwards in the disease, and you go from sort of third-plus line, second line and first line.
And then you may work downwards into earlier stages of the disease into an adjuvant setting and maybe a neoadjuvant setting. So, as we sit down and design a trial, what we need to look at is what is the population that is most likely to show any benefit at all. And quite often when you are developing a new therapy, it’s difficult to show benefit because many of the patients are very ill. So, what we have to do is optimize the opportunity for success of a compound by going to the right target patients.
And quite often as we have learned a lot more about cancer, this does not mean we test a product broadly in anyone with cancer. We typically try and find a profile of a patient that is likely to respond. And many patients now will realize their predictor biomarkers or prognostic biomarkers. So, for example, with immunotherapies, those that work through the PD1 mechanism would probably want to have a PD1 ligand receptor positive patient who is likely to bind to the drug.
And that gives a higher probability of success. So, it sounds counterintuitive that while we want to develop therapies for all cancer patients, when we start clinical trial development, we have to show efficacy in a population that will benefit. And that’s normally predefined and makes the inclusion criteria fairly strict. As we show efficacy and as we can move into broader populations, it makes it a lot easier for us to design more liberal clinical trials. And then we can actually spread those in the geographic domains.
I could talk more about geographic allocation, but let’s hold that for the time being, and let’s see if there’s time later on.
Can I just add to what Andy said because I think it’s really important for your viewers to understand just how active drug development activity is today. We’re looking at over 4,000 pharmaceutical and biotechnology companies, some of them very, very small. But in total, we’re looking at nearly 6,000 drugs that are in active clinical trials. And to Andy’s point, many are really targeting a patient with a very specific genetic profile or a specific biomarker. But it should give anyone who believes that a clinical trial may be an important care option for them, they should recognize that there may be many, many trials out there.
In total we estimate as many as 80,000 clinical trials, nearly 50 just conducted in the U.S. alone—50,000. So, it’s just important that we keep all this activity in perspective.
Right. So, T.J., that’s why all of us as patients need to ask about them, right? Go to different resources, whether it’s an advocacy group that you ultimately spoke with other patients, and obviously quizzing the doctors we go to. Is there something that may line up with my situation, right T.J.?
Absolutely. There is a both top down and bottom up approach here that patients as they become educated—and every patient should be the owner of their healthcare as they become educated. Hopefully they are coming across advocacy organizations, other informed patients, patient support groups—all of which will help inform them different options for disease treatment, including hopefully as Ken mentioned, clinical research as a care option. At the same time, there is certainly very much an opportunity from the top down from the sponsors who develop the trials and from the sites that execute them to educate patients as they come in.
Not just at their own site, but at any site, at any medical facility. That if you have a diagnosis and you are looking into your care options, that you should be asking the question. And we should be giving you more information on the possibility of clinical trials and where you may find clinical trials that are appropriate for you.
Right, the whole enchilada, if you will, of all your options. Andy, so you mentioned about trial requirements. So, first of all, what efforts either at Merck or are you aware in the industry are being made to really talk to patients early on as you are designing trials? Whether it’s the requirements—how many CT scans you’re going to have. How often you are going to have to go to the main trial site. All the different things that sometimes get in the way.
Well, firstly we start with design. And we believe in exquisite trial design, quality by design as well. So, what we want is to run the experiment once and not have a sloppy trial design. We want to make it really robust in terms of scientific integrity and operational execution. So, we have a lot of internal design committees and what we do is we co-op with many groups external to our company. So, we speak to people who run clinical trials at cancer institutes.
We speak to the doctors who manage this. We speak to the trial coordinators. We speak to people involved with the transporting and shipping of medicine how they would do that. And then we of course speak to people in the ecosystem. We quite often speak to investigational review boards before we start trials. We talk to them about our design and what would be best to protect the rights and well-being of patients. And then, of course, the patient-centric approach says that we need patient insights.
And I’ve chosen my words very carefully because the insights are really important. Not all patients—and I’m very respectful that some patients are very intelligent and actually may be involved in this. Some patients can contribute to design, not all can. And so, what we do is we take the insights and we impute those. We often have focus groups. We talk about this disease. We talk about the burden of the disease. And then we talk about how that disease is managed in an ecosystem. And quite often in different countries it’s managed differently.
And so, we have to appreciate the global clinical trials have to navigate a path that may not be a linear path as we’d see it at an exquisite elite cancer center in the United States. It’s community-based, it’s all the rest. So, we take that input, and what we try to do is unburden the trial for the patient. We say, “How can we design a trial that requires the least visits to the clinic—the hospital, the least burden for them. And how can we take some of that burden from the clinic and actually transfer that into an easier environment.
So, document reading and review. Perhaps filling in questionnaires about quality of life. These are things that don’t have to be done in the clinic itself. And then often when we work with clinics, we work with them to help them understand how we as sponsors can make their life easier. And some of those things might be simplifying the informed consent. But I want to stress just one point here is that we can do whatever we like in the design at a company.
One of the things is, the patients are not sponsor patients. Okay, we sponsor clinical trials. The patients are managed by a doctor and a professional. And underneath that principal investigator is a whole oncology team. And it involves radiology. It involves pharmacists. It revolves around a 360 multidisciplinary team. They’re exquisite. They help manage the patient, not the sponsor. We provide the enabling functions for them. And then also that the oversight of the patient’s right, safety and wellbeing is the responsibility of an institutional review board.
And while we may provide templates and simplify templates in text and language, we rely heavily on the institutional review boards to help us with things that may make things easier, such as reimbursement for parking, transport, all of these things. And by and large, the institutional review boards are very supportive of these things. But they are very difficult to quantify in exact terms because of different geographic regions and different norms in different places. So, we rely heavily on exquisitely well-trained 360 team who manages oncology patients with a great PI. They manage patients.
And we work collaboratively with the sites who work with patients on our behalf. So, I just wanted to say the myth is that sponsors interactive with patients. That’s a myth. And the truth is that we engage with clinical sites, and we try and make our design and all the elements—the enabling elements, simpler for the trial sites in order to manage the patients in a simpler way.
Okay. Thank you for that. So, Ken, I want your comment on that. Because okay, we are downstream patients. We have a doctor, healthcare team. And we know somewhere in the background there’s a sponsor that tried to enable good things to happen to get reliable data and hopefully a cure for us. So, how do we—what’s happening? Are we improving things there in that interaction between clinic and patient?
Yes, we absolutely are. And I’ll start by just echoing and acknowledging that Andy has really laid out just an incredible amount of input that goes into the design of a protocol. And that’s really for a really large company. We see many, many examples now of patient advocacy groups or smaller companies turning to a variety of approaches to solicit input from patients and healthcare providers. Some virtual approaches through a social media or digital community. So, there’s lots of ways that feedback is being channeled.
And that’s really important. The flip side, to really answer your question, is that our protocol designs are becoming more and more complex, more and more demanding. A much larger proportion of drugs are now targeting rare diseases that have been stricter inclusion and exclusion criteria. And the designs of the studies—the number of procedures and the number of visits. The number of investigators that are involved, all of that has also continued to grow. And as a result, we do see that our trials are taking longer.
We have yet to see a year when we actually witnessed a reduction in the cycle time to conduct a clinical trial. And we just have to figure out new ways of making the participation process less burdensome and more efficient.
Oh, my. So, T.J., you had been living with stage four melanoma, a life-threatening condition. We have people even on our team who are living with stage four disease. So, when Ken talks about things slowing, that’s not what we want to hear. We want to hear two things. One is, we can accelerate a development of new medicine. And ideally—because this is an issue certainly in the U.S., but I think worldwide, that by speeding the process, cutting through red tape, improving procedures and us participating, the cost can be less as well.
And when we talk about cancer, the costs are going through the roof as you know for people living with chronic cancer. And you know so well, Andy, people who are on some of the medicines that you’ve come out with at Merck. Where people used to die unfortunately in short order, are living a much longer life thanks to new medicines. We want it to happen faster and be financially achievable. Andy, any comment about the pace of science?
Yeah, I would like to make a couple of comments about that. We often hear the sort of story that 80 percent of clinical trials don’t recruit on time, et cetera. We do immense feasibility. Once we have designed a protocol, we send it out to all of the countries that could potentially work with us. We have staff in 47 countries. And they look at two areas of interest. One is the medical durability, is the comparator the one we use in our country. Is the protocol designed the way we practice clinical medicine, not clinical research medicine?
And will that enable us to recruit the patients? That’s the first level. The second level we look at is to ask the question, is this operationally feasible? Can we source the comparator? Do the clinical sites have the equipment? How would we have to ship the biological samples around the world? And based on medical durability and the operational durability, we do a site selection. And we run the indicators through a Monte Carlo simulation. And we simulate this trial. What if we took three countries out? What if we added this more sites? What if we changed this inclusion?
And we come up with a model of what the recruitment would look like. And recruit about 80 percent of our trials according to our model. So, about 80 percent of our trials recruit on our model time. And then if we look at the typical time for drug development, it has been from eight to 10 years for many years in the industry. And when we look at some of the development timelines now—the cycle times. Pembrolizumab (Keytruda), for example, from first study until first approval, was 60 percent reduction in time.
We were looking in the four-year time period. And we are looking at five or six years for many indications. And so, we’ve halved that cycle time for some of the newer oncology products. And there are a number of reasons we’ve done that. One is we have found operational efficiencies. Two is the trial design has enabled us to interim analysis with independent data monitoring committees to assist with that. I’d also like to put in a positive plug for the regulators.
I do believe—and I’ll talk specifically about the FDA, because they are the agency for the United States. They have revolutionized the way they approach the designs and the way they review the data. And they have breakthrough designation status they’ll give to compounds that are really looking like they have strong efficacy. And so, the approval process through the agency has improved remarkably. And they’re open to adaptive designs. And they are open to interim analysis. And they are open to all sorts of things.
So, I really wanted to give credit to our agency who has said, “Where there’s a need for breakthrough medications, we’ll try to find the path.” And so, I do believe there’s a real positive side to this. The challenge is the market is saturated. We have now more than 25 PD1s in development. And to put the 25th one in there, they are so far behind in development. I wonder what that does. It clogs up the system. So, when you look at how can we influence sites, at the top sites we only get one or two patients.
And we compete with 50, 60, 70, 80 other sponsors. And so, it becomes so saturated that, that site has to learn to do systems and process with 70 companies. And what they are doing is almost hedging. They are not focusing on certain things. So, in those cancer centers, they offer treatment for all lines of therapy and all types of cancer, the specialized and nonspecialized. And we are moving out of that sort of geography and moving it community-based oncology practices where it’s less saturated, and we can actually have more traction there and be able to engage more with the clinical trial enterprise for the good of the patients.
Ken, you write books about all kinds of issues around this. So, if we are getting—particularly in oncology to have trials offered at the community practice where those doctors work night and day—the nurses. They are really stretched. More and more cancers, genomic subtypes, most sophisticated testing. How—what would you say the patient can do. T.J. talked about it a little bit. What would you recommend to patients so that at that community oncology practice the patient and the family can kind of discover what may be available for them as Merck and other companies try to get these trials distributed?
Right, well you—talk about the whole enchilada, Andrew. You’re really touching on it. It’s also very exciting times for patients, not just cancer patients, but patients that are dealing with any chronic and severe illness today. And it’s really all about more of a partnership with the clinical care environment and clinical research. And of course, at the heart of it is the patients and their family being as informed as possible, sharing their electronic health and medical information so that they can be connected to trials that might be appropriate for them.
But it’s moving—as Andy said, away from the classic places where trials used to be conducted. And in many cases, they were at these dedicated centers that only conducted clinical trials. It’s a very competitive environment now for patients. So, many sponsor companies like Merck and others are looking at clinical care settings and moving into communities or, in some cases, large health systems where you can have clinical research professionals who will supplement and provide support to the healthcare providers, so they’re not stretched too thin.
But so that they have the clinical research capability onsite at the point of care. For patients it’s a great opportunity because now they have the opportunity to get their own healthcare or treating physician and treating nurse involved in a clinical trial as part of their overall care. And we expect to see more of that over time. We expect to see other virtual trials or opportunities for patients to participate in the comfort of their own home tied in with their clinical care setting.
And all of this is relatively new to the whole world of clinical trials and the investigation of experimental medications.
You touched on something I just want to follow up on. I’ve heard of this term site-less trials where you said you participate in your home. So, T.J. had to go from Ft. Lauderdale to Tampa. I had to go from Seattle to Houston. There are not—this is a big deal, especially if you have little kids as I did, he has. So—and away from work and whatever your situation is. So, is technology going to come in play so Andy can get the data he needs for the FDA, but that we can have technology help accrue that data in a more efficient way.
And I’ll say absolutely. And my colleagues here today I’m sure can comment on this as well. But absolutely. We are seeing wearable technologies and mobile applications that now have the ability measure vital signs and other important baseline information in a validated manner. There are ways that you can access a specific facility for a highly specialized test, specialized imaging for example where the technician can evaluate it remotely. Blood can be drawn at remote locations as well.
So, there are lots of places where we have sort of this more flexible environment that can cater more to the patients and less about a specific physical facility where you have to go to participate in a trial.
T.J., I want to talk to you about diversity. So, you and I are kind of middle-class white guys. But we want to know how new medicines work for a variety of populations, ethnically, economic groups, et cetera. And Andy needs that data. And he goes to the FDA, and the FDA says, “Well, do you have Hispanic people? Do you have Asian people? Do you have African American people?” or whatever the country is because he works globally. And they say, “We want to understand are there differences?”
How are we doing with that. How can we make a difference there so that we really know what medicines make a difference for broader and also distinct populations?
I’m sure Ken can back up some of these things with more hard data than I can. I know that different populations have different levels of trust with the medical system. One thing that you and I both experienced was a lack of options—a lack of good options. And when you get into dire straits, you tend to be a little more trustful of anything that comes along. But we have serious or chronic conditions that have proven treatments that might not be the most effective for certain populations.
And we’re not able to broad the scope to these minority populations or populations that don’t have access to NCI designated cancer centers or top-notch medical facilities. They are not able to get either in a trial that is looking for a drug that would help them or even get access to medicines that have been recently approved simply because their healthcare situation doesn’t allow it. Whether that’s a lack of insurance, a lack of healthcare literacy or simply a mistrust of—there’s a lot of generational mistrust I think in some communities of the clinical trial system.
So, as an advocate, I certainly push caretakers especially—and children caregivers for older populations who are maybe first or second-generation Americans to help facilitate a conversation between the medical professional who’s trusted and a patient that might not be able to get or rely on the information they’re given. Because it really will speak to populations that don’t get the opportunities that you and I have gotten simply because they are either not aware, or there is a barrier there to get to that medical professional.
I appreciate, T.J., you mentioned CISCRP. That’s one of the things that we’ve focused on for 18 years is bringing clinical research education into major metropolitan areas around the U.S. and parts of northern and western Europe where we plan for several months, and then we put on what we call an Aware for All events. And we really work very hard to encourage participation by—or from patients based within minority or underserved communities.
And I’m happy to say that we’ve had a lot of success with that. These are really difficult communities to reach through a lot of the traditional approaches. We have to rely on community centers and clergy and other approaches to really help these communities, for a lot of the reasons T.J. mentioned, trust the educational information, and come out to learn more. And I’m happy to say we’re seeing more and more people of diverse backgrounds that are curious and interested in learning more about clinical research, especially knowing that representative populations provide more information that can inform treatment for different types of patient sub-populations.
I want to go to Andy in a second. Andy, just one second. I wanted to mention and call out—and Andy’s company has been a leader in this. He was talking about PD1 and all of that. But drugs that have been breakthrough in immunotherapy for people like T.J. where—and it’s being explored in broader cancers where otherwise life was going to be short. And how to activate the immune system and really fight the cancer in people living long term. So, the people in those trials—and certainly there were people in the melanoma trials like yourself T.J.
Lung cancer trials and increasingly now others who did get tomorrow’s medicine today. Andy talked about accelerated approval which is great. So, that’s the impetus for the patient and the family. Is there the chance to get tomorrow’s medicine today? Now the obstacles may be distrust. You talked about that, Ken. And also, is maybe accessibility. Is it as a clinic near you? And Andy you talked about pushing that out. And then sometimes it’s related to cost.
Now is there anything that sponsors can do, Andy, related to the costs that people may have in being in certain trials? Where do we stand with that?
Yeah, so I’ll just touch on the distribution first and then get into the costs because they are linked. When we prosecute global trials—we’ve had a very U.S.-centric discussion so far. But cancers present differently in different geographic regions of the world. And so, when we want speed out of our trials. You want me to shorten that timeline and get drugs to market quickly. I do it internationally and in some cancers like esophageal cancer or some of the gastrointestinal cancers, Asia has a much higher prevalence of these cancers.
And we do a greater proportion of work there. We always include multi-country studies. And U.S. may have a greater proportion in other areas. So, we balance that out to optimize speed. Of course, with clinical trials the cost structure around the globe is very different. But let’s talk about U.S. We have spoken about a saturated core of clinical trial sites that we all go to. And I speak generally now for all sponsors. And we are all looking to optimize and get great efficiency.
At the same time, we realize we have many underrepresented geographies and ethnic groups—and not just ethnic groups, but under resourced populations. And so, what we’ve been thinking about is how can we support people, and support people at all levels. And so, we start off with thinking about the cost structure, and we obviously pay clinical sites for what they do. But we will support all sorts of things. We’ve been negotiating with Uber and Lyft, so we can build that into automated transport for patients.
Again, the IRB has to approve that. We are looking at ways to augment that they are not out-of-pocket for things. And we’ve been talking a lot with a group called Lazarex Foundation who has really expanded into under resourced communities and found ways to ensure that they have daycare and different access for those patients. We have worked extensively now to look at outreach programs into communities that typically wouldn’t be in trials. We are focusing in two areas right now as we speak.
One is next generation of HIV medicines, and the other one is in prostate cancer. And we’ve got a large program rolling out in prostate cancer. So, what we are doing is going into sites and we have put together training videos and training materials. And we are looking at cultural competency. So, it starts at the site. Are they culturally competent to engage a different community? And we’ve spoken about working with the community churches, community education systems.
And so that starts with cultural competency. I have a woman, Madelyn Goday, who works on this day and night in my organization. And she’s very strong at this. It’s early days, but if we can show that it works in one or two therapeutic areas and cancer types, we’d expand it further and further. But we can’t just have a shotgun approach and just go and do 100 sites and hope it works. Hope isn’t a good strategy. We are working systematically to engage different people. And as appropriate and approved by ethics committees, we will support all of these communities and help build infrastructure and capacity.
Those are important things for us. But as I said, where appropriate and where it’s sustainable. We can’t just throw money at something in the hopes something sticks. We have to have something sustainable and it goes to what Ken says, and that’s education and providing resources and materials. And we’ve used quite a lot of Ken’s materials in multiple clinical trials. Thank you for that, Ken. It’s been really helpful for us.
Great. I wanted to note for your audience. If you have a question, send it to firstname.lastname@example.org. We have expert panelists here. And this is really—we are all in this together. I think you hear the dedication from Andy at Merck and T.J. as a patient advocate and Ken as a professor and founder of organizations devoted to this. We want obviously accelerate medicines, but have the accurate data of how it affects different people, who is it right for so that the regulators—and thank you for what you said about the FDA here in the U.S., has the information to make a decision on should this medicine be available for people with that diagnosis.
Okay, so what about staying in the trial. So, T.J., how long—let’s take with the Keytruda trial or one of them. How long were you in to for?
Nearly four years. Three-and-a-half years.
Were there ever times when you said, “I’m done. I want to bail out.” You know.
I’ll be very careful how I answer this question for Andy’s sake.
It’s okay, T.J., we’re friends.
No, probably the biggest crossroads I ever came to was when one of my tumors started growing about a year into it. And we weren’t sure if the medicine stopped working or not. We didn’t know what to do. And as it turned out, it was still working. And I think was just one spot that wasn’t responding. But everything else had responded great. However, at the point, as a patient, you’re thinking about yourself first and your family first and the trial second. It’s easy to stay compliant on a trial when things are going well.
But when you’re ahead of the medicine in some ways, and I think patients with chronic illnesses or in some cases rare diseases, are almost more knowledgeable than some of their doctors or the trial protocols about when they’re stopping. They don’t have the luxury of finishing out a protocol and seeing where their disease journey takes them. And the best example I can give of this is a very passionate advocate by the name of Jack Wheelen who we unfortunately lost a couple of years ago, but whose influence has kind of dominated the patient advocacy world for the last decade or so.
And Jack was able to monitor his health almost better than a doctor. And he knew when his trials weren’t working. When we get to that point in a clinical trial setting where we know the medicine is not being effective or where a patient would be better served to move on to another treatment. That’s when we are going to take the next step in clinical research, because now we’re aligning the trial design and the trial goals with a patient and a patient’s family’s treatment goals. And as those two points merge, that’s where clinical research becomes that much more effective as a care option.
That was well said. And I think with all those trials, you’re right, the team—that care team, what’s right for you at that time. Obviously to get the data, but also not at all costs. In other words, if the data is showing something is no longer effective for you, is there another treatment or a trial? I’ll just share my story for a second. So, I was in a phase two trial of combination therapies—which are increasingly common certainly in oncology. And after three months—halfway in the trial, my blood was kind of cleaned up.
And I had nausea and some other side effects. And I said to the trial coordinator, “You know, I think I’d like to stop.” And she said, “You know, our belief is that you still have microscopic illness in your bone marrow—in this case with the blood cancer, and the additional three months in this protocol will make a long-term difference for you. That’s what we believe.” They didn’t have the answer, but that’s what they believed. You know what? I stuck it out. She was right. I had 17-year remission.
If I’d stopped after three months, would I have? So, it’s a dialogue with the care team Andy, right? It’s this ongoing discussion not just entering the trial, but remaining in the trial, correct?
Yes. Absolutely. And I just wanted to impress a really important thing. People talk about people dropping out of trials. In cancer trials we see extremely low drop out. I mean these are potentially lifesaving medicines for all of the companies. But what we do want to make sure about is that when there is progression of disease, and it’s shown that the drug—whichever it is, the control arm or the active arm or the new agent, where there is progression of disease that they get the best available therapy.
And so that often contaminates trials because we have the crossover effect that now they are getting maybe the experimental agent in the standard of care type of thing. But most important thing for us is to track the survival of the patient, regardless of whether they go on another therapy. And we have put a tremendous amount of effort into looking at the informed consent and making sure we work with IRB to track patients long term survival.
Because as you’ve said, you may have a short-term issue that shows that the drug may not be working short term, but long term it may have prolonged and profound effects. Positive or negative, we don’t know that. And so, what we like to do is get long term survival. And we ask patients to consider when they sign the consent for whatever trial and whichever sponsor is sponsoring this, is to consider that knowing their status throughout their treatment—whether it’s on a sponsor’s drug or another sponsor’s drug or x therapy. It is really important — and I ask people to think about that.
Because that really helps us get as much data out of the individual treatment as possible. And that may prevent nonrequired trials in the future or it may say, “Wow, that really informed.” And we’d like to inform all cancer patients. If data we generate can inform other therapies, we certainly want to do that. We do not want to do wasteful clinical trials. So, tracking patients long term or patients—the message to patients is being cognizant of letting the sponsor—and the sponsor could be an institution. Letting them know your status is really important. All they want to know is are you dead or alive.
In the end, just one thing is, are we partners. In the end, our viewers here, are we your partner? And can we feel that not just for their doctor but you guys behind the scenes with the labs and everything, that in the end we are partners. And unless we see it that way, we won’t get anywhere.
Absolutely. I’m glad you used the term partners. Because when we’ve done a prep for this people have said, “Are they investors in the thing?” So, yes, patients invest their time and everything, but they are partners in research. They are contributing so much. They are contributing—they are going into the absolute unknown. And there is an immense trust level that is there. And we owe that back as research professionals is to treat people with respect, dignity and as partners, to make information available, to publish our data to get it out there as quickly as possible. And to make sure we get that back into the participant’s sort of hands.
So, Ken, how are we doing on that because you go back over the years and people say, “I don’t want to be in a trial because I’ll be a guinea pig,” and respect was not seen as part of it.
Well, that’s also a bit of a myth, right? You had a few that claimed that they felt the process made them feel like a guinea pig. The vast majority of people, over 90% of people who participate in a trial, would do it again. So, once they get past that unfamiliar area where they’ve perhaps only heard a few case examples or a few very vocal people who had bad experiences. Once they’ve done it themselves or they’ve been able to work with a group of advocates that really help them think about this process, and they become more educated, generally they’re very impressed with the level of professionalism, the compassion that exists at all levels.
I work with so many professionals—science professionals and pharmaceutical companies and at the research centers, and they all share that kind of commitment that Andy just mentioned. There’s a real desire to partner with the patient to really inform them. I would say one place where we need to see much, much more however is in the return of clinical trial results in a plain language to people who’ve been in trials. That’s a place where as an enterprise—government, research sponsors as well as industry have not really made this a standard practice at this point. And that’s one thing that we’re really working on actively.
Right. Great. So, T.J., you and I are investors—and Ken used that term and Andy used it, and I’ve always believed it. We are investors of our tissue, our body, our future to help other people and hopefully help ourselves. And certainly, for profit companies that may greatly benefit if they have a blockbuster therapy. But we need to be kept informed in the long term, right T.J.? We want to know what a difference our participation made.
Certainly. And I think to echo what both Andy and Ken said is that patients do become partners. Patients who are involved in clinical research, a significant chunk become altruistically invested. I’ve heard more than once, “Even if this doesn’t help me, I’m glad I participated because it might help somebody else.” I know I’ve felt like that, and I’d venture that you’ve had some of that too, Andrew on your journey. So, it’s only—it’s at the very minimal fair, and it’s certainly very justified to expect as a co-participant in this.
And as kind of a co-creator of science with sites and sponsors that we understand what has come of our sacrifice and our time dedication to helping science out. We shouldn’t have to find it out through press releases from ASCO or hope that we hear about it on the nightly news. We deserve to hear what has happened. Not just because it can affect us as people and as patients, but that we put a lot into this too. And then we did our part to further medical research and we want to be part of the—whatever the end of the trial ends up being. We want to be aware of that. Not just for personal knowledge, but to know that it’s going to help this many other people.
Right, to be honored. So, Andy, at Merck you’ve established some internet platforms in particular related to keeping people informed, right?
Well, we’ve got an internet platform that people can log onto. I’m happy to share that with you; in which they can get access to a list of our trials. So, I didn’t prepare this but especially, but I did make a handmade note. And if anyone wants, it’s a very simple log on. Andrew Schorr:
You’re a great artist.
And it’s a simple one. What that will get you access to is two main important things. One is it gives access to information about clinical trials. We have a tab on there that tells everyone about the phases of clinical trials and what to expect in a trial. So, it’s an educational part. Then we have a lot of information about the Keytruda clinical trials were, are running, and they’re called keynote trials. And there you can look at the different indications. And you can look up and it has a telephone number you can call.
Now I must stress is that we run over 1,000 clinical trials in oncology. But many of them are not sponsored by us, they are investigator sponsored trials. So, you can go to clinics, and they run their own clinical trials that are not sponsor-related. And the NCI runs their clinical trials. So, there are a lot of different sources. And many companies will have clinical trials. We also have the website clinicaltrials.gov. I’ve had to use that in the last two days for a colleague.
And you can navigate that and look for different types of trials. And you can look at different products and everything. It’s not perfect. But at least it’s a place to go to. And I don’t want to sound as if I’m one sponsor centric. Many other companies have access to websites, and they really want to try and enhance and direct people to the clinical trials sites at which they are working.
Right, absolutely. And then you were working at the industry level with a group called TransCelerate, and I know T.J. is involved too, to try and establish common procedures as you establish trial sites, as you have communication, as you have training, right? So, that hopefully all boats will rise, right?
That’s correct. TransCelerate is a group that formed about eight or nine years ago. There were 10 initial member companies. I was a founder member of that. And we got together to say, “We have to improve operational efficiency.” So, we do not collaborate on molecular structures and those types—that’s competitive. We collaborate on what we call precompetitive, procompetitive aspects which says, “If we all work together to improve something, we’ll all get the benefit of this.” And we share it publicly.
There’s a website, you can look at it. But we’ve looked at standardizing protocols. We have a common protocol template. We’ve adopted that at our companies, so have other sponsors. The protocol can be developed in a standardized way. We’ve looked at standardizing ways where we can improve monitoring. We’re looking now at ways that we can work with investigative sites through i-platforms, shared investigative platforms. So, a clinical trial site has to provide the information for us as a sponsor and use the exact same standardized questionnaire and information for any other sponsor through a standard portal.
So, we are trying to reduce the burden on clinical trial sites. And we’ve plugged away for many years, and we are seeing greater traction there. We are seeing more efficiency, more standardization. We are seeing greater quality, less rework. And so, while it’s hard to quantify this, what we believe is that the sites are freed up of some of the more burdensome things, and they can direct their attention towards patients, patient safety, and access to clinical trials. So, the work may not be directly related to access for a cancer patient into a cancer trial, but there’s a lot of tangential spin-off of making a site more efficient so they can put their resources and energy in the right place.
Well, thank you for that effort and your leadership. So, Ken, you’ve been around this a long time. And you’ve deal with all the companies and the government and the various agencies. And as you know, in some quarters there’s a distrust for pharma. We mentioned cancer that you get the price tag of a drug, and it’s very expensive. And some people are struggling to pay for it. And there’s just frustration about it. And often in the news media they are the bad guys who are called out for unethical procedure or something that went awry.
So, how are we doing there in overcoming that because we talk to Andy, he seems very ethical, dedicated guy representing a company that’s been around I think well over 100 years. So, how are we doing to move clinical trials on in this area when people aren’t sure what to make of pharma.
Yeah. It’s a huge issue, Andrew. And I think part of the challenge is that all it takes is one questionable behavior, and it makes it difficult for the reputation of the entire industry. Right now, we are dealing with major pharma companies that are actually being fined for having contributed—a judgement, having contributed to the opioid crisis. And when you start looking at some companies aggressive marketing tactics, right? It really sort of sheds a darker light on a lot of the great work that companies do.
What we look at, at the Tufts Center for the Study of Drug Development at the School of Medicine. We look at the overall output, the level of innovation that’s coming from the industry today. And we look at the number of complaints that have been filed with the FDA and other regulatory agencies around the world. And what we see is tremendous growth in the innovation and the quality of the innovation—drugs like Keytruda and other cancer immunotherapies. What an exciting area.
We see that the vast majority of companies really support and live by highly ethical, highly professional, highly compassionate approaches because they all know that it takes just one questionable issue that can really tarnish the reputation of every company operating in the industry. So—again, Andy also mentioned just how regulated we are as an industry, the fact that we have ethical review committees and data safety monitoring boards and so many other external agencies that help to oversee the work that’s done here.
So, I would say for patients who are thinking about clinical trials, it’s good to know the history. It’s good to know what you need to do to protect yourself. But the vast majority find that the people they deal with are ethical, they are professional, they are compassionate. And, as I mentioned, over 90% of people who get involved in trials say that they would do it again.
Thank you. That was a wonderful response. Andy, you mentioned earlier about starting research with the sickest people basically, where there are no options. But one of the questions that came in is, “Are trials only for the sickest people or are there of all those trials you talked about opportunities for people who maybe are newly diagnosed or could be their fairly initial therapy?
Yeah, great question. And thank you to the person who asked that. And the answer is that we start in people—because we don’t know if our experimental agent will work. And everyone assumes that new medicines are all going to succeed. And we work in research and researcher because of that many things fail very early on. They fail in phase one before anyone hears of it. It’s normally a code number at that point. And we may just not make the drug soluble enough, or it may not be distributed enough.
So, we may have a thing that works in a test tube or a petri dish. But to get that into humans and make sure that it’s safe at the dosage we use often fails. We just don’t progress far enough. So, what we want to make sure of is that firstly the drugs are safe. And there’s a trade-off between safety and efficacy. We’re constantly trading off. And so, what we do is we look at that and say when someone has no option and we want to get an option going, that’s where we start.
We’ve actually moved down the disease scale, and we’ve come into adjuvant treatment or secondary prevention. And we’ve gone into newer adjuvant is when you have a small tumor is we pre-treat to manage that tumor before surgery is done. And post-surgery we hope that there’s limited treatment or no treatment. And we actually have removed the cancer, and there would be no evidence of disease. But. of course, using the word cured is something we try not to do, because we prefer to use no evidence of disease.
But absolutely. And the next strategy is prevention of cancer. Our company does a lot of vaccinations in women’s health. We have a product that protects against human papilloma virus which is a precursor for cervical cancer. So, people who are vaccinated with this particular product—and I’m deliberately not using brand names for obvious reasons. But when you vaccinate for HPV, you essentially are preventing the likelihood of a cervical cancer. And there are now prospects in many disease areas where either vaccination or early treatment gives you a tremendous positive prognosis of not getting the disease later on in life.
The answer to your question is yes, we are absolutely looking at ways to prevent getting to a very advanced stage which is very costly to manage and very emotional and stressful and difficult.
I want to thank you. I just want to get a final comment on what you would say to patients or family member. And I want to start with you, Ken. What do you want patients right now to know so that—what tips would you give them so that they’d consider being part of clinical research or stay in clinical research and the benefit it could be for them.
I will say really two things. The first is there’s just a tremendous amount of information out there, and we recommend education before participation. So, do your homework and engage family and friends and people you meet and trust to help you make the decision. And the second point comes off of that. And that is this is not a decision you make alone. Really bring in your treating physician, your nurse. Bring in your support network. And chances are you will learn a lot, and you might even find a trial that is right for you.
Right. And Andy, what about you? A final point—what would you say to a friend or family member or colleague related to considering trials today.
We get this question every single day. And we get it from patients in need. And my answer is we are all patients. We are all going to face this as professionals in our job or professionals outside. And so, I say community of practice. And disease hits all of levels of society in all education professions, et cetera. And so, my thing is to encourage people to do what Ken has said. Work as a team. Get multiple inputs.
And I am sponsor agnostic. Get the best therapy that is available. And that may be the best care option—as I said, the ecosystem in which you get the care is really important as well as the medicines that you get. So, have the discussion. Trust the medical professionals, they are very skilled out there. They are extremely well educated. And I just urge people, “Don’t think on two clicks on Google you are going to solve what your treatment option is.” Really discuss it with people because not all the options are public, and there is not enough information available about how to manage the whole disease through the entire enterprise. Trust the professionals.
Well said. And T.J., you and I are alive today because of trials. What do you want—what’s the thing you want to leave our viewers with?
That they don’t have to be involved in clinical research. I think that’s an important distinction to make. And it’s going to pull together what Andy and Ken said that clinical research should not be considered a hail mary or last gasp option. If you are a patient—and we are all going to be patients as Andy mentioned. You want the best care for you. You want to be able to weigh all of your options. And if you are not considering clinical research, if you don’t know about it or aren’t able to get the information you need about it, then you are not going to be able to make the best healthcare decision long term for your health.
So, take that information that you can get. Find the trusted sources. Be able to reach out to advocates or colleagues or someone that you know that would have the disease or can connect you with good information. And be your own advocate—a little cliché, but really own that healthcare information. And once you are able to collect all of the different treatment options, then you consult with your professional medical team as to what the plan forward—the best plan forward for your individual situation would be.
Right. T.J., my friend, thank you. It’s a delight to see you again. Andy, with Merck, thank you so much for being with us and bringing your years of expertise. And, Ken, being at an independent non-profit center and also at Tufts University there, thank you for all the work you do. I want to thank the Patient Empowerment Network for pulling this all together. And the sponsors who supported us in this effort, AbbVie Inc., Celgene Corporation. Daiichi Sankyo and Novartis.
All these companies and I’m sure many more, working so that research can move forward. We can be true partners in it. And hopefully get tomorrow’s medicine today to make a difference for the community and live a long life, and hopefully a cure, right? I’m Andrew Schorr in California. Remember, knowledge can be the best medicine of all.
Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.
https://powerfulpatients.org/pen/wp-content/uploads/CT-Myths-1.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2019-09-17 10:30:152020-03-13 08:11:57Is It Difficult to Participate in a Clinical Trial?
Cancer patients are living longer as a result of clinical trials that test new treatments, therapies, procedures, or new ways of using known treatments.
Watch along as a panel of experts from the Diverse Cancer Communities Working Group (CWG) Sustainable Healthy Communities, LLC, Baptist Memorial Hospital–Memphis, and the American Cancer Society Cancer Action Network (ACS CAN) explore the questions:
What do patients and their support networks need to know about clinical trials as an option for cancer treatment if they are insured through Medicare or Medicaid?
What requirements differ from region to region and what is covered or not covered?
Hello, and welcome to this Patient Empowerment Network Clinical Trial MythBusters program on a very, very important topic, what impact does Medicaid or Medicare have on a patient’s ability to participate in a clinical trial. My name is Laura Levaas, and I’m the lung cancer community manager for Patient Power. I’m also a Stage 4 lung cancer survivor. I’m two years out from diagnosis, and I’m also on Medicaid. So, this is a topic that’s really important to me on a personal level.
This program is produced by Patient Power. We want to thank the following companies who provided financial support to make this possible. While they don’t have editorial control, we appreciate the support of AbbVie Inc., Celgene Corporation, Daiichi Sankyo, and Novartis for their support.
Today we are joined by some really amazing guests, the first being Mark Fleury from the American Cancer Society Cancer Action Network out of Washington DC, followed by Jeanne Regnante, also out of Washington DC, and Jeanne is with the Diverse Cancer Communities Working Group, Sustainable Healthy Communities, and last but not least, nurse navigator Laura McHugh from the Baptist Cancer Center in Memphis, Tennessee. Welcome to all of our guests today. Thank you for joining us.
So, Mark Fleury, Mark is interesting because he has an understanding, a very deep understanding, about this issue from a regulatory and research perspective. He’s going to share with us what he’s learned about barriers in clinical trial participation and solutions to overcome some of those options.
Jeanne is going to share her viewpoint as part of the Diverse Cancer Communities Working Group. She helps share information about access to care treatment and inclusion in clinical trials for underserved populations.
And Laura McHugh who is joining us by phone (she is a friend of a friend of mine, and she’s really amazing) is a nurse navigator who has worked in the cancer space for 24 years. And she helps guide people in underserved communities every day as part of her working life. She works with Medicare and Medicaid patients on the daily. So, we’re looking forward to hearing from her.
So, back to our program, patients are living longer as a result of clinical trials that test new treatments, therapies, and procedures, or new ways of using known treatments for new ways. The myth here behind Clinical Trial MythBusters today is that being in a clinical trial isn’t covered by medical insurance particularly for Medicaid or Medicare patients. I know for me personally I’m interested in being in a clinical trial and I’m on Medicaid but I don’t even know what that means. So, I definitely need some guidance.
So, as we’re talking about this today, if you have any questions about if you’re a patient yourself or you’re a support person for a patient that has cancer or any kind of disease wanting to know about clinical trials on Medicare or Medicaid, we’re here to help you. Send your questions to email@example.com. So, viewers who are joining us today thank you again. If you’re on Medicare or Medicaid, what do you even do if you’re presented with the option to participate in a clinical trial to treat your condition? Let’s talk with Mark Fleury. Hi Mark.
Hello Laura. Thanks for having me on.
Yeah. We’re so, so grateful to have you on our program today because you have such a deep knowledge in this industry and on this topic. Can you tell us real briefly what exactly you do for the Cancer Action Network? And then I’d like to talk to you about barriers around Medicare and Medicaid.
Sure. So, I work for the American Cancer Society Cancer Action Network. We’re the policy and advocacy arm of the American Cancer Society, and we focus on public policy, so that’s regulation, laws that impact cancer patients. And specifically, my work deals with policies around research and drug and device development, so how can we get those findings that happen in the laboratory into the clinic. And specifically, that goes through clinical trials. So, I’ve spent the last couple of years with a large partnership of other stakeholders taking a really deep dive into looking at clinical trials and all of the challenges patients have in getting themselves enrolled as a part of those trials.
Good. We look forward to hearing more. Can you tell us a little bit about the current state of clinical trial participation in the US right now?
Sure. So, there’s not real solid numbers, but we believe somewhere between 6 to 7 percent of US cancer patients participate in a clinical trial right now. So, that’s a fairly low lumber overall, and it’s also a fairly low proportion of the patients who would be interested. Research has found that between 50 and 70 percent of patients would say yes to participating in a clinical trial if they were asked. But unfortunately, many are not asked. And some of those who are asked are unable to enroll for a variety of external reasons. One of the things that we do know is that the people who do enroll in clinical trials tend to be less diverse and better off financially than the overall population with cancer.
Okay. What are some of the barriers around Medicare and Medicaid patients who want to get involved in a clinical trial?
Sure. So, obviously, first of all, there has to be a clinical trial for the patient based on your clinical characteristics. But assuming that that is the case, for a patient to enroll in a clinical trial, it’s critical that their insurance cover the routine care costs of that clinical trial. In other words, there are costs in a clinical trial that a patient would see regardless if they were in a clinical trial not. Say, for example, the first step of any treatment is a surgery and then the second step in normal care would be one drug but in a clinical trial it’s a different drug.
Well, regardless, you’re always gonna get the surgery. It’s important that insurance cover that routine part of the clinical trial. And unfortunately, historically, that’s not always been the case. Fortunately, in Medicare, they have covered that since 2000. That is not the case universally for Medicaid. And we can talk a little bit more about that later if you’d like.
Okay. Perfect. I would definitely like to follow up on that topic seeing as I’m a Medicaid person myself. Can you touch briefly on what actually is different between the two programs in terms of clinical trial, the actual coverage? You mentioned routine care; is that for both programs?
Well, so what’s important to note is that Medicare is a federally administered program. And so, there is one universal federal policy, and if you’re in Medicare, it doesn’t matter if you’re in Florida or if you’re in Idaho, the policies are identical. Medicaid is an insurance program that while partially funded by federal dollars, it’s administered by each state. And as such, each state has quite different policies. So, if you’ve see one Medicare policy, it’s uniform. If you’ve seen one Medicaid policy, it’s only relevant in the state in which you happen to be. So, it could vary significantly from state to state.
Right. And so, depending on your state, you would need to follow up with your local maybe human services office to get specific questions answered.
That’s correct. Yeah. There are some resources (and I think we can provide those at the end of the webinar) where generally speaking some states have passed laws or signed agreements in which their Medicaid programs have to cover those routine care costs in Medicaid. And we can certainly make available those states. But even within those states, it’s important to look closely at the policies. For example, in Medicare, Medicare also covers any adverse events. So say, for example, while you’re being treated, you had to be admitted to an ICU for heaven forbid a heart attack or something like that. Medicare pays for all of those unexpected expenses. And that coverage may vary state by state in Medicaid.
Okay. Thank you, Mark. We’re looking forward to those resources. And for those of you watching, we will definitely be providing a downloadable guide with all sorts of resources to help you. Thanks Mark.
Okay. I can’t wait to talk to you about this. I have so many questions. I feel like we could talk for an hour. So, aside from the myth, I came into this thinking, “I’m on Medicaid; I probably can’t get into a clinical trial when and if I get to that point.” And then also, “If I am, it’s probably cost prohibitive because I’m on a fixed income.” So, is participating in a clinical trial expensive or cost prohibitive if you’re on Medicare or Medicaid like I thought? I mean, I know Mark touched on some of the issues, but what would you say? How would you answer that?
For low-income patients, the cost of routine care and logistic support needed during a clinical trial is certainly a barrier to participation. And Mark pointed out some of these costs. But specifically in patients in rural communities, remote communities, aging population, children, patients with cognitive disabilities or physical disabilities. These are the same patients who have low access to care in general.
And covering the cost for routine care in a clinical trial and also the logistic support is a clear barrier to participation. So, there are clear barriers there, travel, housing, parking, paying for food, on having access to clinical trials not only for routine care costs like Mark alluded to but also logistical support being included in the clinical trials. So, all of those things are barriers.
And would you say that seniors are also part of this underserved population?
Absolutely, especially seniors that live alone, that are in remote rural areas in the United States. And remember, that’s 20 percent of the population, aging population, in those areas. So, clearly, we need to do better to engage those patients in care and also clinical trials.
So, is it possible for us to draw any conclusions about how many people are on Medicare or Medicaid right now in the US? I did a little bit of internet sleuthing mainly through the Centers for Medicare and Medicaid, and it seems like there – the numbers that I came up with were pretty high, and it’s almost like 40 percent of the population is on Medicare or Medicaid. And so, has it –
That’s absolutely true. Look at by the numbers, there is 329 million people living in the United States, and that’s according to the last census, which is a hot topic these days. There is 60 million people on Medicare, beneficiaries, and about 66 million people Medicaid. So, together, that represents about 40 percent of the population. And we have to remember kids. So, there are 7 million patients on CHIP, which is part of the Medicaid program. So, if you include percentage of people on Medicaid plus kids on CHIP, that’s 22 percent of the population.
So, then circling it back around to clinical trial participation, how can we connect the dots here?
So, I think one of the main issues is clinical trial sponsors and the clinical trial operations folks in the sites working together to do a better job of reaching out to patients, ensuring that everybody is asked to participate, and not just selecting the ones who people think can participate but asking everybody to participate and understanding the eligibility of all patients and working together to help to cover their costs to keep them in chart.
Got it. Mark, I’m gonna pull you back into the conversation here for a minute. Can you touch briefly on what’s happening in the news right now around Medicare and Medicaid that could potentially impact clinical trials? Or maybe, Jeanne, you can speak better to that.
I’ll let Mark take that one.
Certainly, so, Medicaid traditionally has been a program that has served primarily children in many states, children and pregnant women. Starting close to 10 years ago with the passing of the Affordable Care Act, states had the ability to expand Medicaid eligibility beyond those kids and pregnant women. And now we see many states who have expanded the roles of Medicaid recipients to healthy adults who just happen to be lower income.
And so, what that really has changed is the number of people obtaining their insurance through Medicaid. Obviously, there has been a lot of – it’s a state-by-state decision whether or not Medicaid is expanded. The Affordable Care Act as a whole is hanging in the balance in a court case, and there’s obviously been a lot of discussion about whether it should continue or not. So, certainly, the number of people who are supported through Medicaid is a dynamic number, and that certainly is subject to changing policies that are still under active discussion.
I will say that Medicare, again, the coverage for routine care costs in clinical trials for Medicare, long-standing policy since 2000 that has been relatively stable. And I would expect that to continue unchanged.
Thank you, Mark. And Jeanne, I’m gonna come back to you in a minute. For viewers that are watching, thank you for hanging in there with us. If you have any questions that you would like us to address in the program, we’ll get to that at the very end after we’ve talked with all of our esteemed panelists. Send your questions to firstname.lastname@example.org. So, now I would like to talk with Laura McHugh. Are you with us, Laura?
I am. Thank you so much for having me.
Hi. I am so excited to have you. I met Laura McHugh because she is a nurse navigator for a friend of mine who is ALK positive, which is the type of lung cancer that I have. And she works very closely with my friend and speaks so highly of Laura. So, I’m excited to have her on the program today. I wonder, Laura, if you could tell us why you think that clinical trials are important.
I wanted to share why they’re important to me personally. The medication that I’m on right now of course went through a clinical trial process, and it wasn’t even around before the year 2011. I was Stage 4 when I got discovered, which happens often with non-small cell lung cancer because many folks are asymptomatic. So, for me, what that means is if I didn’t have people going through the clinical trial process ahead of me, I probably wouldn’t be here today. So, on that level, is there anything that you can say why you think that clinical trials are important especially for people on Medicare or Medicaid?
Absolutely. I believe that the clinical trials pave the way. All of the genetic testing that’s done now, all of the testing that’s been done all the way down to a molecular level. So, with these clinical trials and all of the things that have been tested, it’s opened up doors beyond what we ever thought we would have for lung cancer. There are so many opportunities and lines of therapy that you never had before.
And across the board, I think clinical trials and participation in clinical trials, all of the people that have done that, just opened the doors for all of the people in the future. We had a lady who was in her 90s, and she met all of the requirements, participated in a clinical trial. And all the way through, she said, “I want to stay on this. I want to do this. It may not help me, but it will help everybody after me.” And that’s just profound.
Right. And so, Laura, tell the audience who you work with. I know that you specialize in thoracic cancers, and I know that clinical trials don’t always just focus on cancer. They deal with multitudes of diseases and conditions. But can you let us know who you work for because he’s famous in a way, right?
Absolutely. I’m actually the physician nurse for Dr. Raymond Osarogiagbon. He is well known in the field of lung cancer. That’s our specialty. We have a multidisciplinary meeting every week and a conference. He sits on the board for NCCN and multiple, multiple other things as far as paving the way for lung cancer. I’ve been actually privileged to be his nurse since he came in 2005. We’ve built our practice together, and, oh, the changes are just – the changes that I’ve seen in the years that we’ve done this are amazing. And he is brilliant; he is. He’s known all over the world. And our focus is lung cancer.
That’s great. Can you shed some light on the role of the patient navigator or the nurse navigator in what you do on a daily basis with your patients especially around clinical trials and folks who are on those government-supported insurances like me?
Sure. So, we base all of our care – we – or I’m blessed to have a research department and two really dedicated research coordinators that I work with very closely. They’re not nurses like myself, but they do all of the coordinating for the care on the studies and all of the above from patients that are uninsured that are on Medicaid, Medicare, even private insurance. And what we do, we see primarily all of our new patients insurance allowing through our thoracic program.
So, I actually have a coordinator with me when I’m in clinic. And so, if we even think a patient is potentially eligible – not even just for a drug study. There are smoking cessation studies that we have, different protocols for that. So, it really starts at the beginning. There’s the surgical studies, different things like that. And every Wednesday is that clinic. And even during the week, if there’s anything going on, they come to our regular clinics as well and do follow up with the patient.
So, I hear chatters here and there – when I bring up the subject of clinical trials, I hear things like, “Oh, trials are only for young people,” or, “Trials are only for old people,” or, “Trials are only for this type of person.” Can you speak to that a little bit?
Wow. Yeah. Well, part of it is if you look at where we sit, there’s always – until now, in recent years, you heard about research but you didn’t really hear about research. So your older population, they were skeptical. It’s a different generation of, “Are you experimenting on me?” And part of your underserved communities, a lot of people didn’t know anything about it. They’re limited on getting to a physician in general much less being able to participate or being in a center that even focuses on clinical trials.
So, I think all of that in the past was very, very real. I believe now people are coming around and seeing, “Wow, anybody can do this.” I think people are still limited. Some people don’t have computer access. It’s hard in a day of electronics, we sit down and we can pull up all of this information, but not everyone can do that.
Right. We do make a lot of assumptions when it comes to those type of factors. So, being that you’re a nurse navigator, I imagine that when you’re seeing a patient, you’re thinking, “Okay, is there a trial that this person might be good for?” I don’t want to say convince, but how do you help people learn about clinical trials and the importance of it because when you and I spoke yesterday, you said you want to make it clear to patients it’s always voluntary, “We’re not dragging anybody into a study. We want to make sure that you want to be there”?
Absolutely. So, again, all of our patients are approved during a thoracic conference, and then all of the ones that we can bring to our clinic within our healthcare system we bring through that clinic, and if not, we bring them to our general oncology clinic. The physician will sit down with the patient. Of course, we’ve met with the coordinators, they’ve looked at everything. And they’ll come to us and say, yeah, they like this or this. The physician sits down and talks with them, and then I go in the room and talk with them as well. We tell them, “This is totally voluntary, something that’s open to you if you’re interested,” talk about it.
The coordinators go in and speak with them as well. We tell them to go home, “If you have any questions or concerns, call back.” And a lot of times they will. You have to be able to digest something. It’s a very overwhelming visit to walk in an oncologist office and be told all of this information and try to sort it all out on the spot. So, a lot of times they’ll go home, they’ll think about it, they’ll call back. Basically, communication, I just feel that’s the most important – it’s communication.
Absolutely. So, to circle back a little bit, do you feel like it’s realistic for patients that are on Medicare and Medicaid to be in a clinical trial?
Absolutely. I think it’s clinically appropriate for anyone that fits. If everything lines up the way it should and they’re able to participate, I think it would be wonderful if everyone would.
This may seem like a silly question, but do folks on those programs get the same care as somebody that has a private insurance?
Absolutely, absolutely from our standpoint. Of course, I’m answering from my institution and what I know that we do. And they do, absolutely. And sometimes there are challenges. I mean, we’ve had patients that were uninsured, underinsured. Again, Medicaid, you have to make sure – Medicare’s a little bit different again because all of the guidelines were set state to state. Medicaid’s different because each state has its own – and if you see someone in Mississippi, sometimes they can’t come across to Tennessee to go to the hospital or to do this. So, it’s a patient-by-patient basis, but overall, I think our patients are being treated, being offered clinical trials, and should participate if at all possible.
Wonderful. And again, just to underline that clear and open communication is important.
I think communication is No. 1 for everything. People are scared. They have questions. They don’t even know what to ask immediately. So, I think all of the support you can give – everybody has a knowledge base and everybody is empowered with that knowledge. Sometimes it’s all about just listening, communicating, and then answering any question they have no matter how simple it may be to us. To a patient, it’s a very profound thing. And it could be as simple as, “How am I going to get back and forth? Do you have a way to help me?”
Thank you, Laura.
Okay. I’m gonna circle back to the group and just ask some questions. I wanted to rewind with Mark and talk about Medicare Advantage. I am on Medicaid for about another year and I’m going to be rolled into Medicare, which under typical – I mean, I’m 44 years old, and so Medicare is typically for people that are 65 and older. And so, for me, it feels a little bit strange, and I’m like, “I just want to know how are they different.” And so, I have called my local CMS office, my local Social Security disability office. And I feel like I get different information. So, it’s sifting through everything. I just wanted to call out Medicare Advantage because you mentioned that. Can you expand on that and how it ties in with clinical trials?
Sure, sure, happy to. So, traditional Medicare has multiple parts. You have Medicare Part A, which is the hospitalization, and Medicare Part B, which is the physician portion, and then a Medicare Part D, which is the drug portion. A few years back (understand the complexities of all the pieces and parts of Medicare) there was a decision to allow private insurance companies to administer all the programs together on an optional basis.
So, if you qualify for Medicare, you can use the traditional what’s called fee-for-service Medicare or you can go through a private insurance company. So, this might be an Anthem or a Blue Cross or another private insurance company like that who has been authorized to bundle all of your Medicare benefits together under one program. Now because it is a privately run version of Medicare, they’re required to offer the minimum benefits, but they do have some flexibilities in how they administer that.
So, a traditional fee-for-service Medicare, as long as a physician advertises that they accept Medicare patients, you can go anywhere you want to. If you live in Florida and you go on vacation into Los Angeles and become ill and you want to go visit a physician there, as long as they accept Medicare patients, that’s fine. Medicare Advantage on the other hand looks a lot more like private insurance in that they sometimes build closed networks, so, you can only go to certain systems or only go to certain doctors. So, that’s an important difference between the two.
And in terms of with clinical trials, how that’s affected, if you want to enroll in a clinical trial and you’re Medicaid Advantage, right now the current policy is for the portion of your care that is related to the clinical trial, you would revert back Medicare fee-for-service, traditional Medicare. That doesn’t mean that you are kicked off of Medicare Advantage, but anything related to that clinical trial would be handled from a payment and a billing standpoint through traditional Medicare.
So, if you’re on a cancer clinical trial, all those cancer clinical trial bills would go through traditional Medicare. But say, for example, you needed to get your flu shot or had a cold or something like that, that would still be handled under your traditional – or under your Medicare Advantage. You wouldn’t be kicked off of it. It’s just the treatment part of your clinical trial would go through traditional Medicare. So, a little confusing, but that’s where we are from a policy standpoint today.
Okay. Jeanne, I wanted to ask you – and again, if you want to defer this to one of our other panelists, that’s A-okay. I’m thinking of folks who have some barriers around those additional costs in a clinical trial. Is it typical or acceptable for the, for example, pharmaceutical company or the sponsor of the clinical trial to pick up some of the costs that may not be covered under Medicare or Medicaid?
The answer to that question is yes, it is appropriate for them to do so. And actually, there is an FDA guidance document (it’s Guidance for Industry) that actually reinforces their ability to do so because there has been some concern that covering costs like logistical costs or hotels or travel or giving people a gas card would create undue influence. So, I think the FDA put out a draft guidance that’s clearing that up and basically reinforcing the fact that pharmaceutical companies are able to do that.
I can tell you from our working group, we have 10 active major pharmaceutical company members in the Diverse Cancer Communities Working Group. And I asked them what they usually do in this space, and during the planning phase of the clinical trial, they go out to their sites to ask for a budget and ask them what they need in terms of routine care costs and also logistical costs. And the site sends that information in. And generally, the pharmaceutical companies cover those costs.
What I’ve found to be the case, which is interesting to me, is that the clinical trial operations team in the sites have a lot to do, they have a lot of work to do. And this was brought up to me by a couple of leaders in pharmaceutical companies, that what they’ve learned is that they also need to ask what capabilities do you need, do you need people support or FTE support to be able to adjudicate and track those costs at a site level and validate them and close them out and pay them. And a lot of times, the answer is yes and pharmaceutical companies are paying for those FTEs at the site. So, those costs are being covered when the site asks for support.
Got it. So, since we’re talking about this topic anyway, that draft to FDA guidance publication, I’m gonna say it. It’s a really long title. It’s a mouthful. But I’m hoping you can break down a little bit of that. So, it’s called Enhancing the Diversity of Clinical Trial Populations, Eligibility Criteria, Enrollment Practices, and Trial Designs Guidance for Industry. What is the meaning –?
So, I do want to paraphrase what the FDA says, but I’m gonna read the portions that I think are appropriate for this discussion. So, there’s a section in this guidance that was put out in June, and it’s a draft guidance, so, it’s open for public comment. And it focuses on study design and conduct considerations for improving enrollment in the industry. There’s a big section. I really would urge everybody on the call to read this section because I think it’s really great and progressive and quite empathetic of a major governmental agency to put out this guidance to industry.
It gives examples. It notes the burden for trial participants in remote and rural locations, for example, and also acknowledges the trial burden on the elderly, children, disabled, and cognitively impaired individuals who require caregiver assistance. So, what the FDA does in this guidance is they encourage industry to reduce No. 1 the number of study visits where possible and use electronic communications or mobile technology to monitor the patient for safety and efficacy because of the challenges of a number of folks in this patient population.
They also encourage industry to make sure that patients are aware of financial reimbursements, and that’s what Laura does. She manages their expectations in the recruiting stage and reinforces the fact – and the guidance also reinforces the fact that the FDA does not consider reimbursement of travel, lodging, parking, time, and other considerations to raise issues concerning undue influence. And they also reinforce that the amount of dollars that might be reimbursed should always be addressed with the local IRB. So, I think this is a very progressive guidance to give the industry so there are no questions on what they can and cannot do.
Okay. Thank you very much. Laura McHugh, quick question, and Mark touched on this earlier in the program, what if something goes wrong in a clinical trial and a patient has to be hospitalized or treated for an unexpected reason? That’s covered, right?
It has been for our patients. If it’s Medicare, what you always look at is standard of care. And the Medicaid patients that we’ve had, when they’ve been hospitalized, to my recollection, we’ve not had anyone that we’ve had difficulty substantiating why it should be covered. I mean, sometimes you have to go the extra step and go back and forth with the insurance companies or Medicaid. But we so far have been able to get it covered.
I have a couple of questions that have come in from the audience, and feel free, Mark, Jeanne, or Laura. I’m assuming that a nurse navigator or a doctor is going to have the best information on where to find out about a clinical trial. But where are the best resources for someone to go? And again, I’m cancer focused because I have lung cancer and I work for Patient Power. And we support all types of folks with cancer. But there are folks that are in clinical trials that are not cancer related. Mark, what would be a source where somebody can find a clinical trial?
Sure. So, in looking at the current cancer clinical trial landscape, we know that the overwhelming majority, probably 75 to 80 percent of patients, who end up on a clinical trial found that clinical trial because someone on their care team recommended it or someone from the clinical trial team approached them. So, it’s most common that someone from the medical system invites that patient. But we also know that a lot of patients get their cancer care at very small practices (they might be single-doc practices or things like that) where clinical research is not a normal part of what they do. And in that case, you would not necessarily hear about clinical trials from your nurse or from your physician.
In those cases, it’s up to an empowered patient to find the clinical trial on their own. And that’s obviously a little bit harder but certainly not impossible. And there are public-facing websites. Some of them are sponsored by the government, things like ClinicalTrials.gov where all clinical trials whether cancer or not are listed in the United States. And NCI has one, trials.cancer.gov, which is just NCI sponsored, which is the National Cancer Institute. So, it’s federally funded clinical trials.
But additionally, many patient organizations both have general educational materials about clinical trials – so, for example, the American Cancer Society at the website cancer.org has information about clinical trials. At the moment, we don’t have a matching window, if you will, but many patient-advocacy organizations also actively help patients one on one with matching. So, many of these are disease specific. So, there are lung cancer groups who you can call at the hotline, colorectal cancer, etc. Many patient-advocacy organizations will do the direct handholding and navigation if your own provider does not do that for you.
I just want to add to that great list that Mark gave in terms of finding clinical trial sites. So, just a shout out to Stand Up To Cancer, they have a clinical trial matching site for any type of cancer. You can contact them, and they will actually match you to a clinical trial site in your area so you can give that information to your provider so they can call them to see if you qualify. Sometimes it’s difficult for anybody, myself included, to understand what clinical trial I might be eligible for just by looking at a site. So, it’s nice to have somebody do that for you.
Also, all the major pharmaceutical companies have if you happen to know about a given therapy or that you might be looking to be on because you heard about it it’s good to ask for help from somebody to find out what company makes it go to their website. And they all have clinical trial information on their sites as well.
Thank you. And I’d like to share a little bit about my personal experience. When I was diagnosed, I was told about a Facebook group for my specific type of lung cancer mutation. And I learned about clinical trials from that group. And if I had never, like you said, Mark, been an empowered patient and been very curious in wanting the best care for myself, I probably would not have found out about those trials because some of them are just fly under the radar; they’re doing their work.
I think these are some great resources, and thank you for sharing those. One more question that I would like to ask the group before we – we have a couple of questions that came in from the audience, which is awesome. What is one solution (Mark, we’ll start with you) that you would like to put forth to address the issue of better clinical trial participation for Medicare and Medicaid patients which really, I mean, goes out to the larger group, I mean, really for anyone?
Yeah. Well, I think specifically within the population of Medicare and Medicaid, as I mentioned at the outset, Medicare has a uniform national policy. So, someone like Laura, if she became a clinical trial professional in a different state, the Medicare policy would be the same it doesn’t matter what state you’re in. Whereas Medicaid, it varies so much, and that can be quite a bit of hurdle.
As I mentioned, I work in the policy and advocacy portion of ACS, and so, we focus on legislation. And so, one of the public policies that we have been advocating for (and there’s actually a piece of legislation before Congress right now), it would harmonize all 50 states plus DC Medicaid policies such that standard of routine care costs in cancer clinical trials would be covered in all 50 states in the same way and there wouldn’t be this ambiguity or uncertainty from state to state in terms of how it’s covered. So, that would be my one wish within this question if I could wave my magic wand.
Yeah. That would very much clarify everything. Ms. McHugh, do you have a solution? What would you like to see happen to get more folks participating in clinical trials specifically those on the Medicares and Medicaids?
Again, from my nursing background, a lot of it’s communication. And I think it’s sitting down with patients and explaining what some of the benefits are, what the risks are but what the benefits are because truly the benefits outweigh the risks. People worry about money and they worry about all of these things. Well, if it’s Medicare, it’s standard of care. Anything above and beyond, if there’s a problem, then you appeal back to the drug company, the provider.
Opening doors, communicating with patients, telling them, “You have a more active role in your own healthcare when you’re on a clinical trial. You’re empowered. You’re educated. You’re the first to benefit from this drug. You have your health professionals close. You’ve got a research coordinator, your nurse, your doctor, access to new drugs that may not be available.” I just feel like communication and – we’re totally sitting down with someone and explaining and taking some of the fear away from what people think about being on a clinical trial.
I have a friend in the lung cancer community that was in a clinical trial. I don’t remember the specific drug, but she is still on it after it came out of trials. And she’s been on it for years, which is amazingly successful. And if not for that trial, she wouldn’t be where she is. And so, that’s just amazing. Okay. And then, Jeanne?
You know what, first of all, I agree with what Mark said and what Laura said. First of all, it needs to be legislated. And No. 2, there needs to be better communication amongst trusted providers, trusted community leaders, primary care physicians to talk to patients to have them understand that a lot of these trials now include placebo versus standard of care and also help them to manage their expectations in terms of what will be covered in terms of their cost. And the folks that need to do that are the closest to the healthcare systems and patient navigators and care coordinators who can talk to an individual specific situation.
I think in addition to all those things, I think that generally industry needs to do a better job of placing trials where the patients are. Although that seems quite trite, patients that are in underserved communities or in rural communities, they don’t often have access to these cancer centers which are big academic centers that do a lot of these trials with big innovations.
And I think that we need to get much more creative to make sure that either the reach out from those academic centers go out to community centers or we do a better job placing clinical trials in community research centers to ensure better accessibility because really, logistical support, even if you cover it, even if the industry covers it or cancer care covers it or the American Cancer Society cover it or a laser X organization covers it, it’s still a challenge and a barrier.
So, I think we need to do a better job overall. The infrastructure needs to place trails where the patients are because cancers are not homogeneous across the United States. They appear in different places with higher risk and higher prevalence. And we need to use that data to place trials where the patients are.
I agree. I’m actually located in Denver, Colorado, and I was doing some research for a blog post recently. And I went to American Cancer Society, Mark, just to look for what are the most recent statistics by state in terms of cancer. And obviously, it’s not lung cancer specific. But I was shocked to find out that Colorado has one of the highest percentages in the country of cancer occurrence. And I was surprised. So, Laura, would it be appropriate – this article that you sent me this morning from ASCO, would this be appropriate to include in our downloadable guide for our guests after the program? This was about the Affordable Care Act because we were talking about how people can get involved if they’re interested. What do you think, should we include this, Jeanne?
Oh, I heard you say Laura.
I think it’s a really well thought out piece to help folks understand how they can get involved with their legislators and understand that this act and this piece of legislation to advocate [inaudible] [00:50:28] specifically for patients that are on Medicaid in the United States so they can get the same benefit of routine care that Medicare patients get.
I do have a question from Steve, one of our audience members, and he says, “Can Medigap Plan F help with paying for clinical trials? If the clinical trial accepts Medicare, would my out-of-pocket expenses be covered? I’m worried that any extra testing would be my responsibility.”
Yeah. I’m happy to jump in with a quick answer on that.
Okay. Thanks Mark.
So, I mentioned a little bit before about what’s required to be covered. When you think about costs involved in a clinical trial, I’ll put them in three buckets. There is the normal routine medical care that you would get. So, for example, if you would normally get surgery and then followed up by some sort of chemotherapy, everybody’s gonna get the surgery regardless. And then say, for example, ordinarily routine care would be you would get a scan every six months after surgery, but the clinical trial because they want to collect more data wants to have a scan every three months instead of every six months. And the clinical trial is testing a new drug after surgery.
So, Medicare would pay for the routine costs, which would be the surgery and then a scan every six months. The clinical trial sponsor would pay for the drug, which is what you’re testing in the clinical trial. So, the patient doesn’t have any responsibilities for that. And since there’s basically twice the frequency of scans, the sponsor would pay for every other scan.
Now what’s important is that while Medicare covers the routine care costs, it covers them the same way it would cover any other cost. So, if you have a co-pay for a doctor’s visit that is routine, just because you’re on a clinical trial, that co-pay doesn’t disappear. So, if you have a Medigap plan that covers those co-pays, it should cover them the same way as if you were not on a clinical trial because the only responsibility for the patient is the co-pays of the routine care costs, and Medicare will pick those up.
So, anything that’s not normal from a medical standpoint will be paid for by the sponsor. Now as Jeanne aptly pointed out, if you’re coming in twice as often for tests, even if the test itself is paid for, you might be paying for the parking garage twice as often or gas to travel twice as often. And those are nonmedical costs that can add up, but they’re not really involved with insurance, but you can sometimes get money from the sponsor or other third-party support organizations like ACS.
We have one more. Annie B, “I’m on Medicare. Where do I find a clinical trial in my town?”
Typically, most of the ways that you find clinical trials, again, you can work directly with where you’re seeking care. So, if you have an oncologist, you can ask them about clinical trials. And if they conduct them, they will screen you for the trials that they have open at their site. If they don’t conduct clinical research, then you would either go to one of these public websites like a ClinicalTrials.gov, you could call an advocacy organization. There are several in the lung cancer space, and we can provide a number of different links to different matching engines or third-party organizations that could help match you. But clinical trials typically are not restricted based on insurance types. So, you would use the same search engines as anyone else would.
Okay. All right. Well, I want to say thank you so much to our esteemed guests for joining us today. We learned so much today about clinical trials, Medicare and Medicaid, the different options. So many takeaways here. We will have a downloadable guide available as well as a replay of the program in case you’d like to dig in a little bit deeper.
Really, I think my takeaway from the whole program is that there are options out there. Clinical trials can be a great solution for your medical care of your disease. I personally am all for it. I know it’s a very personal decision, whether you want to participate or not. But I decided early on that I would definitely enter a clinical trial because I’m willing to sacrifice myself for future generations because there are people that came before me that did the same and I would not be here today if not for that. So, thank you again for joining us Mark, Jeanne, Laura. We very much appreciate you.
We thank AbbVie, Celgene Corporation, Daiichi Sankyo, and Novartis for their support.
https://powerfulpatients.org/pen/wp-content/uploads/CT-Mythbusters.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2019-07-30 16:51:452020-03-13 08:13:44Clinical Trial Mythbusters: How Does Medicare or Medicaid Impact My Ability to Participate in a Clinical Trial?
Cancer is costly. Each year, it costs $180 billion in health care expenses and loss of productivity, says the American Cancer Society. For individuals, it is the life-saving medications they need that can cost the most. According to cancer.gov, 90 percent of Americans say that cancer drugs are too expensive, and the prices have been steadily increasing for the last twenty years. Some cancer drugs debut on the market at a cost of more than $100,000 per patient per year, some for as much as $400,000. With this type of pricing, even insured patients can be facing out-of-pocket expenses in the tens of thousands.
When patients can’t afford their medications, it can lead to people taking them in lower doses or skipping them altogether, and that can lead to serious consequences, such as shortened survival times. High-cost medications can also lead to financial ruin for some patients. Chronic lymphocytic leukemia (CLL) patient James Miller, whose copay for his experimental and life-saving medication is “outrageously expensive” at $790 a month, says that, medications could eventually bankrupt people, especially if the medications are a patient’s only option for survival.
It’s literally a matter of life or death for patients like Miller to find funding solutions for their cancer drugs. Luckily for him, his medication is covered through the manufacturer’s Patient Assistance Program. Drug manufactures created Patient Assistant Programs, commonly known as PAPs, to provide qualifying patients with free or discounted medications.
While just about every manufacturer has an assistance program, one of the first manufacturers to offer a PAP was AstraZeneca. Company representative Colleen Kempf says, AstraZeneca began offering patient assistance over 40 years ago. The program now covers the company’s marketed medicines, and Kempf says, in the past ten years, the company has helped over 4 million patients with access to medications. “Our programs are driven by our corporate value in putting patients first. We believe that we have a role to play to support patients, and since 2005 have expressed this commitment in a very public way through our advertising.” Their PAP slogan, “If you can’t afford your medications, AstraZeneca may be able to help,” might be familiar to many as it is frequently heard at the end of its television adds and leads patients to its website which is where most PAP information can be found.
Find a PAP
The most important thing to know about PAPs is that they are available. They all vary a bit and have different names, but chances are, your drug’s manufacturer has one. AstraZeneca’s is called AZ&ME. Genentech, the manufacturer of the medication Miller takes for his CLL, calls its program Genentech Access. Celgene refers to its as Patient Support, and Takeda refers to its as Help at Hand.
Once you know assistance is available, it’s fairly easy to find it. All it takes is an online search of the name of the drug, coupled with the words “patient assistance program”, and you should be well on your way to the application process.
John Rosenguard, a multiple myeloma patient, learned about PAPs while doing research about insurance carriers. In addition, Celgene, the manufacturer of his medication, led him to its assistance program through an online risk management survey he was required to take when he was prescribed the medication.
There are also websites specifically designed to help patients find assistance. Non-profit website needymeds.org was formed in 1997 with the intent of helping patients navigate PAPs. Now a partner with Patient Empowerment Network (PEN), the vast NeedyMeds database of PAPs can be searched using the link below. All you have to do is enter the drug name to discover whether or not a PAP is available.
While it may seem like the best place to learn about PAPs is the internet, patients and drug companies both recommend you include talking to your healthcare provider about options. Miller learned about the Genentech PAP he uses through his doctor who put him in touch with a specialty pharmacy who provided him with a PAP application. Miller says he would not have known about the PAP on his own, but that without it he would “go broke”. He advises other patients to ask their treating physicians about options. “Any doctor prescribing an experimental drug like that will have a relationship with a specialty pharmacy,” he says.
Miller’s advice is good, but most people don’t seem to be following it, according to cancer.gov, which reports that only 27 percent of cancer patients, and less than half of oncologists, say that they have had cost-related discussions. But, nearly 66 percent of the patients say they want to talk to their doctors about costs. They should.
AstraZeneca’s Kempf says the company ensures that healthcare providers, patients, and patient groups are made aware of its AZ&ME assistance program. “As with any type of information or program, providers will have different levels of understanding regarding available PAP programs,” says Kempf. “The AZ&ME program works closely with healthcare provider offices on applications at their request and we’ve also seen some offices support their patients by assisting with the enrollment process for their patients.”
Each company has a different process for enrolling in its PAP. Some applications require extensive financial information, while others require basic information; Some require doctors to fill out a portion of the application, while others only need a signed prescription. Miller says for the Genentech enrollment process, he had to provide his financial information and that the application had two or three pages for his doctor to fill out. Rosenguard says the Celgene application process was extremely simple and that it took about two weeks for him to be accepted into the program.
The best way to know what the enrollment process is for the manufacturer of your medication is to go to the company website. The websites are easy and straightforward for patients to navigate. For example, the Celgene Patient Support site has large buttons that say “Enroll now” and “Financial Help”. The words are in big, bold type, and each step is written in clear language. The site also provides a phone number, email, and fax information. There is an option to download the application form if you prefer to print it and fill it out by hand. The steps you will take are listed clearly, and what you need to include with the application is listed clearly. The process was easy and efficient, says Rosenguard.
Most applicants shouldn’t require any assistance beyond what the manufacturers can provide on their websites or by phone, but there are some businesses who will help patients complete the enrollment process for a fee. The prices vary, as does the quality of service.
Not all patients will qualify for assistance. While each program has its own qualifying criteria, and there may be different requirements for different medications produced by the same manufacturer, in general, to qualify for a PAP, a patient must:
Have very limited or no drug coverage from public or private sources
Must demonstrate a financial need based on a set income and assets
Provide proof of US residence or citizenship.
“The AZ&ME program is intended to serve patients most in need and has income eligibility criteria that speak to this design,” says Kempf. “The program primarily serves patients that have no insurance coverage or patients that face affordability challenges with their Medicare cost-sharing requirements.”
In addition, the amount of assistance a patient receives and the length of time each patient can stay on the program varies. AZ&ME patients without insurance are required to reenroll in the program annually, and Medicare patients are required to reenroll at the start of each calendar year.
“It is important for patients to understand the eligibility requirements as well as the documentation requirements that are typically associated with applications,” says Kempf. “Ensuring that the application is filled out, complete, and submitted with the required documents, helps ensure an easy enrollment process.”
Once accepted into the program, both Miller and Rosenguard say that there is not much of a time commitment from them. They both receive their medication through a specialty pharmacy. Miller says his is delivered to his door each month, and Rosenguard says he is able to refill his prescription online, and also has a monthly follow up phone call with the pharmacy. In addition, Rosenguard is required to follow risk management guidelines to participate in the Celgene PAP. Guidelines, as specified by Celgene include, following safe sex practices, not donating blood, and monitoring cuts with blood loss.
AstraZeneca also uses a central pharmacy to dispense its medications to patients, says Kempf. “All medications are dispensed by a pharmacy and are sent directly to the patient’s home unless it is a medication that requires in-office administration by the physician. In office administration products are sent directly to the healthcare practitioner,” she says.
Are PAPs Worth It?
For patients struggling to pay for their medications PAPs may be the only option, and the pharmaceutical companies seem committed to providing the service. Kempf says that at AstraZeneca, they are always evaluating patient feedback to see how they can better serve patients, including streamlining the application process.
Rosenguard recommends the PAP programs. He says, co-pays, like his that were $200 a month per medication, can add up quickly. “The benefits were noticeable and met my needs to control costs over the long term,” says Rosenguard. “Plus, it educated me to help others (employees, support group members, friends) who might need this information in the future.”
https://powerfulpatients.org/pen/wp-content/uploads/Untitled-design-24.png600600PEN Editorial Staffhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngPEN Editorial Staff2019-02-19 20:41:412020-01-29 10:56:23Barby Ingle – What Advice Do You Have For Someone Who Is Newly Diagnosed?
In this MythBusters program, Senior Vice President and Chief Medical Officer (CMO) of ASCO, Dr. Robert Schilsky, and 20+ year CML survivor, Mel Mann along with Cecelia Mann, will unpack some of the issues that have led to the lack of diversity in clinical trials and initiatives in place that are changing all of this.
And greetings from near San Diego, Carlsbad, California. I’m Andrew Schorr from Patient Power. Welcome to this Patient Empowerment Network program, the next in our series Clinical Trial Mythbusters, and this program is so important, discussing what is the value of diversity in clinical trials. And, believe me, you’ll hear it is so critical. We have to do better, and we’re going to discuss that over the next hour.
I want to thank the companies that have provided educational support through grants to the Patient Empowerment Network. They have no editorial control, but their support is welcome. And that is AbbVie Incorporated, Astellas, Celgene and Novartis. All right.
We’ve got a lot to discuss today, and we welcome your questions along the way. I want to first introduce someone who, like me, has greatly benefited from a clinical trial and believes that they are alive today because of their participation. And so joining us from Atlanta is Mel Mann along with his wife and care partner Cecelia Mann. Mel, welcome to the Patient Empowerment Network program.
Thank you very much.
And we’re going to hear more of Mel’s story in just a minute. I want to introduce a very prominent medical expert who joins us. He is the senior vice president and chief medical officer at really the largest cancer organization, the American Society of Clinical Oncology, and that is Dr. Richard Schilsky. Dr. Schilsky, welcome to our program.
Thank you, Andrew. Happy to join you.
Okay. And are you in the Washington, DC, Virginia area?
That’s where our organization is based, in Alexandria, Virginia, yes.
Thank you for being with us. I should mention that Dr. Schilsky has had a lot of experience related to trials. He was the head of hematology/oncology at the University of Chicago, which of course Chicago is a very diverse city, and the University of Chicago does a lot of research. And he also helped run a big research group that doctors from around the world are part of, and he did that for many years. So we’re going hear more from Dr. Schilsky in just a minute. But, first, Mel. So Mel, in the late ’90s you were dying, right, of chronic myeloid leukemia, correct?
Yes, I was.
Losing weight and being told that there wasn’t much to do, right?
Maybe a transplant. But you were in Atlanta and you went from doctor to doctor, but somehow you got to MD Anderson, a major research center in Houston. What did they offer you there?
When I first went out to MD Anderson they said they were going to put me on a clinical trial after clinical trial. And the first thing they did was increase my dose of interferon, and that was the medication, the standard therapy at the time, and then they tried different combinations of drugs, and eventually I started on different clinical trials.
Okay. And, Cecelia, you were there in Atlanta and he was scooting over to Houston, it’s not exactly around the corner. Why were you supportive of that?
I was supportive of that because that was the last chance that he had to a cure and for survival. So from the very beginning, whatever type of treatment he needed when he was flying around, whether he was going looking for bone marrow transplants, doing bone marrow drives, and therefore I was supportive of. We had a five‑year‑old daughter at the time, and so anything that Mel needed I was there to support him.
So, Mel, this is a happy story because here we are in 2018 as we do this program and you are with us when many people with CML at the time were not with us that long. Hopefully, a transplant could be curative, but a lot of people passed away. You were lucky enough to come back as they were rolling through different trials and there was a new one that opened up for a drug called Gleevec, a pill.
Okay. So in the summer of ’98 the Phase 1 Gleevec study opened up, and I went out to MD Anderson, and I was patient number two, and I started taking it at a low dose, and it was effective for me. And eventually they increased the dose and it started changing my leukemia around to eventually I reached what they call a major molecular response. And that was 20 years ago. This summer I went over 20 years.
Wow. Well, Dr. Schilsky, is that an example of a patient getting, if you will, tomorrow’s medicine today, what we hope for?
Absolutely. And, first of all, it’s such a wonderful story, Mel. It’s great to hear you tell it, and it’s exactly why we do research, exactly why we do clinical trials, to try to discover the new therapies that people need that will give them the kind of long‑term survival and quality of life that you’ve been experiencing. It’s just‑‑it’s wonderful.
So, Dr. Schilsky, let’s get into the problem. So, generally, there are many clinical trials that take longer than one would hope to fill, and the FDA and I know scientists have been seeing well, gee, how do we know what we’re testing applies to people maybe with different ethnic, gender backgrounds, a variety of situations, and often we can’t find people who fit those categories to be in. What is that‑‑how poorly have we been doing in the past with diversity in trials, and what does that mean for developing new medicines?
Well, we don’t do well in almost any dimension. We don’t get enough underrepresented minorities in clinical trials. We don’t get enough older people in clinical trials. You have to remember that 60 percent of cancers occur in people 65 years and older, and yet only about 10 percent of people participating in clinical trials are 65 and older. So we are having to treat the majority of older people, and I would say the majority of minority people, with data derived from participants in clinical trials who are not like them.
We need to change that for a whole host of reasons. It’s historically been very challenging, and the problems really sort of boil down into three big areas that I think we can discuss a little bit further.
First is awareness. Many people are not aware that clinical trials are even an option for them. Many people think that a clinical trial is a last resort, and I want to dispel that myth right out of the box. Clinical trials can be a very good option for patients right from the time of their cancer diagnosis even if it’s their very first treatment. So clinical trials may be a last resort, but they don’t have to be, and there are many clinical trials that are appropriate for people right following the initial diagnosis of their cancer.
So there’s the awareness issue, and sometimes, frankly, not even the doctors are aware of what clinical trial options are for their patients. And the one thing we know for sure is that the most influential person as to whether or not a patient goes into a clinical trial is their doctor. If the doctor does not recommend it, if the doctor is not aware of it, it’s not going to happen.
But then you get into the more technical issues. There are things, there are rules for clinical trials because they are research studies. They are experiments. There are very well defined rules, most of which are in place to protect the people who are participating in the study. Some of these rules are called eligibility criteria, and they specify the characteristics of people who can enroll in the study. Well, historically, they tend to be very rigid and very limiting, and you’ll often hear people talk about how the only people who can get into clinical trials are Olympic athletes. That may be the case, but it’s not Olympic athletes that we’re treating in the clinic every day, so we need to make our clinical trials more representative so that they’re more applicable to the typical person that a doctor sees in their office.
And then there are the logistical or operational issues of the clinical trial. The clinical trial can be very burdensome. Mel just described how he had to travel from his home in Atlanta to Houston to participate in a clinical trial. Not everybody can afford to do that. Not everybody can take time away from work, time away from home. And the clinical trial requires not only that you travel sometimes but that you travel on a specific schedule because of the requirements of the trial.
So all of these are issues that are‑‑can limit participation in trials, and many of them are magnified in minority populations or in populations that don’t have the economic resources to be able to meet the requirements of the trial.
Right. Let’s talk about that for a minute. So, Cecelia, you go out in the community and speak to people, and you probably meet some people who maybe are diagnosed with a blood cancer, like you’re active with the Leukemia and Lymphoma Society so you may speak to them, and they say even if you brought up about a trial, they say, hey, Cecelia, I’m working two jobs. Or my husband or spouse is working two jobs, and we’ve got two kids, three kids. How could we ever participate? We just can’t get away or we don’t have the family support or whatever. Those are real issues, right?
True. True, those are real issues, and I try to direct them towards resources that Mel and I found out about along the way. The Leukemia and Lymphoma Society, they have resources to help with travel, and American Cancer Society has resources that help with the hotel and lodging. And there are a number of other different types of funds that can be assessed to make that a possibility.
But you’re right, Andrew. I had one young lady at a health fair and a second cancer had popped up, and she was coming there to get information, and she was saying that they were saying it wasn’t too much they could do, and I brought up MD Anderson. And she immediately said, I can’t go out there, I have to go to work, and she turned around rough. And so when they listen to me then always glad to tell them about it and let them know there is an option and that clinical trials work, and I point to Mel, my husband, as a success story also.
So, Dr. Schilsky, you talked about physician awareness. It’s also about more physicians participating in trials at I think what you call the community level. So, in other words, MD Anderson and where you work, at the University of Chicago, those are big city centers and where they are in Atlanta there’s Emory and some other mainly centers, but what about out in the hinterland, if you will? Can somebody who lives there diagnosed with a cancer, how do they have access to a trial that their doctor knows about and maybe that’s more close to home, if you will?
Right. So of course we know that anybody with cancer prefers to be treated in their community, and most are. So one of the goals is to be sure that oncologists practicing in all sorts of community settings have access to clinical trials. Now, one of the ways that happens is that for more than 50 years now the National Cancer Institute has actually been operating and funding a community‑based clinical trials network. It used to be called the CCOP program. That’s an acronym that we don’t have to go into. They’ve recently changed the name. It’s now called NCORP program, but‑‑that stands for, I think, the National Community Oncology Research Program.
But the point is that the program, which is in most but not all of the states in the United States, funds community oncologists to participate in NCI‑sponsored clinical trials, and there are at least 65 or 70 such clinical facilities around the country right now. So in those medical practices patients can find those clinical trials in their communities without having to travel.
There are also other community‑based networks that are active participants in cancer clinical trials. So I think at the end of the day the critical thing for patients, and this is sometimes easily forgotten because you’re so, you know, your thinking and your time and your emotion are all taken up in dealing with the cancer diagnosis. It’s really important, though, to ask the doctor, do they have access to clinical trials. Do they have a clinical trial that might be appropriate for you? And if not you might want to consider where else you could go, hopefully still relatively nearby to get access to a clinical trial.
Mel, so for you, you went around to some doctors who were not aware of anything new to do for you, right? And that’s still true in so many areas of cancer. Now, what do you say to patients about speaking up because Dr. Schilsky just referred to it, people are terrified. They really just want the doctor to have the answer. What do you tell people so that they maybe advocate for themselves?
Okay. So if you’re looking for a clinical trial and I’m out at, like you say, a health fair, we have a‑‑the Leukemia and Lymphoma Society has something called the clinical trial support center, and they have nurses who work early in the morning till late at night. And you call them up and you tell them about your illness, and they check the availability for what clinical trials are out there, and then they narrow it down to what you actually qualify for, and then they take into consideration your finances and other issues.
And then you’re left with a number of possible clinical trials that you have, and you can take that back to the doctor and you can discuss that with him. So that’s one of the things I talk with them about.
I could mention, now this is really more broadly across cancer, too. So there are breast cancer groups, there are lung cancer groups, and I would just make a pitch to find out, is there a local chapter or national number for you, for the cancer you or a loved one been diagnosed with, and that’s the question.
Say, look, A, I don’t want to feel I’m alone, and, B, how do I get‑‑how do I get connected with what could be lifesaving or life‑extending treatments for me and that I could discuss with my doctor. And understanding‑‑and then, boy, if there are obstacles like financial issues, logistical issues, travel issue, is there support for that.
So let’s go back to the inclusion/exclusion or eligibility issue you spoke about, Dr. Schilsky, because, you know, somebody who has cancer may also have heart problems or diabetes or some other issue. Maybe they previously had another cancer, and so for the companies developing new drugs they may be happy with narrow inclusion criteria because they don’t want to have anything get in the way, some previous thing you’ve had, to affect their ability to have a new drug go on the market.
So what kind of work is going on between government and the drug manufacturers so that the criteria, not so tight, but you can still get legitimate scientific answers?
Right. So, as you alluded to, Andrew, there are good reasons that there are eligibility criteria. One of them is to protect the patients in the study from circumstances that would increase their risk of participating in the study. Another is because the companies or whoever is sponsoring the study wants to be able to isolate the specific effect of what they’re studying without having a lot of confounding factors that could muddy the water and makes it difficult to interpret the results. But that said, the bad thing about all that is that the results of the study might not be applicable to the majority of people who could benefit from the treatment because they weren’t included in the study to begin with.
So one of the things that my organizations has been working on very hard over the last couple of years now, and we’ve been doing this collaboratively with people from the Food and Drug Administration and the National Cancer Institute and a lot of clinical experts and a patient advocacy group, Friends of Cancer Research, is to try to expand or broaden or simplify some of these eligibility criteria that tend to keep people off of trials and in particular tend to keep minority populations off of clinical trials.
So, for example, it’s not unusual for someone who has a new diagnosis of cancer to have previously had some kind of cancer earlier in their lives. So we might see a patient who has lung cancer who 15 years ago had a diagnosis of prostate cancer. Well, for that lung cancer patient to go on a trial that has the typical inclusion and exclusion criteria that doesn’t allow for this previous malignancy, they would be excluded even if they had been cured of that prostate cancer 15 years ago.
We also see, and you mention what we call in the medical profession comorbidity. So if someone’s got cancer and they also have heart disease, they also have diabetes, high blood pressure, anything that affects the functioning of your normal organs, can also exclude people from participating in trials, and there are certain limits that we feel can be expanded and still allow the treatment to be given safely.
So just about a year ago now we came out with a set of recommendations for how eligibility criteria can be modified to make clinical trials more inclusive. And now just recently, I’m really pleased to say, the National Cancer Institute expanded their sort of template protocol document that many investigators follow to incorporate our recommendations, so now their standard protocol includes these broader inclusion and exclusion criteria. And the FDA now is working on what they call guidance documents to advise commercial companies that are running clinical trials to do just the same thing. So we are very optimistic now that we’ve got this ball rolling. We’re going to be removing these obstacles, and we’re going to be able to have more inclusive and diverse population of patients who participate in cancer clinical trials.
Great leadership. I hope it works great, and we’ll be happy to support you. So, Mel and Cecelia, let’s talk about the money part of it a little bit. So you were making trips to Houston, Mel, Cecelia was home with a five‑year‑old, and so admittedly there may be hardships, financial hardships, being away from family if you have to go to a trial somewhere else, checking back. What do you say to people when they say, well, I’m just going to go with the traditional stuff. It’s close to home. In other words, if there can be programs that can help them it still takes courage, if you will. So what would you say to people about investing in their life, if you will?
Well, you know what the standard, what the current treatment is and the outcome of that, so if you want to have a different outcome then you have to try something new which is probably going to be a new drug. So you have to weigh that with the cost and the travel. Some people may not have the support, the caregiver support to go a long distance, so you have to take that into consideration. As far as the Leukemia and Lymphoma Society they do have certain funds where they can help with travel pay, co‑pay, insurance premiums, that could help alleviate some of it. So there’s a lot of stuff that’s involved, and it’s an individual decision.
So we’ll just make a comment, though. So, many people have a church or synagogue, friends, neighbors even if they’re living alone, but yet people are sometimes hesitant to ask for help. And I would say speak up. People do want to help you.
Dr. Schilsky, let’s talk about another reality of trials. There’s a history certainly and some fear still in the black community of whether they were tested on, without their knowledge even, going back years and the general thought, you’ve heard it through your career, I’m sure, people say, well, I don’t want to be a guinea pig for a couple reasons. One is we don’t know if it’s going to work. And second of all if there are different arms of a trial I don’t know if I’m going‑‑I’m going to go to all this trouble and expense, I don’t know if I’m going to get the good stuff. So maybe you could speak about that a little bit. First of all, the fears of being experimented on, and then also about whether you will get what could be a breakthrough.
Yeah. Well, for sure, you know, there is this sort of sordid history of inappropriate experimentation on people, and clinical trials are a form of experimentation. They are a form of research. There’s no doubt about that. But clinical trials these days are highly regulated, overseen by independent groups that include patients and clinical experts that come together in committees called IRBs, Institutional Review Boards, and they evaluate on both the risks and the benefits to patients who participate in clinical trials.
They make sure that the trial has an appropriate consent process associated with it, that it’s explained in plain language to patients, so I think these days a lot of those concerns no longer exist. And I hope that people can get beyond the history that led to some of those concerns. The‑‑sorry, I lost a train of thought on the rest of your question.
The issue about are you going to get the good stuff.
Oh, yes. So a couple points there. One point I want to make clearly is that in most cases cancer clinical trials do not include a placebo or an inactive treatment. That’s not always the case, but it’s true most of the time. So patients are always going to get at least the standard of care treatment, and of course the standard of care is what is at that time known to be the best available treatment.
The whole point of doing the research is to determine if the new thing is better, and of course we always hope it will be. It’s not always better, but sometimes it is, as in Mel’s experience. And I think this has to be clearly laid out to patients. They have to clearly understand why the research is being done. In many trials nowadays even if the patient is assigned to get the standard of care treatment there still may be an option to get the new treatment at a later point. So if the standard of care doesn’t work many times there’s still the opportunity to get the new treatment following the standard of care treatment.
So the trial really boils down to not standard versus new but new versus standard followed by new. So eventually everybody may have a chance to get the new treatment. That’s not always the case, but I think the key‑‑my key take‑home, in a sense, is that we’re doing the research because we think and we hope the new treatment is better, but we have to do the research to prove that. And everybody in a clinical trial I think can be assured that they’re going to get, at the very least, the best available standard treatment.
Mel, when you signed the papers to be in a trial, and you probably shared them with Cecelia, especially back in the late ’90s and I participated in one trial in 2000 and another in 2011, there’s a lot of paperwork, things in bold face written by lawyers. I didn’t always understand it. What propelled you beyond that? Was it just that, oh, my god, if I don’t get something I’m going to die? Or how did you two deal with the paperwork and feel comfortable signing on the dotted line?
Well, I saw a lot of hope in the paperwork. For example, one trial I was on was peginterferon, and I had been taking interferon every day, injecting myself, and I had to keep it refrigerated and when I travelled it made it difficult. So with peg I can take one shot a week, so that would make the cancer journey easier. It may not make me live longer, but it will improve my quality of life, so I saw my quality of life improving with that clinical trial. And I looked at the paperwork, and I went through it, and I felt comfortable with it.
And how about you, Cecelia? I mean, your husband says, well, I’m going to be in a trial and I’ve got to sign all these papers. Did you say at any point, wait a minute, that’s scary?
Well, no, I didn’t. I didn’t because with Mel, he had three years to find a marrow match, and he was at the end of year two and no match in sight. And so when he had the opportunity to go out to MD Anderson and be on a clinical trial or several, I was okay with that. I was okay with that. And I looked at it as actually being a blessing. And it turned out to be, and we’re grateful.
But I would say to anyone else who is contemplating a trial and that person and their caregiver, their spouse, to just educate yourself, and get as much information as you can, ask as many questions as you can, but please don’t just throw it away out of hand. It’s definitely worth considering.
Dr. Schilsky, so we have more than 50 million people with a Hispanic background in the United States, and even if many people are speaking English they may speak Spanish at home. And then when you are diagnosed with a cancer there’s a whole new language of stuff that comes into play that even if you’re fluent in English it may not be either what you easily understand or even aligns‑‑what’s being asked of you aligns with your cultural background. Okay? So how, beyond, let’s say, the African‑American community, when you look at the Hispanic community, how do we encourage participation there and get over some of these cultural or language nuances, if you will?
Yeah. So it’s much the same thing in the sense that the same information has to be conveyed but it may have different meaning and different interpretations in different ethnic and cultural groups. Most clinical trials now will have a consent form that is fully translated into Spanish. But, of course, there are many different languages on the globe. When I was practicing at the University of Chicago for many years on the south side of Chicago, we had Polish‑speaking people, we had Russian‑speaking people, we had people‑‑Chinese‑speaking people.
So the requirements actually are that there must be a consent form, at least some reversion of which is translated into the first language of the patient. So if you’re a native Spanish speaker, a native Chinese speaker, you have to have, be able to see a consent form written in that language, and generally speaking you have to have your native language interpreter present in the room to help you go through the consent form and respond to your questions. And that person has to be someone who is independent from the research team so they can give you the straight answer and not be influenced by any member of the research team. So I think all of that certainly helps.
But, again, what helps a lot more is to have members of the care team who look like the patient. So we have problems with diversity in our profession as well. We have very few African‑American oncologists. We have more Spanish‑speaking oncologists, but again we have few Asian oncologists. So we need to do a better job of improving the diversity of our profession, improving the diversity of the care teams. We need nursing staff and research staff and other people who work with our patients who represent them and gain their trust, who look like them, who talk their language. And I think that will go a long way toward making people feel more comfortable about participating in clinical trials.
I was at a conference last week and I heard some of the patient experience, people from different drug companies talking about how they were trying to simplify their forms because I know in 2000 when I entered a Phase 2 trial there were all kinds of black boxes, you could die, you could this, everything in the kitchen sink was in it. I’m still here, and I think because of the trial, and most of the side effects I didn’t have or they were definitely handled extremely well.
So right now, where are we, Dr. Schilsky, with participation? And why is it important? In other words, in this age of personalized medicine why do we need more black people in certain trials? Like, I know in multiple myeloma, one of the areas we cover, there’s a higher incidence in the black population, right, but yet few black people are in the trials for myeloma drugs. Or maybe there are differences with Asian populations or other populations. So is it that you can’t really get a clear scientific answer on the differences? Is that it?
That’s part of it. First of all, we want anybody who could potentially benefit from being in a trial to be able to be in the trial for their own personal benefit. Secondly, we need to learn about the performance of the drug or the intervention in all the diverse populations in which it might be used. And one of the things we have learned is that not all populations respond the same way. Some treatments are more toxic in certain racial or ethnic groups. Some are more effective in some racial or ethnic groups.
And, you know, since you brought up this whole new world of precision medicine, I’ll give you the example of the lung cancer drugs that are used to treat the specific mutations in a gene called EGFR. So that’s a gene which has mutated in about 15 percent of Caucasian patients with lung cancer, but it’s mutated much more commonly in Asian patients. And in fact one of the clues that there was even a gene mutation that was important in determining whether these drugs worked or not was because it was observed that the drugs worked better in the Asian patients in the clinical trials even before the genetic abnormality had been discovered. And the clue was what’s different about the Asian patients than the other patients in the trial.
So the diversity is critical to our learning and critical to our application of the therapy in all the diverse populations that we serve.
If you’re in our viewing community and you have a question, send your questions into email@example.com, firstname.lastname@example.org. We’ll continue our discussion of course, but we invite you to join in.
So, Mel, when you get to talk to people, what do you say? Somebody is sick, diagnosed with a cancer, what do you say? Dr. Schilsky was talking about not seeing clinical trials as a last resort, and you weren’t seeing it that way (?) Inaudible, but today what would you say to people when you talk to them about it?
Well, I will say explore your possibilities because there are all different opportunities at each phase. You may not go into Phase 1 but you could do a Phase 2, 3, 4‑‑or Phase 3, and you don’t know what’s going to happen in each of those phases. So you just have to see what’s out there. And I’m exhibit A, so they look at me and they say, well, I can work, and then not as suspicious, you know. We have Tuskegee, and that was 1972, and it was that dark period of cancer history so that kind of rolls around in their mind, but you can’t let that jeopardize the opportunity such as Gleevec that I took advantage of. So we know that Gleevec worked, and there are other drugs that have improved the quality of life and the lifespan of cancer patients. So definitely research those drugs.
Did you lose heart when you were first in one trial and the medicine wasn’t working for very long? Some would say, well, all right, I tried a trial, forget about it, you know. But you then pursued other trials. What propelled you to do that?
Well, I was still in the game, so I saw that these trials took‑‑well, first of all, I could not find a bone marrow donor, but a bone marrow transplant was pretty drastic in itself so I was looking at these other opportunities as maybe not even having to take part in‑‑have a bone marrow transplant. So that was another incentive. So‑‑and I knew that if I didn’t find one‑‑there was a very small chance, there was only about 5 percent of Americas who are on the marrow registry, so basically I was helping to build a list, maybe not for myself but for people in the future who needed a transplant.
Dr. Schilsky, let’s talk about the pace of research. So, first of all, if we don’t get enough participation in trials how does that slow drug development?
Well, it slows it down enormously because we have to have a certain number of people in each trial to be able to get a reliable answer. And these days it’s becoming even more challenging because as we’re developing drugs that only target a specific genetic abnormality in the tumor which sometimes is very rare so we may be looking for a genetic abnormality that only occurs in 2 or 3 percent of all people with a certain kind of cancer. First you have to find the people who have that genetic abnormalities, then you have to be able to enroll them in a clinical trial. They have to be willing. They have to meet the enrollment criteria. So it can take a long time, and even a global effort to find enough people to fill out a clinical trial.
And most clinical trials in order to produce a reliable result are going to require a minimum of 50 to 100 patients. Some require many hundreds of patients or even many thousands of patients depending on the question being asked. So you can see if people are not participating it’s going to take long time to get those answers.
Now, Mel, you got Gleevec in a trial at least three years before it was approved, and it was approved fast because it was such a breakthrough, right? So you literally got tomorrow’s medicine today, and it saved your life in the process, right?
Yes, because I was past the three years. I was about three years and eight months in my diagnosis, so you add another three years onto that and I would not have been here.
Right. In my case I was in a trial related to chronic lymphocytic leukemia, three‑drug combination, and I received that in a Phase 2 trial 10 years before that was approved. So it was a long time.
So I have a question for you about personalized medicine, Dr. Schilsky. So for instance in chronic lymphocytic leukemia I’m not‑‑I’m Caucasian but I’m Ashkenazic Jewish, okay? So where we’re going with personalized medicine, are we beginning to find subsets among Caucasians, among African‑Americans, among Asians, where there are even more narrow slices to help us understand targeting of medicines and what’s effective for whom? Is that where we’re headed?
Absolutely. And, as I said earlier, we’re seeing that all the time. So almost every common cancer now is being broken down into a whole basketful of rare cancers under the broad umbrella of whatever the cancer type. So lung cancer, there’s probably six or eight different kinds of lung cancer now that each have a specific genetic abnormality, that each requires a specific treatment. And many of those treatments now are FDA approved, but the first thing you have to know is does the cancer have the genetic abnormality and then what is the appropriate treatment to use. We’re seeing that in breast cancer, in melanoma, in many other kinds of cancer types.
There’s another‑‑there’s a related issue here, though, of course, which is that not everybody metabolizes drugs the same way, and so another reason to have diverse populations in a clinical trial is to learn about side effect profile of the drug, learn about the right dose of the drug to use. And we know full well, for example, that African‑Americans metabolize some drugs differently from white people, and so, depending upon how the drug is working in the body, an African‑American person might require a higher dose or a lower dose of the same drug that a white person would require in order to get the same therapeutic effect.
So it just speaks to the point again where if you don’t have diverse populations in the trials you can’t learn this stuff so that doctors then have the information they need to be able to prescribe the drug in the best way for their particular patient.
Okay. Here’s some questions we’ve been getting in. Kaitlin wrote in, Mel, she wants to know, do you still participate in follow‑up activities related to the trial you were in? So tell us about your participation and sort of follow‑up.
My follow‑up is I go out to MD Anderson twice a year, and it’s just a one‑day, one‑hour doctor visit where they take the blood work and they check and see if everything’s stable. And then when I’m back home, twice a year I have my blood work checked back at home, and that’s the extent of the follow‑up. I still have to take medication, one pill a day.
Right. And is that still covered by the trial?
Well, it’s covered by the trial, but my insurance also covers it. I did Gleevec for life because of the trial.
Okay. Dr. Schilsky, let me just ask you, is that a benefit typically of trials? Like with these oral cancer medicines which you know can be so expensive, if you’re in a trial for one do you get it for life or an extended time or how does that work?
Depends a little bit on the trial and the sponsor for the trial, but the one thing for sure is when you’re on a trial whatever the investigational drug is, whatever is being researched, that’s provided for free. And any testing that would be considered to be for research purposes is provided by free‑‑for free. So that’s a benefit of participating in the trial.
Typically the drugs continue to be provided for free for as long as the patient continues to benefit. Now, sometimes if the drug ultimately gets FDA approved then it may be necessary at some time in the future for a patient to switch over from the research drug to the commercial drug, but of course at that time the drug is FDA approved and if the person has insurance it will generally be covered by their insurance.
Okay. We got a question in though for you, Dr. Schilsky, from Darrell. We were talking about genomic testing to understand what version of a disease we have either because of our ethnic background or some other thing that’s going on with us. As you know, insurance companies for a while have been balking at some of these more sophisticated tests, yet we and our doctor need that for us to get what may be right on target for us. So maybe you could talk about work that ASCO’s doing at all related to that. We want the testing done, but we also want to get it paid for so we can get that right, precise care.
Yeah. It’s a complicated question because the testing is done at different points in the person’s illness. And so typically a test on a tumor specimen that’s necessary to determine a standard of care treatment, and many of these tests are referred to as companion diagnostic tests. Those tests typically are covered by insurance because the treatments themselves are also covered by insurance and the only way to know if you can get the treatment is to have the test done.
Now, where it gets a little bit uncertain is when you get into this sort of large‑scale genomic testing where a patient’s tumor might be tested for many hundreds of genes not really knowing what you’re looking for and not really knowing what you’re going to do when you find it. And that is where you’re beginning to bump up a little bit against, research and that’s where the insurance companies, sometimes some are reluctant to pay for that kind of testing.
Now, at least one of those large genomic profiling tests earlier this year was approved by both the FDA and Medicare and now will be reimbursed. So that’s the good news, and I think that’s the direction that most insurance companies are heading in.
One of the things that my organization is doing to try to understand how best to use these tests and how best to use targeted cancer drugs is we’re doing our own clinical trial that’s available in 20 states around the country, so not the entire country yet, but has already enrolled more than 1200 people on the study over the last two and a half years. And we’re doing this study to understand how this kind of genomic testing is done, what kind of treatment is recommended based on the results of the genomic test and whether or not that treatment actually works.
Cecelia, you mentioned earlier about the lady walked into the health fair and when you started talking to her about trials she said I got to go to work, and she walked out. And Dr. Schilsky was talking about eligibility requirements, but there are other issues where the study may be asking you to come back to some place or have multiple tests with some frequency so it’s not just leaving work one time. It may be leaving work 20 times. Have you had people voice that concern to you, that it’s just‑‑what’s being requested is just too much?
Yes. I think the lack of convenience for people who don’t have the funds or don’t search out the funds would definitely hinder them being on a clinical trial or being open to hear about the clinical trial. So, yeah, convenience and awareness. We try to spread awareness that, yes, after we talk about the disease, the myeloma and the symptoms then we go into the resources. And, you know, I make sure I tell them about calling the information line and talking to the masters level oncologist professional there and finding out about the latest trials, the latest treatments in addition to what they’re doing currently with their doctors or their family members or friend that is diagnosed with one of the blood cancers.
Dr. Schilsky, she’s getting at helping people sort out what trials are available. So medical science is a moving forward, and I’m sure you as an oncologist say, hallelujah, there are more trials than ever, but it’s often not only daunting to understand any one of them but to go through a bunch of them to understand what might be right for you. So how can ASCO help that? Is it just simply educating your doctor, or what can we do for families affected by cancer so they can get at what could be a match for them?
Yeah, it’s a great question. So there are a lot of resources available, as Cecelia has mentioned. We can help patients understand and even begin to sort of, you know, wade through and winnow down the potential clinical trial options for them. One things that we’re working on and very interested in doing is sort of flipping the current paradigm by which trials are done. So right now, typically speaking, the patient has to travel to whatever site has the trial available. If they’re fortunate enough that that’s their own doctor’s office, that’s great, but, as in Mel’s case he had to travel to MD Anderson to get the trial.
The technology these days is at a point where we think we can flip that. Instead of making the patient go to the trial we’re going to work toward making the trial come to the patient. So if your doctor is aware that a trial exists somewhere in the world it should be possible for the doctor to basically just go to a website, find the research study, find the consent form, find the other documents that are necessary and present that to the patient. And if the patient qualifies just sign them up instead of making the patient travel hundreds of miles away to the one place that has the trial available.
Some trials have already gone down this road, and they’ve actually been recruiting very successfully, but it’s still not the usual way in which things are done, and we think we need to try to flip things around a little bit to make it easier for the trial to come to the patients. Let the trial travel. Let’s not make the patient travel.
That sounds great.
Can I add something to that, Andrew?
Okay. As a veteran, I was part of the VA system, and I had to go out to MD Anderson, but this summer they started something called Navigate, the VA had started something called Navigate, which partners with the NCI. And it’s at 12 sites around the country, and it’s to bring the clinical trials to the VA. It’s right there. So if it’s an NCI clinical trial then the veteran can get on that clinical trial. And there’s a lot of African‑American veterans who can take advantage of that.
For sure. That’s terrific. So we’re going in the right direction. One other thing I think that needs to change is we talked about the scientists, whether they’re federal government scientists or drug company scientists, and they want to get answers to a whole bunch of scientific questions. So they may say, as you write the protocol I think it is, well, you have to get so many CT scans and you have to get so many blood tests and stuff like that. And it can become onerous, Dr. Schilsky.
What’s happening in trial design so that, A, we talked about eligibility, you can get into the trial, but the things you’re asking of me may have logistical hurdles as well that you’re kind the lightening up on it to get to the key scientific question without all these other bells and whistles that make it tough on me.
Yes. I like to think of it as the need to know and the nice to know, right? There are certain things you need to know in the trial to be sure that the treatment is working, the patient is safe and not having any severe side effects and things of that sort. A lot of that stuff is the same stuff that doctors order every day on their patients as part of routine clinical care, and so much of what needs to be collected in clinical trials really aligns pretty well with standard of care.
Now, that said, because clinical trials are research and because there’s always new frontiers to explore, sometimes testing in a clinical trial extends beyond what the standard of care is. Sometimes patients are asked to give extra specimens of their blood, of their normal tissues, of their tumor tissues. Extra biopsies might be required, things of that sort. Patients need to understand why they’re being asked to do that, what those specimens are going to be used for, how is it going to advance research.
And, frankly, they’re very important to expanding the scope of the research. So, for example, oftentimes those specimens are used. If the treatment doesn’t work in a patient having those specimens can help the scientists understand why the treatment didn’t work, and that opens up a whole new horizon to explore to potentially make the treatment better in other patients.
Mel, do you recognize that by being in a trial and the work that you and Cecelia have been doing that you’ve probably helped thousands of patients by first being in a trial and then you and Cecelia talking about it?
Yeah. Yeah. I guess that’s kind of hard sometimes. You don’t see yourself in that role, but as I look back on it, yes.
Cecelia, you’ve probably talked to a lot of people. Have you seen a change where‑‑you’ve been doing this for a number of years where earlier on people said no, no, no. Are people more receptive? Do you see a change going on? Let’s say in the African‑American community, do you think people are a little more receptive?
Yes, I think so. I think they are more receptive, and this has a lot to do with education and awareness, and that’s what we are out there doing when we are out there in the community. And the more they hear about it and the more they read about it and the more they can see examples like Melvin, and we know one or two other people that we’ve met that were also on a clinical trial. One is in our church, and he had a type of leukemia, and we didn’t know why he was sick. But he is doing very well.
And so the more we can get those examples out there in the community of successful clinical trial patients, it really helps and goes a long way toward helping people of color relax and come aboard. And I just say, please, do your research, educate yourself and ask questions and please stay open and don’t dismiss clinical trials out of hand.
And, Andrew, if I could just add to that. I just want to make the point that it’s people like Mel who are creating the future. Everything we know about how to treat cancer we learned from the people who participated in the clinical trials. We’ve been doing clinical trials in cancer for at least 70 years, and all of the standard of care treatments that we have today came from the participation of people in clinical trials. And that’s how we make progress. That’s how we’ll continue to make progress.
So it’s the clinical trial participants who, sure, they’re in it for themselves. We understand that. They’re looking for a new treatment, a better outcome, but they are the heroes of oncology because they are paving the way, trying the course and ultimately making a better future for every cancer patient who follows them.
Amen. Let me just recap a couple of things, and correct me if I get anything wrong, either of you. So, first of all, Dr. Schilsky, I know there are more trials now than ever before, and they’re now looking at these rare subtypes as well, and so if we participate we may get the benefit of tomorrow’s benefit today. Cecelia was talking about assistance programs, people to help you sort it out, that you are noticing how there are difference among us about the ways that drugs are effective or not, and that’s so important to learn.
If we partner with you, Dr. Schilsky, and the many thousands of oncologists and researchers that you represent, can we get to the goal line faster? In other words, are you hopeful that if we really consider trials and participate in trials and stay in trials and the different groups that we can get closer to cancer cures?
Absolutely. I mean, we have more and better cancer treatments today than we’ve ever had before. We have all sorts of new and hopeful treatments on the horizon. We have to prove that they are safe and effective treatments to get them out there into routine clinical practice, and that’s where the clinical trials come in. So the more people who participate in trials the more quickly those trials can be completed and give us an answer, then the more quickly those drugs will make their way into standard clinical practice where everybody can benefit from them.
Okay. So a couple of to‑dos for our audience if you’re a patient or a loved one or however you hear this. Ask your doctor about whether trials apply to you or your loved one who is diagnosed with cancer even if it’s on day one. You don’t have to be at death’s door. You’ve been diagnosed or a diagnosis is suspected, what tests can we do, how do we know what we’re dealing with, and when we look at the treatment options is a trial a possibility if that makes sense, right?
Okay. Step two, are there resources to help me overcome any obstacles I may have to participation, first understanding the trial, understanding it in my first language, sorting out is it right for me, getting to where it is and then staying in the trial because many people, unfortunately, don’t stay in the trial and so how do be help the trial get to the goal line?
And then lastly, Dr. Schilsky, it sounds like you’re doing a lot at the community level to have more doctors have an easier time of the bureaucracy that we’ve had with trials before and the understanding of this flood of trials that’s happening, right?
Absolutely. And, you know, to be perfectly honest, the clinical trial community has‑‑we ourselves have created some of the bureaucracy, some of the excess regulation, some of the barriers to participation. It’s up to us to strip those away and solve the problem and make clinical trials more broadly available. We are working very hard now to make that happen.
Okay. So whatever community you’re in. I’m in the Ashkenazic Jewish community. Mel and Cecelia are in the African‑American community. We have people watching who are in the Hispanic community, the Asian community. If you have benefited from a trial, talk it up, right? Cecelia, people should talk it up, correct?
Exactly, yes. Please, talk it up.
Mel, thank you. I wish you really continued good health. How many years has it been since you were diagnosed?
Well, in about two months it will be 24 years.
Twenty‑four years, and for me it’s 22 years. And had there not been trials either that we were in or somebody else was in we wouldn’t be here.
So, thank you. And also, Cecelia, thank you for being a community activist when it comes to trials and being supportive of Mel as he’s been in a trial because some other people would say, you can’t go there, you can’t do this, and you’ve been supportive every step of the way. Thank you for that.
Oh, you’re so welcome. It was a pleasure to do it.
Mel and Cecelia Mann from Atlanta. And Dr. Richard Schilsky, you’ve devoted your life to this, Dr. Schilsky, and I just want to say on behalf of the cancer patient community thank you and thank you for the leadership that ASCO is trying to do, both with changing research requirements, working with government, working with industry, and you thank you personally for your devotion to us. I really appreciate you being with us.
It was my great pleasure. And, again, congratulations to Mel and Cecelia.
Okay. Thank you all. So this is what we do with our Clinical Trials Mythbusters program. Please tell others about it. The replay is available very shortly and all kinds of little highlights that we’ve done today. But what’s important is wherever you are is remember we can’t develop new medicines unless all of us work together to participate to get the scientific answers that apply to us, whatever our unique situation is, and then we can work with government to approve new medicines, get them on the market and so many people can benefit in the US and worldwide.
Thank you so much for being with us on this Patient Empowerment Network program. I’m gratified to be part of it. Thanks too to our financial supporters AbbVie, Astellas, Celgene and Novartis and their dedication to drug development and supporting and sponsoring clinical trials. I’m Andrew Schorr near San Diego. Remember, knowledge can be the best medicine of all.
Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.
https://powerfulpatients.org/pen/wp-content/uploads/What-Is-the-Value-of-Diversity-in-Clinical-Trials_-1.png600600PEN Editorial Staffhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngPEN Editorial Staff2018-12-05 19:48:512020-01-08 12:33:32What Is the Value of Diversity in Clinical Trials?
This is the last installment in our three-part series profiling breast cancer survivors. In Part II, the women gave insight into the importance of their mental health and their own attitude as critical components of care. They also shared some of the ways in which they coped with cancer. Today, the women talk about the possibility of recurrence. So, we pick up with the final stage of Shannon’s preventive measures. Based on her history, she knows her cancer can come back, but she wanted to do everything she could to prevent it.
Shannon’s treatment didn’t stop at reconstruction. She opted to have an oophorectomy, which meant she had her ovaries and fallopian tubes removed. Remember, her moms’s cancer had returned and been terminal, so Shannon wanted to take every preventive measure she could. “My fear and my worry is that hers came back 16 years later and she died at 65. If the same thing happened to me, I would die before I’m 60,” says Shannon. Her breast cancer diagnosis meant she was at higher risk for female cancers and she wanted to do whatever she could to have as much time as she could. “I’m relatively young,” she says. “I wanted to give myself as long as I could.” In order to have the procedure, Shannon had to take medication that would put her into menopause and the side effects that came with menopause affect her quality of life, so she says she goes back and forth on whether or not she would do the oophorectomy, if she had it to do all over again.
Although she did have melanoma a couple of years ago, Tina has been 27 years without recurrence of breast cancer. “I didn’t really feel safe until five years out,” she says, but adds that you never really know if it’s coming back, and that you should always be vigilant about checking for lumps. There is a risk of late recurrence, i.e. breast cancer that comes back more than five years after diagnosis and treatment, and it is more likely if it was later stage when first diagnosed, and if the cancer was HR positive.
Like Tina, Betty also had a second cancer. Her colon cancer was discovered in 2009 and her doctor estimated that it had been growing for ten years, but because of where it was growing in relation to the colon wall, the tumor was able to be cleanly removed and no treatment was required. Because her breast cancer was ductal and not in the tissue, and her doctor was able to get very clean margins, Betty says she doesn’t worry about it returning. “I’m more afraid of the colon cancer returning,” she says.
Diana has been nine years without recurrence, but she says, “My guard is always up.” Maybe it’s because her mother and grandmother both had breast cancer, and, despite being BRCA negative, she believes her cancer is hereditary. Shannon feels the same way and says she believes 100 percent that her cancer is genetic. The genetic testing available is limited compared to the number of genes in the human body so, Shannon says, “There’s a long way to go.” And, while it’s early for Shannon to think about recurrence, she can’t help but consider it. “I don’t want to spend every day thinking about cancer. I don’t want that to be my life,” she says, “but it is in the back of my head.” Not knowing how the cancer might come back makes Shannon especially uneasy because she doesn’t have a plan for it.
When Meredith finished treatment, her doctor said he didn’t expect to see her back for recurrence. The odds were in her favor that she would remain cancer free. Meredith, like Betty, says she got the best cancer to get if you’re going to get cancer, but unlike the other ladies, Meredith was not expecting to get cancer. She didn’t have the same family history. Her only red flag was that she had an aunt that had ovarian cancer and she thought maybe her grandmother had breast cancer when she was 90. Meredith was young, she had three small children, and breast cancer was not on her mind. In fact, she was so sure she didn’t have it, that she took her 18-month-old daughter with her when she got the results from her lumpectomy. But, Meredith, who is also BRCA negative, did have cancer, and while her cancer was ductal, it was bigger than it should have been, and there was also a spot on her other breast that needed to be watched. Wanting to be proactive Meredith opted for a double mastectomy with reconstruction. She also had chemotherapy, because the cancer was found in a lymph node, and she lost all her hair. While she possibly could have gone without radiation, she opted for it. Again, she wanted to be aggressive and as proactive in her treatment as possible. She wanted to make sure her cancer was gone.
About a month ago, Meredith found another lump under her arm. She had a scan that was all clear except for the spot where the lump is located. She and her doctors are hoping it is just scar tissue, but she’ll have a lumpectomy this week and then she’ll wait for the biopsy results, which she is guessing will take several days. “The waiting is the worst,” she says. Liz, as a caregiver, felt the same way about waiting, “The worst part of all of it was waiting for the results.”
Tina, who also had young children at diagnosis, recalls that she just wanted to live long enough to raise her children. She says she found it difficult to accept the idea that she might die before her kids were grown. That thought is clearly on Meredith’s mind as well. “I remember saying, ‘Just give me five more years,’ and now it’s been seven years, and I’m saying, ‘Just give me seven more years,’ but no amount of time is enough,” she says. You can hear in her voice that she’s trying to be brave, and she says, “Hopefully, it will all be fine,” but it’s scary because, even though Meredith got the best cancer you can get if you’re going to get cancer, it is still cancer.
Anxious to hear Meredith’s results? We are, too, and as soon as she gets her results, she’s promised to follow up with us. We’re hoping for good news, and we will let you know as soon as we can.
Jennifer Lessinger is a professional writer and editor who learned the value of patient empowerment during her struggle with a hard-to-diagnose and complex endocrine disorder.
https://powerfulpatients.org/pen/wp-content/uploads/ptprolfileIII.png600600Jennifer Lessingerhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngJennifer Lessinger2018-10-26 15:15:122019-10-09 08:55:35Patient Profiles: Breast Cancer Part III
In the first part of of this three-part series, you were introduced to Betty, her daughter, Liz, Shannon, Tina, and Diana. You learned that women who are diagnosed with breast cancer are not really all that surprised to get a diagnosis. They are often expecting it. Having breasts is a risk factor, and women have breasts. In today’s installment, you’ll meet Meredith. She wasn’t expecting to get breast cancer. But, before we meet her, we’ll pick up where we left off in part one: the matter of hair loss and chemotherapy.
Whether or not a survivor lost her hair during cancer treatment, it’s one of the first things she says about her experience. Shannon even lost her eyelashes and they never came back. She says she knows it’s a little thing, but it still bothers her. Betty, didn’t lose her hair. Like a growing number of women, she did not have to have chemotherapy. She says avoiding chemo made a huge difference in her experience. At the time, Betty was receiving treatment at a research hospital, and there was a test available to her that would determine how beneficial chemotherapy would be in treating her cancer. She was told that if she scored between a one and a 19, she would not need chemotherapy. Betty scored an 18. While she had to pay for the costly test out of pocket and wait for insurance to reimburse her, she says it was worth it. “It was so helpful,” she says. “Because of what chemo does to your body, you don’t want it unless you need it.” Side effects from chemotherapy can include anemia, diarrhea, fatigue, fertility issues, hair changes/loss, memory loss, neuropathy, menopause and menopausal symptoms, mouth and throat sores, nausea, vaginal dryness, vomiting, bone loss/osteoporosis, heart problems, and vision problems. The test Betty had is now being more widely used and, as you may have read on the Patient Empowerment Network blog in the June Notable Newshere, researchers have learned that 70 percent of women don’t need chemotherapy when they have the most common type of early-stage breast cancer, with low and moderate risk of recurrence. The test looks at cells from a tumor biopsy to determine what is known as a patient’s recurrence score. Patients are scored between zero and 100, with zero being the lowest risk of recurrence. Researchers now know that women who score less than 25 do not necessarily need chemotherapy.
Betty also did not need to have a mastectomy. She had a lumpectomy followed by intraoperative radiation therapy, a 30-minute procedure that involves surgically placing a ball of radiation in the spot where the tumor had been. The procedure meant that Betty only had to have a single radiation treatment, and it helps reduce the side effects of radiation. Betty was eligible for that form of radiation in part because of the size of her breasts. She says she told the doctor, “I’ve been lugging around these big things my entire life and they are finally paying off.”
The intraoperative radiation was another benefit of being at a research hospital. At the time of Betty’s treatment, the procedure wasn’t being widely used and wasn’t available through her local doctors. Receiving treatment at a research hospital also made a difference in Diana’s care. She recalls going in to her local doctor’s office for her test results, and no one in the office would make eye contact with her. Then, when the doctor came in to see her, he told her she had breast cancer, that he wanted to treat it right away, that he’d see her next Tuesday, and then he left the room. Diana says she was left there shaking. “He has a heart, but he didn’t show it,” says Diana, who then went to a research hospital for a second opinion. Her new doctor was much better, she says, and adds that the shoulder of his lab coat was always dirty from the smudge of make up left behind after his patients hugged him.
The importance of good doctors that you are comfortable with seemed to be one of the critical components of care to all the women.They all talked about how much they liked and appreciated their doctors. “A really good physician realizes psychological and spiritual care are just as important,” says Tina, who sought the services of a psychologist after her treatment. She was struggling with anxiety and depression and found that the counseling really helped her to work through her emotions about having cancer, which emphasizes another, perhaps the largest, critical component of care during treatment: emotional and mental support and health. Diana says she found support online and emphasized the importance of staying positive through treatment. “Count your positives,” she says. “That is the key.” Along the same line, Betty says, “The number one thing is attitude.” Actually, she and Liz say “attitude” in unison, and Betty adds, “I think attitude is a big piece of it.” Meredith Cronin who was diagnosed at age 37, says “Attitude is everything.” Meredith, who had three children under the age of six when she was diagnosed, says she understands how easily you could get depressed as a cancer patient. “I always say that I felt blessed that I was young and so busy that I didn’t have time to be depressed.”
Shannon, who was accused of being negative because of her detailed planning to get breast cancer before she actually had breast cancer, says she wasn’t negative; she was realistic and it was that take on it and her preparation and planning that helped her maintain emotional balance.“It didn’t affect me emotionally as much as I think it would have,” she says. Shannon coped through research and attention to details, and she describes her experience in the kind of detail that makes you think she’d just had it done yesterday rather than three years ago. Betty used a different method of coping and says she’s been able to let a lot of the experience go. “I don’t dwell in that place,” she says. She and Liz also found a lot of humor in the experience. The clickity-clack of someone’s shoes, Betty’s preoccupation with cutting out recipes from a magazine, or the ridiculousness of what must have been an excruciating procedure, were all fodder for coping. It’s not that Betty doesn’t take cancer seriously, but she was better able to cope with the diagnosis by finding humor in the situation. “We laughed through our tears,” she says. Liz says that early on in the experience, they imagined the worst possible scenario, which made handling what really happened more doable. The ways of coping with cancer are as varied and vast as are the treatment options.
Jennifer Lessinger is a professional writer and editor who learned the value of patient empowerment during her struggle with a hard-to-diagnose and complex endocrine disorder.
https://powerfulpatients.org/pen/wp-content/uploads/ptprolfileII.png600600Jennifer Lessingerhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngJennifer Lessinger2018-10-24 16:27:482019-10-09 08:56:32Patient Profiles: Breast Cancer Part II
It’s October and the pink frenzy is in full force. Breast Cancer Awareness Month (BCAM) is impossible to miss given the pink ribbon avalanche that arrives each Fall. While there is no denying that BCAM has played a significant role over the past two decades in raising public awareness of breast cancer, there is nevertheless growing criticism of its off-balance approach to awareness-raising, with many key messages becoming lost in a sea of “pink-washing.” Interestingly, some studies have even found that pink branding may actually lead the public to take breast cancer less seriously.
“The biggest issue I have with Breast Cancer Awareness month is that it’s not even really awareness,” writes Elizabeth McKenzie, who was diagnosed with breast cancer in 2012. “Awareness is mindfulness of all aspects of breast cancer, which to a certain respect, is different for all of us, based on medical differences in disease processes, treatment access, and personal, social-emotional and cognitive processes.”
Much of the criticism centers on breast cancer campaigns which over-sexualise the disease, equating breasts with womanhood and femininity. Rod Ritchie, who was diagnosed with breast cancer in 2014, points out that “October is a bad time for male breast cancer survivors because the trivialisation and sexualisation of the disease by the pink charities reinforces public awareness that breast cancer is gender specific. Since there’s little attempt to educate men that they need to be aware of symptoms too, we are diagnosed later and have a poorer prognosis. So, how about adding some blue to the pink, encouraging research on us, and screening those with a genetic propensity?”
Over a decade ago on an October morning, I was diagnosed with breast cancer; a double reminder each year of the role this disease has played in my own life. Looking back, I now see that my view of breast cancer was one-dimensional. Standing today on this other side of cancer I see a broader picture, a richer landscape of many shades beyond pink. This post is intended to provide a truer picture of the lives of breast cancer patients in its many varied hues. Wherever you are in your experience, whether you are caring for a loved one, recently diagnosed, finished treatment, or living with a recurrence or metastatic cancer, I hope this post will speak to you.
Lessons We’ve Learned From Each Other
Some of the most valuable lessons I’ve learned about breast cancer have come, not from my doctors, but from fellow patients. My doctors didn’t tell me about the effect of chemotherapy on my future fertility. I didn’t learn that treatment could damage my heart. And I finished treatment with no clue about late treatment side-effects or the risk of a cancer recurrence.
While valuing her oncologist’s expertise, Catherine Foy, who blogs at My Triple Negative Life, acknowledges that “within the online breast cancer community there will be someone awake somewhere in the world that can provide support and advice. For example, based on someone I followed I got my Vitamin D level checked which was very low and I am now on supplements. Other examples include creating awareness of late treatment side effects and reducing the feelings of isolation that some may experience.”
Liz O’Riordan, a breast surgeon diagnosed with recurrent breast cancer, and co-author of The Complete Guide to Breast Cancer, also refers to the feelings of isolation she experienced during her treatment. “I felt incredibly alone during my breast cancer treatment. I didn’t meet another patient at any of my chemo or radiotherapy sessions. All my advice was from amazing people on Twitter,” she says. Liz offers this advice to patients undergoing chemotherapy: “The two best tips I was given to reduce the side effects of chemo were: (1) Drink. Lots. Even when it taste disgusting. Ideally 3 litres a day. Try flavoured water or cordial. Carry a bottle everywhere you go. (2) Exercise. Walk for 30 minutes every day. You’ll hate me for making you. Some days you may have to stop and spit and pant and retch. But do it. You will feel better for it. And they were right. I did feel better.”
Barbara Jacoby of Let Life Happen agrees that we can learn valuable coping lessons from each other. “Whether it is a question of side effects that one is experiencing from a prescribed medication, or questions regarding treatment options or experiences or procedures, if you query a patient support or advocacy group, you are likely to find more answers and information from those who have had actual personal experiences with these issues. There is no doctor or group of medical professionals that has all of the information about real patient experiences and outcomes like any group of actual patients who have dealt with just about anything and everything imaginable on every level,” she says.
Both Catherine and Barbara believe that the information you get online should be shared and discussed with your doctors as the basis of shared decision making (the conversation that happens between a patient and clinician to reach a healthcare choice together). “I value my oncologist’s advice and experience,” says Catherine, “and would usually discuss with him any new developments that I may have encountered through the various platforms on social media.”
Terri Coutee, a two-time breast cancer survivor, and founder of DiepCFoundation, a non-profit organization providing information on options for breast reconstruction after mastectomy, also embraces the concept of shared decision making and wants others to experience it too. “I have had chemotherapy, radiation, two lumpectomies, a double mastectomy and breast reconstruction using my own tissue,” she says. ”Each of these occurrences was fraught with difficult decisions and hours of research to optimize my own health care plan. I left offices of various health care providers with armfuls of brochures and information to sort through and organize.”
Terri encourages patients to download the Breast Advocate App, a new tool to aid the shared decision making process. The app was developed by plastic surgeon Dr Minas Chrysopoulo, whose patient population is primarily those affected by breast cancer or at high risk of developing breast cancer. “Shared decision making is an extremely powerful approach to deciding our treatment plans. Simply put, it empowers us and helps us advocate for ourselves,” explains Terri. “As patients, we owe it to ourselves to embrace everyday conversations with our health care teams,” she says. “The information on the Breast Advocate app is informative, intuitive, and specific to your individual diagnosis or situation. There are treatment options with evidence-based articles to discuss with your healthcare team. It even features a community section. I encourage you to check out the wealth of shared decision-making information and download the Breast Advocate app to your phone.”
Siobhan Freeney, founder of Being Dense, an organization which raises awareness of Breast Density and its associated links to breast cancer and screening, was completely unaware of the issue until she was diagnosed with breast cancer. “40% of women have Dense Breasts,” she explains. “A Mammogram is the only way to determine and measure Breast Density. In Dense Breasts the reliability of screening mammograms can be reduced by as much as 50%.” When you have a Mammogram, the radiologist reading it can tell if you have Dense Breasts. Siobhan recommends you should ask for a copy of your radiology Mammogram report and ask if your breasts are dense. “If you have Dense Breasts you need to know and you should ask your Doctor/Radiologist about more personalised screening such as Breast Ultrasound or MRI,” she advises.
Metastatic Breast Cancer: The Other Side of BCAM
Learning about metastatic breast cancer (MBC; also called stage 4, secondary, or advanced breast cancer) from online blogs and social media networks was revelatory for me. MBC is breast cancer that has spread beyond the breast — to the bones, liver, brain, or another organ. Even if the cancer is found in another organ, it’s still referred to as breast cancer. Like Beth Gainer, who says, she learned “that anyone who’s been diagnosed with breast cancer is at risk,” I too have found, in Beth’s words, “what the metastatic breast cancer community has had to say has been a real eye-opener.”
MBC has been referred to as a story half-told, the other side of BCAM we don’t hear enough about. As Catherine points out, “For me, breast cancer awareness month is for those who are not yet diagnosed or those newly diagnosed. The pink scene looks to future research and provides good information for those starting or in the midst of treatment. There is less focus on those of us living or dying with breast cancer.”
Nancy Stordahl is unequivocal in her criticism of the failure each October to adequately raise awareness of MBC. “Despite all the pink, all the races, all the pink ribbons, most people still know little or nothing about metastatic breast cancer,” she writes. “No wonder so many with metastatic breast cancer feel left out, isolated, alone and yes, even erased.”
Joanne Taylor, a metastatic breast cancer patient and advocate, is pushing for more information and awareness of MBC. She created this infographic to show the red flag symptoms of advanced breast cancer.
While metastatic breast cancer is terminal and cannot be cured, because of improved treatments more women are living longer than ever with it. Even so, many misconceptions and lack of information about this diagnosis persist. “A stranger called Elizabeth Richards contacted me and like many other women she knew very little about MBC, yet the more she found out, the more angry and amazed she became that the illness was side-lined,” says Joanne. “Elizabeth’s view was that as long as metastatic cancer wasn’t mainstream people would not be aware of the limited treatment options available to them. If they knew, they’d demand more.”
One of the biggest misconceptions is that MBC is an instant death sentence. “We thought with BCAM coming up we would do something different to show how people could live well with MBC, so we started the #busylivingwithmets campaign,” explains Joanne. “Elizabeth had the idea, it was positive, it showed what people can do if they have access to drugs and surgery. I was her inspiration! If I can do it, others can as well – if they are given the right options.”
Lessons of Resilience, Connection, and Hope
Cancer can be a lonely and isolating experience, but it doesn’t have to be. “A friend told me to accept whatever help was offered, says breast cancer survivor Connie Rosser Riddle. “It was her way of saying to quit being Superwoman, that it was okay to be in need, vulnerable. It was best to be specific when folks asked, “What can I do for you?” My answer combined what that person was best at and where I needed help, and that made a good fit for both of us.”
Audrey Birt, diagnosed with breast cancer three times, shares lessons of courage, connection and resilience on her blog. “Cancer taught me I’m more resilient than I would have believed, it helped make me braver,” she says. “It also taught me that life cannot be controlled. This made me more able to live in the moment, and for the moment. That’s probably not so good for my bank balance but it’s great for my life balance in a way. It taught me to reengage with writing through my blog and in a funny way it changed my life and connection to others. But it also taught me my fragility and that’s a lesson I’m still learning, one day at a time.”
Ultimately however, the lessons you learn will be unique to you. “There is not a single person, story, book, lecture or talk, which will teach us all we need to know to understand the impact of cancer on our lives. That’s what we have to figure out for ourselves when we go through our own cancer experience,” says therapist, Karin Sieger . “Having been diagnosed twice with breast cancer all I can say is try and stay open minded – to your body, the illness, treatment options. You always have choices. Don’t get stuck in fear and don’t get stuck in complacency either. Live your life to the best of your ability and stay true to who you are – with or without cancer.”
A Stanford Medicine X e-Patient scholar, Marie Ennis O’Connor is an internationally recognized keynote speaker, writer, and consultant on global trends in patient engagement, digital health and participatory medicine. A board member of the Patient Empowerment Foundation, a network of people, foundations, organizations and medical institutions dedicated to empowering patients worldwide, Marie’s work is informed by her passion for embedding the patient voice at the heart of healthcare values. She writes about the experience of transitioning from breast cancer patient to advocate on her award-winning blog Journeying Beyond Breast Cancer.
https://powerfulpatients.org/pen/wp-content/uploads/This-certificate-is-proudly-presented-to.png600600Marie Ennis-O'Connorhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngMarie Ennis-O'Connor2018-10-19 15:48:242019-10-09 09:01:27Beyond Pink: The Other Side of Breast Cancer Awareness and Lessons We’ve Learned From Each Other
Female breast cancer awareness, with it’s pink ribbons, and Save the Ta-tas t-shirts, and fundraising 5Ks, sweeps into October each year with the same prevalence as pumpkins. No other cancer has managed to garner as much support, attention, or money. But, even without the pink campaigns, the prevalence of breast cancer is not a secret. An estimated one in eight women is diagnosed in our country, and there are about 1.38 million new cases worldwide each year. You’d be hard-pressed to find someone who doesn’t know a breast cancer survivor. This month, in a three-part series, Patient Empowerment Network is taking a closer look at five survivor stories and one caregiver. These women represent the more than 3.1 million women in the United States who have a history of breast cancer. In today’s installment, you’ll be introduced to five of the six women, and you’ll learn that getting a breast cancer diagnosis wasn’t really a surprise to any of them.
Breast cancer survivors are interlaced through all of our lives, and there is something very endearing about how openly willing they are to share their stories. They freely talk about their diagnosis and treatment, but more than that, they talk about their darkest moments alone in the hospital, or their need for counseling after treatment. They discuss the lengths they will go to endure invasive treatment that may prolong their lives, and they share their prayers to live long enough to see their children grown. They are so deeply candid that it’s as if they are inviting you to be a guest for the day in their exclusive club.
Only it’s a club you don’t really want to be a part of, says Betty Abbott, who was diagnosed five years ago. She was 72 at the time, and her cancer was ductal and non-invasive. She says it’s the kind you want to get if you’re going to get it. But, is there really a kind of cancer any woman wants to get? While the death rates for breast cancer have been decreasing since 1989 thanks to increased awareness, early detection, and advances in treatment, breast cancer is still the second leading cause of cancer deaths in women in the United States, second only to lung cancer. In 2018, approximately 40,920 women are expected to die from breast cancer.
Cancer is cancer, no matter the stage, the type, or the form. “That’s the thing about breast cancer…it’s still cancer,” says Liz Abbott. She’s Betty’s daughter. The two are very close, and Liz was with Betty every step of the way through diagnosis and treatment. Liz hasn’t had breast cancer…yet, but she fully expects to get it. She knows the statistics. Even though less than 15 percent of people who get diagnosed with breast cancer have a relative diagnosed with it, a woman’s chance of getting breast cancer nearly doubles if a first degree relative (a mother, sister, daughter) has had it. So many women get breast cancer, so many families of women, that for some women it’s no longer if they will get it, It’s when. Since her mom’s diagnosis, “our new realities are very different,” says Liz, who can’t help but worry if breast cancer is her own daughter’s path as well.
It was the path for Shannon Knudsen, who was diagnosed three years ago, when she was 43. Like Liz, Shannon was very close with her mother and walked with her through diagnosis and treatment of breast cancer. Shannon’s grandmother and great grandmother had breast cancer as well. So, for Shannon, breast cancer was never an if. “I never thought I wasn’t going to get it,” she says. “It was always a matter of when.” So Shannon wasn’t surprised by the diagnosis, but she says she was angry. You see, while she was prepared and had a plan, cancer still managed to throw her what she calls “an interesting little twist”.
Since she watched what her mom went through, being diagnosed at 49 with recurrence as leukemia 16 years later that was ultimately terminal, Shannon was diligent about staying on top of cancer research, and as soon as she learned that genetic testing was available, she looked into having it done. She was absolutely positive that her family carried the BRCA gene mutation. The BRCA1 and BRCA2 genes produce proteins that help repair damaged DNA cells. When either of the genes has a mutation and the genes don’t produce the protein or function correctly, DNA cells are more likely to develop changes that can lead to cancer. There are specific mutations of the genes that increase the risk of female breast and ovarian cancers. People who have inherited the mutations, which can come from the mother or the father, are more likely to develop breast and ovarian cancers at younger ages.
As soon as Shannon’s insurance covered the testing, she had it done. Fully expecting a positive result, Shannon was prepared to have a preemptive double mastectomy with reconstruction. But, Shannon’s results were negative. She doesn’t carry the BRCA 1 or BRCA 2 mutations.“I was shocked,” says Shannon, and she feels like the results gave her a little false security. That’s where the anger came in, because in August 2015, when her mammogram and subsequent 3D testing showed a black and jagged spot of concern, she knew that meant she was bound for chemotherapy, and that was something she had always planned to avoid by having preventive surgery. “I was 100 percent prepared to do that, and it didn’t work out that way,” says Shannon. Cancer, as it often does, had other plans.
Cancer had other plans for Tina Donahue as well. “It was a really, really difficult time in our lives,” says Tina of her diagnosis in 1991. It’s not that Tina wasn’t expecting to get a diagnosis at some point. She also had a family history of the disease, her maternal aunt died from breast cancer, and Tina was a nurse so she had a keen understanding of her risk, but when she was diagnosed at 44, she had just been promoted to an executive vice president position at work, and she had three young sons. She was also in school to get her MBA. Cancer was not part of her plan, and she thought she was going to have to quit school when she was diagnosed. However, thanks to the support of the other women in her study group, Tina didn’t have to quit school. She says the women rallied around her, told her not to quit and helped her, encouraged her, and tutored her through.
When it came to treatment, Tina and Shannon, though diagnosed more than 20 years apart, had very similar methods. “I just wanted to hit it as aggressively as I could and give myself as much life as I could,” says Shannon. She told her surgical oncologist that hers would be the easiest consultation ever. She had done the research, she knew the risks, she knew exactly what treatment she wanted. Tina, who also wanted to treat her cancer aggressively says she told her doctors, “Give me everything you’ve got.” Both women had a double mastectomy and reconstruction with silicone implants. Tina says her implants lasted 23 years before she noticed they started getting folds in them, which was a sign that both implants, though contained, had burst, and she had to have them redone. Shannon’s implants are a newer technology called gummy bear implants and are designed so that they won’t burst. Tina says the silicone felt and looked more natural, and Shannon says that was important to her as well. Tina also says that if she hadn’t been a nurse who had seen a lot of recurrence in women who had had a single mastectomy, and if she hadn’t been witness to her aunt’s experience, she may not have opted for the double mastectomy.
Diana Geiser did not opt for the double mastectomy, but now says she wishes she had. Diagnosed at age 50, Diana says she struggled with the decision at the time and remembers feeling like she wanted to keep part of herself. “Now I wish I’d done both,” she says explaining that one of the draw backs is that her natural breast gets bigger or smaller with weight fluctuations, but her reconstructed breast does not.
Regardless, all three women had four rounds of chemotherapy. They all had clear lymph nodes, and were hormone-receptor-negative (HR negative), meaning that it was likely that hormonal therapies wouldn’t work for them. Tina, still wanting to treat her cancer aggressively, says she wanted to kill everything and had low dose chemotherapy. She lost some of her hair, but not all of it. For Shannon and Diana, the pathology reports came back showing their tumors were aggressive, Shannon’s highly so, making chemotherapy necessary. They both lost all of their hair, which is something that must be incredibly pertinent to breast cancer survivors, because whether they did or they didn’t lose it, they all tell you about their hair.
Jennifer Lessinger is a professional writer and editor who learned the value of patient empowerment during her struggle with a hard-to-diagnose and complex endocrine disorder.
https://powerfulpatients.org/pen/wp-content/uploads/Untitled-design-1-5.png600600Jennifer Lessingerhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngJennifer Lessinger2018-10-16 18:32:512019-10-09 09:00:29Patient Profiles: Breast Cancer Part I
Clinical trials offer tomorrow’s medicine today, but more often than not, only a small fraction of patients ultimately enroll in a trial due to barriers posed by financial logistics, distrust and travel, to name a few. In this MythBusters program, we will examine the barriers to enrollment, evaluate patient needs and discuss resources to help guide people through the clinical trial process with the help from two experts, Dana Dornsife of Lazarex Foundation and Myeloma Survivor Reina Weiner.
Andrew: Hello from Carlsbad, California, near San Diego. I’m Andrew Schorr from Patient Power. Welcome to today’s Patient Empowerment Network program, clinical trials myth busters and actionable advice, resources for knocking down obstacles to trial participation. I wanna thank the companies that have provided financial support for this program. They have no editorial control, but we definitely thank them for their support. Those supporters are AbbVie Incorporated, Astellas, Celgene Corporation, and Novartis.
Okay. We have a lot to talk about. First of all, I’ll just say I’ve been in two clinical trials; one Phase 2 many years ago at MD Anderson for the leukemia I have, chronic lymphocytic leukemia. And that gave me tomorrow’s medicine today. It worked, but I had travel far to do it and there were costs involved.
And then I was in a second Phase 3 trial close to home, and that was good too, and discovered another cancer that I have, myelofibrosis, through the monitoring in the trial. So, I’m a believer, but there are obstacles, and let’s talk about some of these. And we’re gonna give you some very specific resources to overcome these obstacles, so that hopefully, if a clinical trial is right for you, you can participate, you can feel good about it, and you can move medical science along to help everyone who is dealing with that condition.
So, what are some of the issues? Financial, of course; logistical issues, of course; distrust, are they really gonna take care of you or are they gonna protect your safety? Is it really right? And are you being given the straight scoop? What about travel costs? I went from Seattle to Houston, Texas a few times. Costly, okay? Stay in a hotel. It’s costly. Get a babysitter, leave work; costly.
The guinea pig syndrome; you’ve heard about it so many times. Are they gonna experiment on you, and are they really protecting you, and are you a number, or you are a person with cancer, or your loved one? And then is your medical team that you’re talking to about your treatment, are they informed about clinical trials? Or are they pooh-poohing clinical trials because they don’t wanna do the paperwork, or it’s happening down the road and not at their clinic. Lots of issues; we’ll talk about that.
Okay, I got some great helpers. So, first let’s go to Asheville, North Carolina, and you are used to live in Charlotte. Reina Weiner joins us. Reina, welcome to our program today. There we go. Say that again, Reina, you were muted.
Reina: Thank you.
Andrew: Okay. Now we should tell you that last June, well, June of 2017, Reina had a autologous transplant for multiple myeloma. And along the way, leading up to that, over many years she was in four trials. So, first of all, Reina, let’s start with what’s most important. Post-transplant, how are you feeling today?
Reina: I’m feeling very well. Thank you, Andrew.
Andrew: Okay, and what’s coming up at the beginning of September?
Reina: What is coming up at the – oh, a big party is coming up. Our children are throwing us our 50th wedding anniversary party, so that’s been cool.
Andrew: Yeah. Well, congratulations. And you’ve been dealing with what became multiple myeloma since 1999. We’re gonna come back and track that in a minute, as far as the steps along the way, the concerns you had or not at different times about being in four clinical trials.
And now let’s go up near San Francisco in the East Bay of San Francisco Bay, Danville, California. Dana Dornsife. And Dana is the Chairman of the Lazarex Cancer Foundation. Dana, thank you so much for being with us.
Dana: Thank you, Andrew.
Andrew: Okay. Now ladies and gentlemen, I want you to know Dana and her husband and her family, overall, they’re incredibly philanthropic across a number of issues that are faced globally, and also in the US. But one of them is helping people with the financial issues that prevent them from being a clinical trial. So, Dana, this is a personal story for you, so maybe you could just tell us why did you start the foundation? It was a family issue.
Dana: It was a family issue, and that family issue really revealed to me a gap that exists in cancer care for advanced-stage patients who want to remain in their battle with cancer through clinical trial participation. My youngest sister’s husband, Mike, was diagnosed with pancreatic cancer in his early 40s. He was given one half of one percent chance to live, and at the time 35,000 people a year were diagnosed, and 35,000 people a year were dying from pancreatic cancer.
So, we decided as a family that if Mike wanted different results that we would need to do something different. And Mike and Erin went ahead and pursued standard of care, and I was tasked with identifying clinical trial opportunities for Mike. And, of course, that sounds very linear, but in fact, for a layperson it was a very difficult task to undertake. I did identify some trial opportunities for Mike. He did participate in a trial and responded well for a period of time, he had good quality of life.
And during that period of time he was meeting people who were asking him, “Hey, what are you doing? I wanna do what you’re doing.” And he would say to them, “Oh, just call my sister-in-law, Dana. She’ll help you.” And that’s literally how this organization began. Through those phone calls that I was receiving from other pancreatic cancer patients, I began to understand that Mike was able to take advantage of medical breakthroughs in clinical trials because he had a family who could afford to support him through the process. And all of these other families that I was talking to, they just didn’t have the financial wherewithal.
So, we started Lazarex in order to fill that gap and help people identify clinical trial opportunities, and then provide financial assistance to them to help cover the out-of-pocket expenses that create huge barriers for patients who are already experiencing financial toxicity due to their disease.
Andrew: Well, thank you for what you do. And we’re gonna talk a lot along the way about resources. There’s a downloadable guide that you’ll be provided with, along with a link to the replay of this program. And that’s gonna have specific resources that you can access, whether it’s financial issues, other issues you may be facing. So, look for that.
Today we’re really focused on actionable resources. So, let’s go to Reina for second. So, Reina, you had been in the pharmaceutical industry.
Reina: Yes, I had.
Andrew: So, you knew about drug development, and you understood about clinical trials. So, I’m willing to bet you were pretty proactive. People who weren’t in the field, they don’t know from clinical trials, and maybe they’d been worried about it. They’ve worried would they be experimented on, would they be a number and not name, would they get quality care. But you were probably, I have a feeling, pretty proactive. And you write about that. I know you have a book as well. So, is that Step 1 for people to speak up for themselves?
Reina: It is Step 1; absolutely, Step 1. And what I found is, first of all, people don’t know about trials. And if you go to a small community practice where they’re very busy, they don’t have the time, they don’t have the staff to really educate patients about trials, the best, best step for patients to take is to ask, “Is there a clinical trial that might be appropriate for me?” That’s huge.
Even when I went to a very well respected hospital and there was a researcher who was following me as I had smoldering myeloma and the numbers kept going up and up and up. I said is there – because I was living close to the NCI – is there a trial that would be appropriate to me at the NCI. And he said just a minute, turned around, went to his computer, found the trial and that’s how I got in.
Andrew: But it wasn’t at where you were receiving care or being monitored at that time. It was somewhere else.
Reina: It was not. It was at somewhere else.
Andrew: Okay. Dana, is one of the obstacles, not just financial, or maybe it’s even the business of cancer where if an oncology practice that you’re going to that’s maybe close to home is not doing the trial, maybe it’s not even in their financial interests to tell you. I mean, is there an awareness issue, do you feel?
Dana: There’s a huge awareness issue there, Andrew. And it all starts with knowledge is power, right, so I completely agreement with Reina’s comment about one of the first questions you need to ask is, is there a clinical trial out there for me because many doctors who are in community environments don’t offer that information. It’s not what they do every day. They’re there to administer standard of care. Only 6 percent of doctors actually engage in conversation with their patients about clinical trials, and that’s usually the 6 percent who are associated with research universities, right?
So, knowledge is power. If the patient doesn’t know about a clinical trial, they’re never going to participate. But once you find out and once you’ve identified an opportunity, the second biggest hurdle is that out-of-pocket expenses associated because most patients have been dealing with their disease for a longer period of time, and they’re basically broken in every way: physically, emotionally, spiritually, and, sadly, financially.
So, patients start to make decisions about the outcome of their care based on the size of their checkbook, and not focused on what’s best for them. And so, Lazarex eliminates that financial barrier as well to help patients say, “Yes, I can participate,” and we can get them where they need to be when they need to be there.
And that’s just the tip of the iceberg, Andrew, because there are many other barriers that exist; socioeconomic barriers, language, culture, historical barriers. And we are tackling all of those barriers one at a time. But really, the biggest two barriers are knowledge and financial.
Andrew: Right. And you mentioned about the historical barriers. Some people know about the Tuskegee experiments with African-American people, so in the African-American community, there still is a distrust among some people. Yet if you think about it from the FDA’s point of view where a company that’s developing a drug, or the NIH, they say okay, how does it work on broader populations or different ethnic groups or different ages or genders, et cetera?
They want to understand that data, and so not just having a number of people participating in the trial, but having it reach people who are in different situations, if you will. And so –
Reina: And if I may – ooh, I’m sorry.
Andrew: Reina, please, go ahead.
Reina: Well, if I may say that because people don’t know about it and the trials, the best trials, are trials with a variety of patients, but they do try to accrue populations who are certain ages, certain genders, ethnic groups, whatever they can get. And only 3 to 5 percent of patients participate, cancer patients, participate in clinical trials, and so much is lost if people don’t participate.
Andrew: Here in San Diego it’s sort of a pocket of a lot of medical research. There’s a lot up in your area, Dana, in the Bay Area, San Francisco Bay Area. I mean, it’s in North Carolina in the research triangle where that’s home state for Reina. And not to disclude others, and then certainly up around Boston. There are like companies all over the place and many of them are in earlier drug developments.
So, when you talk about immuno-oncology now, can we harness our immune system with the help of some medicine to fight the cancer, and I know some people who’ve received it; lung cancer patients who are living, et cetera, melanoma patients who are living for an extended time. These companies can’t move forward unless there’re people who are in the trials. So, the FDA says where’s your data? And they’re saying well, we’re trying, but we haven’t been able to complete this trial. Right, Dana? So, we can’t move towards cures unless we all come together.
Dana: That’s exactly right. So, let me just throw a few statistics out at you that I found astounding when I learned of them. So, we have a 48 percent failure rate of clinical trials, and it’s not because the drug didn’t work. We will never know, quite frankly, if the drug would have worked or not. And we will never know because there weren’t enough patients enrolled in the trials to find out.
So, 11 percent of trials never enroll a single patient, if you can believe that. So, here we are with an almost 50 percent failure rate, and yet we have 600,000 patients a year in this country who are dying from cancer. So, there’s this incredible disconnect between the thousands of patients who would participate in clinical trials if they could, and the thousands of clinical trials that need patients to participate in order to succeed. And without successfully completing those trials, those drugs are never going to get market to help the cancer patients that they are intended to serve and help.
That’s why Lazarex Cancer Foundation exists, and that’s why removing the barriers to clinical trials is so important. Our process does not lend itself well to that. And I just want to take a step back, Andrew, to address the minority participation in clinical trials. We all understand because of epigenetics and, yeah, advances in medical science that we need to have the full spectrum of our population participating in clinical trials. But that doesn’t happen. When you look at the 5 percent of patients who actually participate and you break it down ethnically and racially, less than 5 percent are from minority communities combined.
So, in theory, though we say we understand the importance of that, we’re actually not in practice doing what needs to be done. And so a lot of our work is also focused on reaching out to those socioeconomically challenged and racial and ethnic minority communities to raise awareness and help people like you’re doing on this program dispel the myths around clinical trials, so that they’re more inclined to ask better questions.
Andrew: Right. So, so important, and I applaud for that work. We’re gonna talk about the financial process in a second. Reina, so you were involved in a National Institutes of Health or National Cancer Institute trial.
Andrew: A couple of them, I think, and one at Memorial Sloan Kettering in New York’s premier resources. So, we talked about your tip was you gotta speak up and ask about trials, where they’re at that center, wherever you are, by XYZ oncology in a suburban area, whatever it is or not. So, what’s Step 2? So, for instance, now I understand there are people – and Dana, I’d like your comment on it too.
At some clinics now where there are clinical trial – there are nurse navigators, but often sometimes there are clinical trial navigators too, but often you gotta ask about that too, right, Reina? I mean, it’s speaking up and looking for the resources that are available to you there or wherever you choose to go, right?
Dana: Yes, and there are organizations like Dana’s who help people do clinical trial searches because that’s a bit overwhelming when you are already frightened, you already have the financial issues coming up. And like you mentioned, logistical issues. So, there is Dana’s organization; therefore, myeloma, the SparkCures. There’s the MMRF. There’s the International Myeloma Foundation. There is something called Cis Crypt. And so, they will help you find a trial.
And there are lots of regional trials groups, so you may not need to go to the big, big research center. They might be able to do it locally for you. But I always want to bring up the fact that there’s so much misinformation about trials and what it entails. There’s a tremendous amount of fear. And when I went on the first trial, as I wrote about a little blog recently, everybody said to my husband – well, not everybody, but an awful lot of people said why would you let your wife going in a clinical trial? She’s definitely gonna be a guinea pig.
And I can tell you very, very, very clearly that you get so much care. There’s so much documentation. And the patient’s health is never sacrificed for the research ever. And so, and you sign a consent form, so you’re very clear about what is going to happen. And yes, there’s more there’s more bloodwork. Yes, there are more biopsies. And it’s part of research. And when you sign up, you sign up. And I had more than I’d like to even talk about, but I feel very grateful and very humbled for the care that I received.
And I can tell you, too, that I talked to other people on the trial. And yes, they hope to gain better control of their cancer. But, in addition, they really hope to help the next group of patients who are coming up, so that these new treatments actually happen.
Andrew: I feel the same way. I was in a trial at MD Anderson in 2000, and the three-drug combination I got was not approved till ten years later, but they learned a lot. And you were on a three-drug combination, which I think still has not been approved for first line, but it’s is widely used, I think.
Andrew: So, in multiple myeloma. I wanted to mention some other resources, the Leukemia & Lymphoma Society also has a resource center. You can call them. So, there’re these different groups that help you identify a trial, and doctors who specialize, so let’s say pancreatic cancer, you mentioned earlier Dana. I got a call from a friend in Miami, “How do I find a pancreatic cancer specialist?” And I connected them with PanCAN, Pancreatic Cancer Action Network in Los Angeles, who knows who are the doctors who have the most experience with that.
Now, Dana, so then the next thing comes up is alright, I’ve identified the trial, but it’s not where I am. So, now we talk about logistics and finance. So, let’s say somebody calls your foundation. Tell us how it works. So, I don’t have the resources. Maybe they live in Northern California and the trial is in Southern California or in Salt Lake City. What happens next?
Dana: So, Andrew, in some cases it’s not even that distance. In some cases it’s getting from Sacramento to San Francisco, which is literally a one hour, one-and-a-half hours without traffic, in your car. And sometimes it’s a tank of gas, a bridge toll, and parking. I mean, we’re not talking about thousands of dollars in some cases, but it’s still the difference between life and death.
When someone calls Lazarex Cancer Foundation, they can contact us directly. We have a financial application that we use to determine the degree of eligibility for patients to get their out-of-pocket expenses reimbursed. Or they can be referred to us by their social worker at the institution where they are receiving, or thinking about participating in a clinical trial.
We take a look at the household income of the patient, and I believe our guidelines are very generous. We go up to seven times the federal poverty guidelines for patients. And we arrived at that number through trial and error. Our goal is to help as many patients as possible participate in clinical trials, and turn away as few as possible. And then we reimburse on a sliding scale from 100 percent to 75 percent to 50 percent depending upon your household income.
And it’s a pretty easy process to go through in order to be enrolled and receive the reimbursement. And then we reimburse our patients monthly, on a monthly basis. And in some cases, we’ve been working with patients, we follow them, like Reina, through two, three, four clinical trials. And we’ve been supporting them in trials for years. And without doing what we do, they may not be here with us today.
Andrew: Well, I’m sure you’ve saved some lives and lengthened some lives. Reina, so you were in the pharmaceutical industry and in oncology, I believe, before all of this started happening to you. And you’ve continued teaching nurses and devoting yourself to education and your book and your blogs. Thank you for all that. Maybe that’s what life’s about.
But knowing on the inside there are pharmaceutical programs, in some cases, I think, particularly for rare cancers where they may provide assistance. They can’t pay you to be in the trial, but there are at times assistance and travel logistics, particularly for rare cancers where maybe the trial is not, not one hour away. Am I right, Reina? Are you familiar?
Reina: Oh, there are. And sometimes when I was working, there would be a patient who had a cancer that really was not aligned with a particular treatment that would be effective for them. And so, the doctor wanted to try an off-label use of a product, and so then they would come to me and asked me if I could get the pharmaceutical company to provide the drug for free.
And sometimes it takes a little doing, but I was concerned about the patient and hoping to get them a better quality of life, if not an extended period of life. And so, yeah, the company would do that. Not every day, not all the time, but if the company had evidence that this was a patient who would benefit from the off-label use of a product then they would help them out.
Andrew: Okay. So, Dana, related to other organizations providing assistance, and I recently interviewed someone from the Family Reach Foundation where they help with rent or things, groceries, things like that. So, somebody says, “Oh my God, I’m afraid of a trial, I can’t go there,” or if they hear about it and they say, “Hmm, well, maybe I could, but I’d have to leave work, or maybe my spouse would have to leave work, we’d have to find somebody to pick up the kids from school, oh my God.” There are organizations that can help with some of these family processes, aren’t there?
Dana: Absolutely. And I think we’ve provided the Patient Empowerment Network with a list of those. 21st Century C.A.R.E. is an organization that provides patients with immediate financial assistance for expenses related to active cancer treatments. Cancer Care provides assistance for cancer-related costs. There’s a Cancer Care Co-Payment Assistance Foundation. We get that question a lot.
We’ll help with the out-of-pocket travel expenses, and in fact, some of the medical and diagnostic expenses that aren’t covered by insurance. When you’re participating in a trial, sometimes you have to get more stems than insurance will cover or whatever. But co-pays are a big deal for people to be able to afford those, and so, that is another organization that can help. Patient Advocate Foundation, which is an underinsured resource directory.
So, there are a lot of you nonprofits out there who exist to support patients through the fifth process. It’s just a matter of helping patients really understand and put together all of those resources in a way that they can access them.
Andrew: Okay. So, Reina, you’ve been through it four times, and you’re a pretty savvy person. Not all of us know as much, so help us now. So, one of the questions in a trial is, and in cancer, am I gonna get what I describe as the good stuff, knowing that the good stuff that’s being tried may not be good. I mean, it may not work out. There are trials that go bust. Not just for not getting people, but they got people, but it wasn’t as effective as they hoped it would be.
But let’s say we’ve done our homework and we go to a certain clinic, but it’s some sorta controlled trial. We don’t know whether we’ll be in the arm. So, was a concern for you? Were you gonna get the good stuff, and why do it?
Reina: Well, no, really, Andrew, because I know that like if it’s a Phase 3 trial, so you’re comparing standard of care versus the newest and hopefully the latest and greatest. If it turns out that one arm of the trial really shows a significant improvement, patients are always switched to the more effective arm of the trial. They don’t leave you on this arm of the trial thinking well, what the heck, we’ll just leave you there and see how the research pans out. So, they are always switched over to the most effective.
So, I wasn’t really concerned about that. And in the Phase 2 trial, it’s just seeing if the product was effective. And so, that was obviously not a concern for me. So, it worked out, and I do think, though, like what Dana does is absolutely wonderful at totally, totally, totally past wonderful.
But I always try to let people know who have friends and family who are facing some chronic significant illness that don’t just call and say let me know, let me know if I can help you because that’s so ambiguous. And most people will not call because they have pride or they think they can do it all by themselves.
So, I always try to suggest to people that if you’re calling somebody who you think might need some help, be specific. Call and say, “Can I walk the dog? I’m going to the grocery store in an hour. Is there something I can pick up for you? Can I mow the grass?” Anything that will help, but make sure that you are specific in your offering.
Andrew: I want to talk about a related issue. You use the word pride. Some people, maybe in some cases it’s even shame. They developed a certain cancer. Where these are maybe middle-class people who’ve had some resources. They’ve been paying their mortgage. They’ve been paying their expenses, making do. But now they get hit with a cancer diagnosis, which is catastrophic, and there is help available, Dana, but they’re too proud to ask for it when this could happen to anybody. And maybe you’ve even countered that along the way or know there’re people out there. What would you say to people, to not be shamed and to speak up?
Dana: Yeah. Well, sadly, one in three women will be diagnosed with cancer, and one in two men. And so, this is not an uncommon scenario, right? The likelihood of knowing someone who will receive a cancer diagnosis is very likely. So, I think that patients have to understand that pride doesn’t help you in your process with battling this disease. You have to take advantage of every opportunity that’s out there in order to come out on the positive side of this experience. And if you don’t take advantage of every opportunity, you may not.
And so, it’s one of those things that we just have to deal with right from the beginning, and just say okay, again, knowledge is power. I’m going to surround myself or engage with the people that are around me who want to help me. And you have to put that team together because you will need your team with this disease.
Andrew: Okay, so great advice. Reina, part of your team maybe could be the first doctor you saw who gave you the diagnosis, but they might not be the one where a trial was offered. So, first step is you talked about speaking up, but it takes a lot of courage to say to the doctor in the white coat with all the letters after their name, you know, thank you so much, Doctor, and I’ve either found out about a trial, or your turned and typed it in somewhere else. I hope you don’t mind, but I am going to go over there. Maybe you can advise me along the way.
But that takes courage because people are terrified, and they may be bold in principal in that situation with the person in the white coat. What advice would you give?
Reina: Ooh, well, that’s a big one for a lot of people. And, really, you know what, I imagined that it would be people who are older, who come from a generation where the doctor has the final word. But what I found out when I was writing my third book is that there were younger people who also feel very uncomfortable speaking up, asking a doctor, and so forth. But really, what to really put in your little mind and in your heart is this your life.
This is not just kind of a trip to the mall. This is really important for you to either improve the quality of your life or extend the quality of life, so take a deep breath, be very polite, and I think most doctors who are professional and open-minded will hear what you have to say if you present it in a way that they can hear. And if they really don’t hear you then it might be time to have a look around to see who will.
And, really, the bottom line is you need to trust yourself. And if you feel that this is really right, that there is a clinical trial that you would be eligible for and you can participate in with Dana’s help, with the financial, with the logistics, and so forth. Like I said, you just take a deep breath. And most doctors, like I said, really want the best for you.
Andrew: Okay, let’s talk about something that comes up. One of the things for people is the criteria of different trials. Dana, I don’t know if this is in your area, too, related to financial, but people let’s say okay, I wanna be in a trial, but the criteria are so narrow that I really wanna be in the trial, but they say I can’t.
Dana: Yeah, so that is a sad reality in many cases. And I refer to this as Clinical Trial Nirvana Syndrome where as a drug sponsor for trial, you want to attract the healthiest patients you can to participate in your trial, so that you have the greatest chance of success. But, unfortunately, in many instances, in most instances, a cancer diagnosis is accompanied by other comorbidities like heart disease or diabetes or other maladies that would preclude a patient from being able to participate in a trial.
So, that is an area that we are looking into and trying to – we have several proposals out there with various aspects of our government to try and really take a closer look at that, to try and make the trial makeup in relation to patient participants better mirror the realities of our situation because the likelihood of someone, if the drug gets approved, taking that drug and having a comorbidity is pretty likely.
And yet we won’t know what will happen there, right. So, we have to drill down on these issues and it’s a great, great issue to bring up. So, we’ve got a lot of work to do ahead of us.
Andrew: Right. And another thing that comes up too, and Kevin sent in a question. Kevin, thank you for this, matching what’s available in clinical trials to where you are in your journey with an illness. So, on Day 1 you’re diagnosed. I know Esther and I, we were crying and almost on the floor. And I thought I would be dead the next day. And it really took a while to overcome the terror of the diagnosis. And so, we were not even – well, the doctor wasn’t talking about trials; we wouldn’t have been hearing it anyway.
And some of us, thank God, with some trials, with some cancers now, are blessed with living longer and we start to learn. And then we want to know, in our situation, what applies to us. So, I know there are a lot of efforts being made to match trial offerings to where you are and what you might need to know now, what might need to be offering.
And some of you have heard this term, artificial intelligence, where we in the Internet business are all trying to fine tune what we’re suggesting or putting in front of you based on who you are and where you are, recognizing privacy and all those kinda things to make it more manageable.
We still have a long way to go. I mean, we have clinicaltrials.gov, but it’s not tied to where I am, who I am, where I am in my journey. It’s just what’s being done in a certain illness, right, Reina?
Andrew: So, we have to refine our tools.
Andrew: We have to refine our tools. Well, we’ve been getting in a number of questions. So, here’s one. So, David; so, he says as the excessive use of CT scans in clinical practice moves away from being the norm, have they lessened their use in clinical trials? In other words, this is about testing, and maybe it’s about the requirements.
Dana, I don’t know if you have feelings about it, but the scientists who are doing these trials, they wanna know everything. They would like to test us. So, the CT scan, and I mean I’m gonna have one next week, but it has radiation, right?
Andrew: So, let’s do a bunch of CT scans. No, let’s do a bunch of bone marrow biopsies. No. So, I’m saying I’m sorry. Not just do I have to pay something for these tests, or is there a co-pay or whatever, but also am I gonna be radiated? Am I gonna be poked? So, what about those issues? Is there dialogue going on, not just to help us financially, but also make it less onerous, I guess?
Dana: Yes, in fact there is dialogue going on about that, and it’s good, heartfelt dialogue. And it’s coming from a myriad of stakeholders, right, not just from patient advocacy organizations, but also from within industry insurers. And the whole goal is to okay, let’s stop looking at patients as a chart or a number on a piece of paper, and let’s understand that these are living, breathing human beings who are voluntarily participating in this clinical trial process for the benefit of not only themselves, but future patients to come and our industry.
And let’s start treating patients as humans who are participating, and let’s see what we can do to lessen the number of visits or minimize the number of scans and blood work, et cetera. So, there is active dialogue around that, and I think there’s a much higher degree of sensitivity on behalf of the teams who are actually putting the protocols together now.
Andrew: All right, I think so. And I know in some cases they’re doing what’s called trial simulations with a panel of patients and saying okay, we’re trying to answer these scientific questions and see if this drug that’s in development can do better for patients and would require so many office visits. Or so many, you come to the site, but so many could be done, maybe with your local doctor if that’s closer to home. So many blood tests, so many CT scans, so many biopsies. Imagine lung cancer patients with another lung biopsy. Not fun, and often not available.
So, there are all these kind of questions. And I think that’s going on although it needs to happen more. Now Dana, do you talk to the pharmaceutical industry? We had a question from Vi Life wanting to know related to trial awareness. Beyond the financial, do you work with pharmaceutical companies at all, as you are now, today? I mean, what we’re doing here is just to raise awareness about trials or other programs that you may do.
Dana: So, we are engaging with pharma right now. We were very fortunate to work with the FDA earlier this year in securing language around reimbursement of patients’ out-of-pocket expenses associated with clinical trials. There was some very nebulous language out there that was really preventing pharma from being able to support programs like ours.
And what we’re doing now is, in addition to we’re bridging this gap for patients that exist every day by reimbursing patients, but that is not a sustainable business model. It’s noble, but we have to have our tin cup out every day. And the number of patients we can help is directly related to the amount of money that we have in our account, right?
So, in addition to that program, our Lazarex Care Program, what we are also doing is trying to fix this problem and do it in a sustainable way. And in order to do that, we actually have to shift the burden from the patient back into industry, right, and help industry understand why they should include these out-of-pocket expenses as part of the clinical trial protocol every time, right, so they can enroll trials on time, on budget, save R&D dollars, preserve patent years, right?
I mean, there are a lot of reasons why pharma would want to participate in a program like that, in addition to the fact that it’s the right thing to do, right? And then we get more drugs to market faster, and we provide a platform of equitable access for everyone. So, we are engaging pharma in discussions right now about funding this program, we call Lazarex our IMPACT Program, that’s being rolled out at comprehensive cancer centers across the country. And it stands for Improving Patient Access to Cancer Clinical Trials.
It has been received very well and I’m happy to say that Amgen actually stepped up and funded this, so we are rolling it out here in California, and we are hoping that we’ll have similar opportunities in a couple of other areas in the United States. So, they are interested, and they want to improve clinical trial enrollment retention, and especially minority participation.
Andrew: Right. Boy, that you. Again, I keep saying thank you for what you’re doing, but you’re a real leader in the field. I’m going back next month to the Biden Cancer Initiative Summit continued by Vice President, Biden, former Vice President Biden, and his wife who continue to do leadership in this. And there’ll be a lot of senior people there and I’m hoping we can talk. And I know this issue of how can we advance cancer care through research in partnership with patients is a big one. So, Dana, thank you for helping lead the way in getting this going. And thanks to Amgen just as an example.
Reina, so, we talked about the cultural differences of people being in trials. We talked about the pride people may have in asking for assistance, the fear people have maybe participating in trials. You still have a – not now. I mean, you’re doing so well and you’ve been through trials and it’s worked out well. But there must have been some bumps along the way. Were there any misgivings at different times? And if so, how did you overcome it?
Reina: Oh, yeah. Well, there were definitely misgivings, I am sure. The first trial was when I asked the doctor if there’s something going on at the NCI. And there was no misgivings about that because that was a very observational trial. The second trial was much more progressive and I felt kind of a little uncertain about it, and so I asked the researcher at this well-known institution if I should participate because the trial, I should back up a little bit, that was for either smoldering myeloma patients or active disease patients.
At the time I was smoldering, and most physicians didn’t believe that that was a good idea to treat smoldering and wait until it became active. So, I asked this one researcher and he said absolutely, not, do not participate in the trial. And then I called someone else also from a very respected institution where I had been, and he said well, if you join that trial you’ll be crossing the Rubicon, which I didn’t even know what the Rubicon was at the time. I had to go look it up.
But, basically, once you start treatment, you kind of go on that journey and there’s no way to step off. But then I thought about it, I thought about it, thought about it, and finally I decided to trust myself because I had been to the NCI. I felt very safe there. And I decided to move ahead with it.
So, yes, I had plenty of misgivings about that. The other trials, not really because that trial changed my life and it gave me a very reasonable complete response. And the other ones, like I said, they just kinda fell in with the collecting a good amount of stem cells for a transplant and so forth.
Andrew: I wanna talk about family issues. So, the decision to be in a trial affects the family, whether it’s somebody’s driving you to the doctor, somebody’s taking off work, their worry, how they feel about trials, their own view of it, family logistics, costs, et cetera. We’ve talked about that. So, you wrote this blog about people questioning your husband, I guess, was your wife gonna be in a trial? So, how did you overcome that, whether if not with your husband, just with your community that you weren’t like crazy?
Reina: Well, they already know I’m crazy, so that’s a total aside. But, really, trying to educate people about the misinformation about trials; say, look, I will never be a guinea pig because that’s not what trials are about. And it’s very well controlled and there’s a lot of data that follows you. The care that I got was excellent.
And I try to dispel, like I said, a lot of the myths; that you signed consent form, which clearly explains what the trial is about, what your commitment to it is, and you can also drop out for any reason. There was hope that you don’t because they would like to have some results that then will lead into future treatments for patients. But you can drop out, so, really, taking that opportunity to educate people about what a clinical trial is like and that there are no guinea pigs.
Andrew: I wanna just – oh, yes, please, Dana.
Dana: Yeah, if I could just offer something in that regard. For people who haven’t gone down this path, the journey with cancer, having a cancer diagnosis is not like other chronic diseases, right, like diabetes, for instance, that you can typically control with insulin or whatever, right? For a cancer patient who has failed standard of care, who’s gone through maybe second- or third-line treatment options, but still has progressive disease, that patient will die if they don’t do something, right?
And so, clinical trials offer tomorrow cures today in some instances, right, and we don’t always have positive results in clinical trials. But for a patient who’s at that crossroad where their doctor has delivered those words, “You need to get your affairs in order,” right, it’s not a matter of am I crazy if I participate in a clinical trial. What it is a matter of is do I wanna live? And if so, what clinical trial can I participate in? It’s a very different decision tree.
Andrew: Right. And I certainly say that all the time. I got a call, as I mentioned, from a friend in Miami. The mother has a very serious cancer. And I said part of the initial discussion, even the initial discussion, Dana, can also be are there clinical trials that we should consider along with standard therapy? So, certainly, if you’ve failed or they’ve failed you, the treatments no longer work, what is the 360 degree view? And if you don’t do it here, so they do it down the road, or do they do it across the country? And what are the issues for you participating?
So, a lot of thinking, but it’s gotta be part of the discussion. So, so sadly now, what are we seeing; 3 percent, 4 percent, 5 percent of adults participating in cancer clinical trials in the US. Not good at all. And are we hurting ourselves with the chance of future therapies that can be more effective, or even cures because some of these companies sometimes are venture-backed. They don’t have money forever, you know, and they’re trying to get to the goal line to go the FDA.
Look, here is another question we got in. Tamara, our producer, just sent in. She says well, what happens when you join a clinical trial and it doesn’t have a beneficial impact? So, Reina, they didn’t know that the trials would necessarily work out for you. So, what happens then? Do you go on another trial? What do you do?
Reina: Well, if it doesn’t and you don’t seem to be responding to the therapy on the trial, or you find it intolerable yourself, then they will always return you to your oncologist who you had been seeing previously. But, on the other hand, they may offer you another trial that’s available that you would be eligible for as well. So, I mean, I really try to stress to people that the researchers are looking out for you. They want the best income, in addition to accruing the data that they hope.
And I can tell you that when I was on a trial at the National Cancer Institute, when I had questions, especially about the trial with smoldering versus active disease for myeloma, they would spend a couple of hours for me, explained with me, can I say that, right? Yes, spent a couple hours with me explaining all of the aspects of the trial, so yeah.
Andrew: I wanna point up an example that some people have heard of a woman I’ve become friendly with in the myeloma community, Reina. Cherie Rineker. So, Cherie’s down in Houston, and she was dying of myeloma. And she’d been in trials and treatments. She was at MD Anderson. Bob Orlowski is one of the top doctors in the world, and her doctor. And she was in different trials and then things were not working.
And she was put in touch with another researcher doing this CAR T investigation for multiple myeloma, which is pretty new, pretty new. And they’re learning a lot. It’s not a slam dunk, but so far it’s worked for her. It saved her life. She went to Nashville, Tennessee from Houston where she lives, and maybe I’m not sure the financial issues, Dana, about going. But that’s where she’d been in successive trials. And some were not working or no longer were working. There was another approach.
I wanna ask about another concept I’ve heard called siteless trials. And I don’t know, Dana, you’re nodding your head. Maybe you are familiar with this. One is a siteless trial because we talked about these trials going on at these academic medical centers, but not much elsewhere.
Dana: So, I have tell you, I don’t have a lot of experience with siteless trials, but there is a lot of dialogue taking place around rather than having the patient go to the trial, bringing the trial to the patient, and I think that’s the impetus behind a siteless clinical trial.
I think cancer has some unique challenges, especially blood-based cancers in clinical trials, and the oversight of patients participating in those trials that make siteless trials a bit of a challenge. But I think the place to start is in other diseases, or perhaps where you have a cancer diagnosis that’s not a rare form of cancer, whatever that requires, a high degree of oversight.
But the whole goal in doing this is to understand how we can get more people into these trials and make it less obtrusive on their life, right, so that more patients would be inclined to participate, increase our enrollment retention, our minority participation, and, ultimately, reduce the burden on the patient to participate.
Andrew: Esther and I’ve given a lot of talks at different conferences, and we said you have to see patients who might be considering or are in a trial as investors. So, they’re gonna invest with their body, their time, sacrifices, and other things in their lives for the hope of being cured if they could, or doing better.
And there needs to be the communication, financial support, logistical support in really treating people with a lot of respect as a person. Reina, do you agree with that, that we have to get to that concept where we’re taken care of? And you felt that way, but we need to do it for more people and have more people feel confident that it’ll work out that way.
Reina: Oh, certainly. Certainly, I do. And the education is really essential. And after I was in the first trial, I talked to everybody who would listen to me. And even if they didn’t, I would talk anyway just to try to say this is a place where you can go where you will receive what is hopefully the newest and the best treatment that’s available. That you will be cared for as well as you can possibly be, and that everything is documented. You know all the options that you have staying on the trial, giving consent, making sure you have all the information that you need to feel comfortable.
And Dana’s organization, hopefully, helping people out financially and logistically. There are ways to get into trials that at times are very successful. For me personally, I don’t know that I would be alive now if I had been on that trial, and that’s really my claim to fame, what can I say?
Andrew: And, Reina, I would say the same thing. Had I not been in a Phase 2 trial for chronic lymphocytic leukemia in 2000, I wouldn’t be around to have had retreatment last year, which has work quite well; 17-year remission. And I wouldn’t have been able to do this, and really have a purpose in life. So, I’m very grateful for being in the trial.
Dana, I can’t tell you – we were talking about gratitude, for you; came up in your family. You saw the gap for, not so much your family, but so many other families. The issues, financial issues, and you’ve been very philanthropic and, obviously, trying to have leadership in getting at some of these – we have a very imperfect system right now, so we have a long way to go. But for our viewers, if you’re living with cancer now, if your loved one is living with cancer, there are resources, people like Lazarex, people have been through it, like Reina.
We’re gonna give you this downloadable guide. And you’re gonna connect with these resources. Don’t… Put your pride away. Dana said it so well. There’s a very high likelihood we’re gonna be affected by cancer in our families, and there is help to navigate what’s kinda complicated right now, but is doable and can offer you the chance of doing better. Dana, did I say it right?
Dana: You did. You did. You did a great job, Andrew. Thanks.
Andrew: Okay. Well, thank you. And thanks to the Lazarex Cancer Foundation and, really, all you’re doing. And let’s hope that we can improve this process, increase participation, and have so many of these companies and the government that are trying to get scientific answers. We participate as respected patient investors. And we do better well. Reina, any final words from you with your 50th wedding anniversary coming up?
Reina: I’m very grateful. I’m very grateful to be here. I’m grateful for all the clinical trials, all the physicians who have taken care of me and who listened to all my concerns and fears. And I am super-duper grateful to my husband who has supported me, helped me, been there, been my caregiver, and washed the food for me when I had the transplant, and really, all the people who have been on the journey with me. So, if you are considering a clinical trial, if there is one that you might be eligible for, give it some thought. It’s a really important choice for you to make.
Andrew: Reina, thank you so much, all the best. Happy anniversary, early. Dana, best to you. Dana Dornsife, joining us from the Lazarex Cancer Foundation in the San Francisco Bay area. Dana, good health to your family, and thank you for all you do. Thanks for being with us, Dana.
Dana: Thank you.
Andrew: And, Reina, all the best, and thank you for those great words of wisdom. And we’ll meet in person sometime and I’ll give you a big hug, okay?
Reina: I hope so. I hope so. You take care of yourself, Andrew. Thank you so much.
Andrew: Thank you for joining us for this Patient Empowerment Network program Clinical Trials Mythbusters. We hope to do more. I wanna thank the companies that have helped provide funding for it; Abbvie Incorporated, Astellas, Celgene, and Novartis, for their support.
Thank you for joining us. I’m Andrew Schorr from Patient Power down near San Diego. Remember, knowledge can be the best medicine of all.
https://powerfulpatients.org/pen/wp-content/uploads/Clinical-Trial-MythBusters.png600600Kara Rayburnhttps://www.powerfulpatients.org/pen/wp-content/uploads/New-Logo-300x126.pngKara Rayburn2018-09-18 17:13:312020-01-08 12:30:24Clinical Trial MythBusters: Actionable Advice for Knocking Down Obstacles to Trial Participation