If each of us humans is a snowflake, unique in our genomic makeup, where’s my snowflake medicine? I asked that question from the platform at the ePharma Summit in New York in 2013, and have yet to get an answer. The challenge for the bioscience industry is, I believe, the classic randomized clinical trial. That design goes through four phases:
- Phase 1: a small group of people are given the drug under study evaluate its safety, determine a safe dosage range, and identify side effects
- Phase 2: a larger group is given the drug to evaluate its efficacy and safety in a larger population
- Phase 3: large groups – plural – of people are given the drug to confirm its effectiveness, monitor side effects, compare it to other commonly-used treatments, and collect information that will allow the drug /treatment to be used safely
- Phase 4: the drug is marketed while study continues to assess long-term effects and efficacy
Of course, before they even get to Phase 1, there have to be both the idea for the new treatment, and animal studies to determine what the substance or compound under study might do to a mouse or a monkey.
Science isn’t easy. The phrase “trial and error” came out of science labs, with many trials running up against the error wall by Phase 2. Since bioscience companies can sink about $1 billion-with-a-B into getting just one drug to market, it seems that the traditional clinical trial has turned into a pathway to NOT making scientific discoveries that can benefit humankind.
Then there’s the whole “who’s in charge here?” question. Clinical trials are now a global effort, with US and European pharma companies testing new treatments in Latin America, Russia, and China to gain traction in those emerging markets while simultaneously developing me-too drugs for their domestic markets. So, who’s in charge, the US Food and Drug Administration (FDA)? The European Medicines Agency (EMEA)? A player to be named later? The answer to the question seems to be “all of the above,” which adds to the complexity of the clinical trial process.
As digital technology has made data easier to collect and share, it would seem that clinical trials would be a great place to start intersecting with the quantified-self movement. The shift to electronic health records, the widening adoption of all sorts of health tracking devices, and the rise of (relatively) cheap genomic sequencing should signal an ability to identify conditions, and populations, eager to participate in clinical investigations. But so far, it hasn’t.
What might challenge that stasis? In November 2013, three major pharma companies – Novartis, Pfizer, and Eli Lilly – announced via the White House’s website that they had joined together in a clinical open innovation effort. That page on the White House’s site is gone now – changes in Presidential administrations will do that – but here’s a direct quote from that announcement:
“In order to connect patients and researchers, Novartis, Pfizer and Eli Lilly and Company, are partnering in the U.S. to provide a new platform to improve access to information about clinical trials. The platform will enhance clinicaltrials.gov and will provide more detailed and patient-friendly information about the trials, including a machine readable ‘target health profile’ to improve the ability of healthcare software to match individual health profiles to applicable clinical trials. As part of the project, patients can search for trials using their own Blue Button data.”
Five years later, and we’re still stuck on the slow train when it comes to really reinventing the clinical trial.
I’m one of a growing group of people who think that the entire life-sciences process chain needs to be re-tooled for the 21st century. In my view, the best place to start that re-tool is at ground level, with the patients and clinicians who deal with challenging medical conditions daily. If a doctor has a number of patients who might benefit from some clinical study, why isn’t there an easy way to find a researcher looking into that condition? If a patient has an idea for a clinical investigation into his or her illness or condition, why can’t they find a researcher who’s interested in the same condition to team up and start a science project?
I can only hope that the regulatory agencies involved in life science oversight (hello, FDA!) can move beyond the aftermath of Thalidomide – for which epic disaster we’re still paying a price when it comes to the timeline for drug approval in the US – and toward a process of “all deliberate speed” that doesn’t forsake speed for deliberation. Both are necessary, neither should be more heavily weighted than the other.
We all can, and should, take part in scientific exploration into human life, and human health. Got an idea for a clinical trial? Share that idea in the patient communities you hang out in, and ask your tribe to help you bring that trial to life. To quote Arthur Ashe, “Start where you are. Use what you have. Do what you can.”
We’ve got to start somewhere, right?
Casey Quinlan covered her share of medical stories as a TV news field producer, and used healthcare as part of her observational comedy set as a standup comic. So when she got a breast cancer diagnosis five days before Christmas in 2007, she used her research, communication, and comedy skills to navigate treatment, and wrote “Cancer for Christmas: Making the Most of a Daunting Gift” about managing medical care, and the importance of health literate self-advocacy. In addition to her ongoing work as a journalist, she’s a popular speaker and thought leader on healthcare system transformation from the ground up.
This blog post was originally published on Trial Mix, Science 37’s blog, and is reposted here with permission. Learn more about Science 37’s approach to clinical research or view the original post here.
Patient-centered research studies — which reduce the burden of participation — allow patients to reap the benefits by working smarter not harder.
As a scientist, I see the value in clinical research, but my time spent as a cancer patient showed me that priorities realign in times of health crisis. I cared a little less about the scientific value of what I was doing, and more about taking care of myself and spending time with my family. I know that clinical research studies do require extra effort from the participants, but patient-centered studies — those that are designed with the patient in mind — can let the patient work smarter, not harder, while still reaping the benefits.
Clinical Research Requires Some Extra Effort
I have lots of wonderful friends, many of whom have participated in clinical research studies, so I asked them to help me understand some of the hardest parts of their experiences. In addition to hearing about the experiences my friends had as adults, I also got some insight from a friend who’d been in a clinical trial as a child, and another whose son is in a food allergy research study now, giving me little peek at how clinical research impacts kids. Aside from the annoying paperwork like consent forms and surveys, here are the top three hurdles they mentioned:
- Time: It’s precious and finite, so all those minutes and hours spent in transit, in waiting rooms, and in exam rooms prevent clinical research participants from going about their normal routine.
- Money: While there should be no additional out-of-pocket cost for medical care to participate in clinical research, there is still a potential financial cost. Several friends talked about the need to pay for parking, miss work, or find childcare for each additional trip.
- Extra procedures: Shots, additional medications, or daily trips to the nurse for kids who take study drugs during the school day complicate a patient’s routine, and extra scans and invasive examinations really take a toll on a person. Beyond that, of course, no one wants to go through the pain of extra biopsies or the anxiety that comes with additional scans.
Patient-Centered Study Design Can Ease the Burdens of Participation
A more patient-centered study design could remove or reduce some of these hurdles. For example, providing extra assistance like patient navigation to connect patients with resources to provide transportation, childcare, or emotional support could reduce the burden. But since closer monitoring helps the investigators learn more from the research, many of these hurdles can’t be completely eliminated.
More Than Altruism: Clinical Research Benefits Participants
While some people look at all those barriers and decide (totally justifiably) that in their circumstances, the burdens of participation are too much for their family, many of the people I spoke with who had taken part in clinical research told me the extra work was so very worth it.
One friend wrote: “Facing that needle every week and working up the nerve to jab it deep into my thigh was the hard part for me…I still wouldn’t trade the experience for anything. That study gave me my life back.”
From another: “(There were) ups and downs, but I’d do them again. I’ve had substantial shrinkage of my tumor.”
A third shared: “The extra biopsies were painful and scary. But I felt like the doctors were watching me extra so it was worth it.”
My friends could see that not only did they contribute to some particularly awesome science, but each of them were able to reap benefits in their own lifetime — either because a pill could replace the need for a weekly self-injection, the drugs actually slowed their disease, or they knew they were being monitored so much more closely than a patient under standard care.
Working Smarter, Not Harder Improves the Patient Experience
While I’m quick to tell you that the increased monitoring a patient receives from all those doctor visits, blood draws, and surveys is vital to the clinical research process, it would be disingenuous to imply that the system can’t be improved.
Investigators need to design each study to obtain the most valuable data and draw valid conclusions. By involving patients in the design process to make the studies truly patient-centric, the research protocols can be written to ensure each study respects the patient experience and reduces the negative impact on a patient’s life. The Science 37 team partners closely with patients and investigators to ensure that trials are designed thoughtfully to minimize the impact on patients and their families, making it easier for them to benefit from awesome science.
We know it’s worth it to participate in clinical research, but we still want to make it less work for the patients involved.
About the Author
Jamie Holloway is a both a scientist and a survivor, earning her PhD in tumor biology from Georgetown University — where she spent long hours researching breast cancer — a few years before her own breast cancer diagnosis. Now living with no evidence of disease after treatment for early stage triple negative breast cancer, she bridges the gap between scientists and researchers as a Clinical Research Advocate for Science 37, and as the Patient Advocate for the Metastatic Breast Cancer Project at the Broad Institute. She works with researchers as part of the Georgetown Breast Cancer Advocates and writes about her personal experience with cancer on her blog Run Lipstick Chemo, and as a contributor to the Cure Magazine community. A wife, mother, runner, and lipstick addict, Jamie shares her story from the perspective of both a patient and a scientist.
She was 25 weeks pregnant and felt like she had the flu. It was early July 2015 and she made her way to her obstetrician’s office because the antibiotics she’d taken over the Fourth of July holiday hadn’t made her feel better. A couple hours later Elizabeth Carswell was in the hospital being diagnosed with Acute Myeloid Leukemia. She was 33 years old.
“Obviously, the scariest part was the first two months when I was still pregnant,” says Carswell, known as Liz to her friends. She was immediately transferred to Jackson Memorial Hospital in Miami and began chemotherapy. “It was a very tough decision to be treated, but I wanted to meet my baby.”
The first two rounds of chemo were unsuccessful and her doctors wanted her to undergo a more aggressive treatment. That meant it was time to deliver the baby. Liz was 32 weeks along and the surgery was extremely risky. Her blood counts and platelets were considered too low for surgery, but despite it all, baby Hudson was born weighing in at 4 pounds. Liz continued her treatment in the hospital while Hudson was admitted to the NICU for five weeks.
When Hudson was released from the hospital, Liz was not. Her boyfriend, Hudson’s father, Frankie Lightfoot had to return home to work, so Liz’s father and her sister moved to Miami, rented an apartment near the hospital and cared for the baby. Liz was never alone. Her father and sister alternated caring for the baby and spending the night in the hospital with her. Her friends showered her with gifts for the baby and travelled to her bedside to decorate her hospital room. Meanwhile, her doctors were trying to ready Liz for a bone marrow transplant, but could never get her levels where they needed to be and eventually her Jackson Memorial team told her it was time to go home and spend whatever time she had left with her baby.
That’s when a friend connected Liz with the Leukemia and Lymphoma Society which led her to a clinical trial at the City of Hope Cancer Research Hospital in Duarte, California under the care of Stephen J. Forman, M.D., F.A.C.P. She went the next day. The trial involved very aggressive full-body radiation and a stem cell transplant. Her brother, a perfect 10 match, was her donor.
The transplant put her into remission. It was November 2015. Then, in August of 2016, she found some lumps in her breast. The leukemia had returned in mass form. More radiation and seven more rounds of chemo later she is technically in remission. But, due to a gene mutation that makes her highly likely to relapse, Liz continues with regular rounds of chemo for maintenance.
“My doctor expects recurrence,” she says. That’s why she will undergo another stem cell transplant within the next couple months. This time, from an unrelated donor so that a new immune system can be introduced. She will spend another six weeks in the hospital, but she believes this is what she has to do. “I know it’s needed,” she said. “I trust it’s needed. I trust my doctor. He’s pretty amazing.”
Liz is undaunted by what lies ahead. “There are lots of ups and downs, but a positive attitude is a must if you’re going to survive,” she says. Though she credits Hudson, now two, as her main motivation. Early on she felt it was Hudson growing inside her who was keeping her alive and now being his mother keeps her going. “I’m not scared to go through it again,” she says of her upcoming transplant. “I’m excited to do it again. I’ll do anything that will give me a chance to watch Hudson grow up.”
Liz has kept a record of her journey on her Facebook page, Prayers for Elizabeth. She continues to keep her page updated and in addition to helping her, she hopes that sharing her story can help others. You can keep up with Liz here.
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.
When farmers try to manage insects in their crops, they figure out what dose of pesticide yields the best results. They do this based on understanding which bugs are the most harmful to their crops and what level of poison best combats them. This analogy may send shivers up the spines of organic eaters out there, but it does have relevance to the way chemotherapy is used in treating cancer.
It turns out that not all cancer cells (i.e. insects in the crops) are equally harmful within a tumor. Some are much more aggressive. When a broad-based chemo is used to kill all the cancer cells, it often leaves behind the most resistant ones, allowing them an opportunity to take over the tumor.
A quick look at evolution explains this phenomenon. Dr. Robert Gatenby, chair of radiology and co-director of the cancer biology and evolution program at Moffitt Cancer Center and Research Institute, says “We tend to think of cancers as a competition between the tumor and the host, but at the level of the cancer cell, cancer cells are mostly competing with each other,” he says. Competition within a tumor reflects a basic evolutionary principle, one which cancer cells utilize as well as normal cells.
Given this theory, Gatenby and his team wondered about the perfect level of chemotherapy that would repress the more aggressive cells, rather than encourage them to overtake the less aggressive cells. In the experiment, they took cells from two different breast cancers and grew them in mice. The mice were then treated to two separate chemo protocols. Group One mice were given lower-dose chemo and then skipped sessions if their tumors shrunk. Group Two mice were given continuous but gradually lower doses of chemo.
The researchers were surprised that 80% of the Group Two mice showed better response in reducing cancer growth. Some of the tumors disappeared completely.
This research is in its early stages and requires quite a bit of personalized chemotherapy analysis. One fly in the ointment – patients have to overcome the mindset that cancer treatment kills all cancer cells. They have to be content with the knowledge that not all cancer cells need to leave the body in order for a treatment to be successful.
In other words, leave the ladybugs and kill the locusts.
Interview With Dr. Michael Thompson (@MTMDPhD), Medical Director, Early Phase Cancer Research Program, University of Wisconsin
9 1/2-year CLL patient Carol Preston interviews Dr. Michael Thompson about some of the barriers to clinical trial accrual and how healthcare professionals might overcome them. Today, there is only about 3-5% of adult patients enrolled in clinical trials versus the 60% in pediatrics. Why is that? Watch the video below to find out.
As 2015 comes to an end, we would like to take a moment to highlight a few of our most popular posts and to thank the people who contributed to the popularity of these posts. We cannot thank the authors enough that have contributed, such as Marie Ennis-O’Connor, Tori Tomalia, Cindy Chmielewski, Dr. Michael Thompson, Edward Leigh, and Deana Hendrickson. Your efforts to Patient Empowerment Network are greatly appreciated.
PEN would also like to thank our sponsor AstraZeneca and our partner LUNGevity for their support in creating these posts.
Most Popular Posts of 2015
Virtual patient communities offer support and information to cancer patients.
Tori Tomalia, a lung cancer patient explains the stigma of lung cancer, and that it doesn’t happen to only those who smoke.
When applied to clinical trials, wearable technology is a potentially powerful research tool to gather clinical data in real-time and provides remote patient monitoring.
Clinical research is changing. No longer the sole preserve of clinicians and researchers, the Internet and new digital technologies are reinventing the way in which patients take part in the clinical trials process.
Carol Preston talks to myeloma patient Cindy Chmielewski about her role in advocating for clinical trials. Cindy explains how her cancer journey took her to self-advocacy and advocating for others in her community through social media.
Dr. Michael Thompson discusses with Carol Preston the reasons behind some patients apprehensiveness towards clinical trials.
What can you do in support of Lung Cancer Awareness Month
LVNG With is a community for people living with a lung cancer diagnosis. These people share their story and help inspire others like them.
Edward Leigh, Founder and Director of The Center for Healthcare Communication, shares his tips for creating a “medical résumé” to ensure great healthcare experiences.
In the Empowering Lung Cancer Patients Town Meeting, Deana Hendrickson talks about how important it is to connect with other patients and advocates online.
Also, be sure to check out
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The Clinical Trial Toolkit is a resource to help you find clinical trials and financial assistance. You can view our Patients Helping Patients Blog for various aspects and perspectives of clinical trials. Also, don’t miss our Videos of patients, caregivers, and healthcare professionals!
Interview With Dr. Michael Thompson (@MTMDPhD), Medical Director, Early Phase Cancer Research Program, University of Wisconsin
Carol Preston’s interview with Dr. Michael Thompson discusses issues related to clinical trial design. The obstacles to good patient-centric clinical trial design are numerous. The informed consent is a text-only legal-type document containing difficult to understand verbiage. Instead, it should be more patient-friendly, with images, graphics and easy-to-understand instructions.
Patients are becoming more empowered and more aware of the issues and are speaking out and willing to participate to make clinical trial design more patient-friendly and effective. More patients are aware of trials and want to educate and inform others. Clinical trial design and informed consent need to be updated and made more efficient so that more patients will be able to easily consider clinical trial enrollment.
Check out the full conversation and the rest of Dr. Thompson’s ideas in the video below:
Oftentimes when we hear the word “cancer,” we hear nothing else. Our brains stop processing information. We think we’re going to die, that there is little or no time to weigh options and/or get our affairs in order.
Fortunately, that last part the vast majority of the time isn’t true. We DON’T have to rush into anything. We DO have time to weigh our treatment options. And for many patients, those options can include enrolling in a clinical trial.
What is a clinical trial?
There are several types of trials
- Treatment: to test a new drug or approach to surgery
- Prevention: to test new approaches with medicine, vitamins, minerals to help lower the risk of developing certain cancers
- Screening: Best ways to detect cancer early
- Quality of Life: Explore ways to comfort, quality of life for patients
Why participate in a clinical trial?
Through clinical trials, doctors and researchers find better ways to prevent, diagnose and treat cancer. Patients can benefit by receiving cutting-edge care or emerging medications. Rarely are placebos, or fake medications, used in clinical trials. Patients for the most part are being given the current standard of care or the new treatment, to determine the following:
- Phase I: Is the new treatment safe?
- Phase II: Does the new treatment work?
- Phase III: Does the new treatment work better than the current one?
Is a clinical trial right for me?
There are risks and benefits. A good place to start making an assessment is with your physician. Often, we’ll hear about a clinical trial from our doctor. However, you don’t have to have your doc’s OK to enroll in a trial, and it’s key to determine which trial is right for you.
While there is no one source to learn about all cancer clinical trials, you can break it down into clinical trial lists and matching services.
The National Cancer Institute (NCI): 1-800-4-CANCER (422-6237 NCI sponsors most government-funded trials. You can search by the type and stage of cancer, by the type of study (treatment or prevention) or by zip code.
The National Institutes of Health (NIH): NIH database is larger than NCI but not all trials are cancer studies
CenterWatch: CenterWatch provides a list both of industry-sponsored and government-funded clinical trials for cancer and other diseases.
Private companies: Pharma or biotech companies may list studies they are sponsoring, either on their websites or through a toll-free number. You can search a company with the words ‘clinical trials’ in the search. If that company is conducting trials, it will appear in your search. For example:
You get the idea.
As for those clinical trial matching services, each one works differently. Some may charge a fee to the trial sponsor. It could impact the way studies are ranked or presented to you. We suggest you start with the free sites. Sources include:
The American Cancer Society Clinical Trials Matching Service: 1-800-303-5691 ASC works with a company called Eviti to connect patients with trials. It’s free. It’s confidential. It’s reliable. The lists are updated daily, and it allows patients to contact health care providers running the studies. The website also explains how to determine if you are eligible for a trial, if a trial is right for you.
EmergingMed: 1-877—601-8601 Also free, confidential and reliable for cancer patients seeking a trial.
Various organizations have partnered with EmergingMed and offer widgets that link to the EmergingMed trial finder. For instance, both the sites below link the viewer to the EmergingMed clinical trial finder:
There is a wealth of information here. But you don’t have to become a Medical Doctor to digest and evaluate it. Start with your physician. If he or she doesn’t have enough information, choose a website or two on this list to navigate for information about trials. That’s what I’ll be doing should the day come that I need additional treatment. We are our best advocates about what’s right for us and when.
From our partner, Treatment Diaries, this is a diary entry of a melanoma patient who participated in a clinical trial.
Diagnosis: Metastatic Melanoma in 32 year old female.
I had a mole removed on my back, turned out to be melanoma stage III. Since that mole I’ve had 5 more removed all positive. I’ve had surgery on the first one and then lymph nodes taken out because they found microscopic cells. September 2014 is when they diagnosed it as metastatic and inoperable.
So a clinical trial is a chance for me to make a difference and potentially beat the beast. I have had two
immunotherapy drugs Interferon and now currently on Yervoy. PET scan and MRI are clean for now. I have a lot of side effects from the Yervoy which I’m on right now. Dr put me on steroids to reverse those side effects. I get rashes, colitis, headaches, fatigue, and heartburn from yervoy and muscle weakness; retain water, mood swings, anxiety, emotional from steroids.
I have a four month old baby and recently got married and moved states. A lot of changes in my life the last year and coping the best I can. I have serious moments of a break down but try to hold it together for the sake of my precious baby. Currently I am trying to take lower dosages of my steroids so I can go in for more scans. Dr says we are done with Yervoy because of side effects. But there are other treatments we can look into. That’s my story and please feel free to contact me anytime! I feel the more support I have the better my days turn out! I’m here if you want to talk I’ll listen. Sometimes that’s all I need is to have a shoulder!
I go in on Thursday for a treatment called Infliximab.. It’s supposed to stop these side effects from the Yervoy, such as muscle aches and colitis. I’m really nervous because the shot itself has a lot of side effects from what I’ve read. Dr says I’ll only need one shot and it should help me get off the steroids which are a must! I’ve been on steroids since end of August… Right now I’m on 25 mg a day but my highest has been 100 mg. I don’t want to do the shot but I’m at a point it might be my only option. I want to feel normal again with no pills. Will that day ever come, who knows? I pop pain pills like candy and steroids like a race horse.
Starting to think I need a therapist to talk to because mentally I’m not handling things well. My husband says he believes everything will work out, but he’s not giving me the emotional support I need. I don’t think he understands… Takes care of the medical bills but as far as emotional support I’m not sure he can be there for me because he doesn’t understand my fear. Fear of limited days and wanting to enjoy life more. I feel I do the same thing over and over every day… I have a 5 month old so we are stuck in the house a lot and hubby just goes to work and comes home… Same routine everyday… I want to feel alive and excited! I want a date night or to feel sexy and I need affection, ok I’m done rambling I’ll keep an update on how the shot treats me. As far as Yervoy I’m done with it the doctor won’t let me do anymore. Too many side effects! After I get off the roids I’ll have another pet scan. More later…
Real patient experiences shared privately at www.TreatmentDiaries.com. Read more, share if you like or join in the conversation. Making sure you feel less alone navigating a cancer diagnosis is important. Connecting you to those who can relate and provide support is what we do.
From our partner, Treatment Diaries, this is a diary entry of a melanoma patient first diagnosed in 2012.
Diagnosis – Malignant Melanoma 3B
Male – 57 years old
First diagnosed Feb 2012
5 operations in a 1.5 year period upgraded to stage 4 Metastatic Melanoma
BRAF POS, as of Aug 5,2013.
Fully Disabled since Oct 30, 2013
Currently on Zelboraf Clinical Trial
Been NED since December 31, 2013
My recent MRI followed by a pet scan were both clear once again! I am still on Zelboraf at 1 pill a day. I have begun using CBD oil twice daily along with all the other things I do this to help me stay healthier and to keep the beast at bay. Come the end of October, I will have been off work for 1 year, on disability/ssi for income. I still make it to the gym 1-3 times a week and still work out with friends. On top of that, I take rides on my motorcycle and visit family. I still have fatigue and I sometimes nap a lot still as a result of the side effects of Zelboraf but I no longer have any evidence of disease.
Pursuing a clinical trial was scary at first but really my only option. My only option turned out to be the best thing for me and for my family. I hope my story will inspire others to keep an open mind and to hope for the impossible. Hopefully this will help others see that some of what the doctors do does help for the better, I don’t know? Well we all know this is the one club no one wanted to join, but we are in this together and tomorrow’s a new day!!!!
Still thriving as of July 22, 2015.
This abbreviated story of a melanoma cancer diagnosis and clinical trial experience which spans multiple diaries and is available on www.TreatmentDiaries.com. Sharing your story is empowering. Join us for completely private and anonymous exchanges about your health.
Please check back soon as we work to build more resources.