Confused About Immunotherapy and Its Side Effects? You Aren’t Alone

“You don’t look like you have cancer.”

More than one patient undergoing immunotherapy to treat cancer has reported hearing statements like that. Immunotherapy is one of the recent advances in cancer treatment that belie the stereotypes about the effects of cancer treatment. 

The side effects of immunotherapy are different from those associated with chemotherapy and radiation. However, that does not mean immunotherapy does not have side effects. Patients and care partners need to be aware of these potential side effects and to be vigilant in addressing them with their oncologists because they can signal more serious complications if left untreated.

What is Immunotherapy?

Despite the increase of immunotherapy treatment options in recent years and considerable media attention paid to advancements in this field, there remains confusion about immunotherapy and its side effects. Many cancer patients are unaware of whether immunotherapy treatments are available for their specific diagnosis. Others don’t know that genetic profiling of their tumors is usually required to determine if immunotherapy is an option and not all treatment centers routinely conduct genetic profiles of tumors. A  survey by The Cancer Support Community found that the majority of patients who received immunotherapy knew little to nothing about it prior to treatment and were unfamiliar with what to expect.

Immunotherapy works by manipulating the patient’s immune system to attack cancer cells. It is perceived as gentler and more natural than chemotherapy and radiation, without the same destructive effect on the body’s healthy tissues.  This, combined with a lack of prior understanding of immunotherapy, can lead patients and care partners ill-prepared for possible side effects.

Furthermore, immunotherapy is a category of therapies, not a single type of treatment. There are a variety of immunotherapy drugs, most of which are administered via infusion.  Side effects will vary by drug, the cancer and its location, treatment dose, and the patient’s overall health.

The following are the most common types of immunotherapy.

  • Checkpoint inhibitors use drugs to block proteins in the patient’s immune system that would otherwise restrain the immune system, often referred to as taking the “brakes” off the immune system.
  • CAR-T therapy modifies the patient’s T-cells in a lab to enhance their ability to bind to cancer cells and attack and kill them.
  • Oncolytic virus therapy uses genetically modified viruses to kill cancer cells.
  • Another therapy uses cytokines (small proteins that carry messages between cells) to stimulate the immune cells to attack cancer.

Immunotherapy can be part of combination therapy. It might be combined with chemotherapy. It might be used to shrink a tumor that is then surgically removed.  Or multiple immunotherapy drugs might be used simultaneously.

What Are The Side Effects?

With immunotherapies, side effects typically occur when the immune system gets too revved up from the treatment. The most common side effects for immunotherapy treatments are fatigue, headache, and fever with flu-like symptoms. Some people also experience general inflammation often in the form of a rash. Many melanoma patients report blotchy skin discoloration, called vitiligo, during treatment. These milder side effects can usually be managed with over-the-counter remedies and adjustments to daily activities.

For checkpoint inhibitors, the fastest growing segment of immunotherapy treatments, mild side effects occur in 30% – 50% of patients. Serious side effects typically occur in less than 5% of patients. (See “Understanding Immunotherapy Side Effects” from the National Comprehensive Cancer Network and the American Society of Clinical Oncology.)

Less common side effects are blisters, joint pain, thyroid inflammation, and colitis (inflamed colon resulting in diarrhea with cramping). Some patients who receive CAR T-cell therapy develop a condition known as cytokine release syndrome, which causes fever, elevated heart rate, low blood pressure, and rash. 

In rare cases, immunotherapy has resulted in lung inflammation, hepatitis, inflammation of the pituitary, and detrimental effects on the nervous and endocrine systems. In most cases, the conditions clear up when treatment ends.  However, there have been outcomes in which immunotherapy caused diabetes or tuberculosis.

“Overall there are fewer side effects [with immunotherapy],” explained Dr. Justin Gainor, a lung and esophageal cancer specialist at Mass General during an Immunotherapy Patient Summit hosted by the Cancer Research Institute. “But the immune system can affect anything from the top of the head down to the toes. Any organ has the potential to be affected.”

As the application of immunotherapy has expanded, so has our understanding of the potential side effects. Like most medical treatments, how one person responds to immunotherapy can be different from another even when the cancer diagnosis and drug therapy are the same.

The essential thing patients and care partners need to know about side effects is they should always be reported to their oncologist or nurse oncologist.

Why Patients Should Talk to Their Provider About Immunotherapy Side Effects

Because immunotherapy has created newer therapy options, there isn’t the volume of experiences as with older treatments. The infinite number of variables that patients provide once a treatment moves beyond clinical trials and into the general patient population generate more diverse outcomes.  And, as most therapies are less than 10 years old, there hasn’t been an opportunity to study the long-term effect of these therapies. This is why oncologists advise patients and their caregivers to be extra vigilant in noting any changes experienced during and after treatment.

Many side effects are easy to treat but medical providers want patients to be forthcoming in discussing any and all side effects. This is in part to improve understanding of side effects, but also because a mild cough or a case of diarrhea might be harbingers of a more systemic issue that will grow worse if left untreated.

Patients should not be hesitant to discuss side effects because they fear they will be taken off immunotherapy.  Sometimes a pause in treatment might be necessary, but the earlier the oncologist is made aware of a side effect, the less likely that will be necessary.

In addition, patients undergoing immunotherapy should always take the name(s) of their immunotherapy drugs and the name of their oncologist when seeing medical professionals outside of their cancer treatment team. This is especially important when visiting the ER.  Because immunotherapy drugs are newer and highly targeted to certain cancers, many medical professionals remain unfamiliar with drug interactions and treating related side effects.

Immunotherapy On The Rise

Immunotherapy treatments have resulted in reports of remission in cases that would’ve been deemed hopeless just five or 10 years ago.  The Federal Drug Administration (FDA) has approved various immunotherapy treatments for melanoma, lung cancer, head and neck cancer, bladder cancer, cervical cancer, liver cancer, stomach cancer, lymphoma, breast cancer, and most recently bladder cancer.  (Here is a list of  immunotherapies by cancer type from the Cancer Research Institute.)

“It’s revolutionized how we treat our patients,” says Dr. Gainor of Mass General about immunotherapy’s impact on lung and esophageal cancer.

Advances in immunotherapy research and trials continue to generate optimism and excitement. A clinical study in Houston is looking at using immunotherapy to prevent a recurrence. Researchers in Britain recently announced a discovery that might lead to advances in immunotherapy treatments to a much broader array of cancers.

While there is excitement around the field of immunotherapy and it has resulted in unprecedented success in treating some previously hard-to-treat cancers, it remains an option for a minority of cancer diagnoses.  It works best on solid tumors with more mutations, often referred to as having a high-mutational load or microsatellite instability (MSI) high. And it is not universally successful for every patient.

With hundreds of clinical trials involving immunotherapy alone or in combination with other therapies, it is certain more treatment options are on the horizon. As more therapies are developed and more patients with a greater variety of conditions undergo immunotherapy, we will also increase our understanding of potential side effects.

Side effects should not dissuade patients and care partners from considering immunotherapy if it is available or from advocating for genetic tests to deteimine if it is an option. Many patients undergoing immunotherapy have previously undergone chemotherapy and report that the side effects are fewer and milder by comparison.  The important thing is that patients and their partners know what to expect and communicate with their treatment team.

If the next 10 years in immunotherapy research and development are anything link eth elast 10, we can expect more exciting advancements in the battle against cancer. For more perspective on what’s ahead for immunotherapy see the Cancer Research Institute’s article: Cancer Immunotherapy in 2020 and Beyond.

Understanding Clinical Trials: A Jargon Buster Guide

When it comes to cancer treatment you or a loved one may be considering participating in a clinical trial as a treatment option.  Clinical trials are designed to evaluate the safety and effectiveness of a treatment. They may involve researchers administering drugs, taking blood or tissue samples, or checking the progress of patients as they take a treatment according to a study’s protocol.

Learning about clinical trials can be a steep learning curve – not least because the process comes with a lot of new terms, acronyms and jargon.  To help you, I’ve put together this list of the most common terms you will find when you are researching clinical trial information. This is not an exhaustive list but it is a helpful starting point. At the end of this article you will see links to find more information.

Adverse Effects (AE)

Also called Adverse Events, or Adverse Drug Reaction, AEs are any harmful event experienced by a person while they are having a drug or any other treatment or intervention. In clinical trials, researchers must always report adverse events, regardless of whether or not the event is suspected to be related to or caused by the drug, treatment or intervention.

Arm

Subsection of people within a study who have a particular intervention.

Bias

Bias is an error that distorts the objectivity of a study. It can arise if a researcher doesn’t adhere to rigorous standards in designing the study, selecting the subjects, administering the treatments, analysing the data, or reporting and interpreting the study results. It can also result from circumstances beyond a researcher’s control, as when there is an uneven distribution of some characteristic between groups as a result of randomization.

Blinding

Blinding is a method of controlling for bias in a study by ensuring that those involved are unable to tell if they are in an intervention or control group so they cannot influence the results. In a single-blind study, patients do not know whether they are receiving the active drug or a placebo. In a double-blind study, neither the patients nor the persons administering the treatments know which patients are receiving the active drug. In a triple-blind study, the patients, clinicians/researchers and the persons evaluating the results do not know which treatment patients had. Whenever blinding is used, there will always be a method in which the treatment can be unblinded in the event that information is required for safety.

Comparator

When a treatment for a specific medical condition already exists, it would be unethical to do a randomized controlled trial that would require some participants to be given an ineffective substitute. In this case, new treatments are tested against the best existing treatment, (i.e. a comparator). The comparator can also be no intervention (for example, best supportive care).

Completed

A trial is considered completed when trial participants are no longer being examined or treated (i.e. no longer in follow-up); the database has been ‘locked’ and records have been archived.

Control

A group of people in a study who do not have the intervention or test being studied. Instead, they may have the standard intervention (sometimes called ‘usual care’) or a dummy intervention (placebo). The results for the control group are compared with those for a group having the intervention being tested. The aim is to check for any differences. The people in the control group should be as similar as possible to those in the intervention group, to make it as easy as possible to detect any effects due to the intervention.

Efficacy

How beneficial a treatment is under ideal conditions (for example, in a laboratory), compared with doing nothing or opting for another type of care. A drug passes efficacy trials if it is effective at the dose tested and against the illness for which it is prescribed.

Eligibility Criteria/ Inclusion and Exclusion Criteria

Eligibility criteria ensures patients enrolling in a clinical trial share similar characteristics (e.g. gender, age, medications, disease type and status) so that the results of the study are more likely due to the treatment received rather than other factors.

Follow-up

Observation over a period of time of participants enrolled in a trial to observe changes in health status.

Informed Consent

A process (by means of a written informed consent form) by which a participant voluntarily agrees to take part in a trial, having been informed of the possible benefits, risks and side effects associated with participating in the study.

Intervention

The treatment (e.g., a drug, surgical procedure, or diagnostic test) being researched. The intervention group consists of the study participants that have been randomly assigned to receive the treatment.

Investigator

A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted by a team of individuals at a trial site, the investigator is the responsible leader of the team and may be called the principal investigator (PI).

Multicentre Trial

A clinical trial conducted according to a single protocol but at more than one site, and therefore, carried out by more than one investigator.

Number needed to treat (NNT)

The average number of patients who need to receive the treatment or other intervention for one of them to get the positive outcome in the time specified.

Outcome Measures

The impact that a test, treatment, or other intervention has on a person, group or population.

Phase I, II, III and IV Studies

Once the safety of a new drug has been demonstrated in tests on animals, it goes through a multi-phase testing process to determine its safety and efficacy in treating human patients. If a drug shows success in one phase, the evaluation moves to the next phase

  • Phase 1 tests a drug on a very small number of healthy volunteers to establish overall safety, identify side effects, and determine the dose levels that are safe and tolerable for humans.
  • Phase II trials test a drug on a small number of people who have the condition the drug is designed to treat. These trials are done to establish what dose range is most effective, and to observe any safety concerns that might arise.
  • Phase III trials test a drug on a large number of people who have the condition the drug is designed to treat. Successful completion of Phase III is the point where the drug is considered ready to be marketed.
  • Phase IV trials can investigate uses of the drug for other conditions, on a broader patient base or for longer term use.

Placebo

A fake (or dummy) treatment given to patients in the control group of a clinical trial.  Placebos are indistinguishable from the actual treatment and used so that the subjects in the control group are unable to tell who is receiving the active drug or treatment. Using placebos prevents bias in judging the effects of the medical intervention being tested.

Population

A group of people with a common link, such as the same medical condition or living in the same area or sharing the same characteristics. The population for a clinical trial is all the people the test or treatment is designed to help.

Protocol

A plan or set of steps that defines how something will be done. Before carrying out a research study, for example, the research protocol sets out what question is to be answered and how information will be collected and analysed.

Randomized Controlled Trial (RCT)

A study in which a number of similar people are randomly assigned to 2 (or more) groups to test a specific drug, treatment or other intervention. One group has the intervention being tested; the other (the comparison or control group) has an alternative intervention, a placebo, or no intervention at all. Participants are assigned to different groups without taking any similarities or differences between them into account. For example, it could involve using a computer-generated random sequence. RCTs are considered the most unbiased way of assessing the outcome of an intervention because each individual has the same chance of having the intervention.

Reliability

The ability to get the same or similar result each time a study is repeated with a different population or group.

Sample

People in a study recruited from part of the study’s target population. If they are recruited in an unbiased way, the results from the sample can be generalised to the target population as a whole.

Subjects

In clinical trials, the people selected to take part are called subjects. The term applies to both those participants receiving the treatment being investigated and to those receiving a placebo or alternate treatment.

Trial Site

The location where trial-related activities are conducted.


References

The Canadian Institutes of Health Research (CIHR)

TROG Cancer Research

ICH.org

NICE

Further Resources

American Society of Clinical Oncology’s Cancer.Net trials site

National Cancer Institute (NCI) Clinical Trials lists open and closed cancer clinical trials sponsored or supported by NCI. 

ClinicalTrials.gov database of privately and publicly funded clinical studies

CenterWatch Clinical Trials Listing

Cancer Clinical Trials Are For All Patients: Latest Research from the 2019 Quality Care Symposium

This podcast was originally published by Cancer.net on September 3, 2019, here.

 

In this episode, we’re going to discuss 2 studies on patient experiences with clinical trials that will be presented at ASCO’s 2019 Quality Care Symposium. This annual meeting brings together health care experts to share strategies for cancer care issues and integrate these methods into patient care.

Transcript:

[music]

ASCO: You’re listening to a podcast from Cancer.Net. This cancer information website is produced by the American Society of Clinical Oncology, known as ASCO, the world’s leading professional organization for doctors who care for people with cancer.

The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience, and conclusions. The mention of any product, service, organization, activity, or therapy should not be construed as an ASCO endorsement. Cancer research discussed in this podcast is ongoing, so the data described here may change as research progresses.

Monika Sharda: Hi, I’m Monika Sharda, an editor on the Cancer.Net team and your host for today’s podcast. In this episode, we’re going to discuss 2 studies on patient experiences with clinical trials that will be presented at ASCO’s 2019 Quality Care Symposium. This annual meeting brings together health care experts to share strategies for cancer care issues and integrate these methods into patient care. I have with me 2 oncology experts who will help us understand these studies and why they’re important. Our first guest, Dr. Merry-Jennifer Markham is a hematologist at the University of Florida in Gainesville. Welcome, Dr. Markham.

Dr. Markham: Hi, hi. Thanks for having me.

Monika Sharda: And we also have with us Dr. Neeraj Agarwal, who is a medical oncologist at the University of Utah’s Huntsman Cancer Institute. Thanks for being with us, Dr. Agarwal.

Dr. Agarwal: A pleasure. Thank you.

Monika Sharda: So before we delve into the studies, I want to make sure we explain what clinical trials mean for any listeners who may not be familiar with the term. Can you provide a brief explanation of what a clinical trial is and how they’re used in cancer care?

Dr. Agarwal: Yeah, of course. So if we look at the definition of National Cancer Institute, how the clinical trial is defined that is a type of research study that test how well new medical approaches work in our patients. And these studies test new methods of screening, prevention, diagnosis, or treatment of a disease. These are often called as prospective clinical studies, but I make it simple for my patients. I tell them that to me the definition of a clinical trial is how to get cutting edge technology, which can be a treatment or a device, to my patients 5 years before FDA approval of that drug or a device. How to expedite availability of those cutting-edge technology to my patients is the definition I use for clinical trials.

Monika Sharda: Thanks. That’s a great way to put it. So let’s start by discussing the study that comes out of Seattle, Washington where researchers looked at whether participating in a clinical trial helped people with metastatic non-small cell lung cancer live longer. Can you tell us a little bit about how the study was conducted, Dr. Agarwal?

Dr. Agarwal: Yes, and this study, as you mentioned, was conducted in Seattle Cancer Alliance consisting of University of Washington and Fred Hutchinson Cancer Research Center, both based in Seattle, Washington. What the researchers did, they looked back at the records of patients with non-small cell lung cancer or simply advanced lung cancer who were treated in their institutions between January 2007 and December 2015. And they included 371 patients.  One-third of those patients, almost 30% of patients were enrolled on 1 or more clinical trials. And other patients were not enrolled in the clinical trials. And they compared, basically, those patients. They looked at the survival of patients who were able to get on a clinical trial versus who did not. And very interestingly, patients who were enrolled on a clinical trial, their median survival was twice as much as those who did not get to enroll on a clinical trial. The overall survival in patients who were on clinical triasl who got to get treated on a clinical trial—at least one clinical trial—was 838 days compared to patients who did not go on a clinical trial who only lived for 454 days. This is even more interesting is because the researchers compared the patient’s disease characteristics, demographic characteristics, and they made sure that patients were evenly distributed from those characteristics. It’s not that patients who had more aggressive disease or who had a higher history or longer history of smoking, they got to be under control arm, which is that they did not get on the clinical trial. So patients in both groups were evenly matched for demographic and disease characteristics. So this basically tells me that if you get to enroll on a clinical trial, the overall survival is higher than if you do not.

Monika Sharda: And do we know why that might be? Why patients that were enrolled in clinical trials tended to live longer?

Dr. Agarwal: As I said, clinical trial allows me and my patients to have those technology or those drugs available to the clinic 5 years before FDA approval. And that’s the ballpark. It can be 7 years. It can be 10 years. It can be 3 years. But in general, 5 years is the mark I use with my patients. So if a patient is getting to be treated with a drug 5 years before that drug would be available by prescription, there is an advantage of time, because if we look at the median survival of this patient population, there is no way they could have just waited for that drug to get approved and be available by prescription in the clinic. So I think that’s a huge advantage, that they had access to a drug for their cancers which was not available to those patients who did not get to go on a clinical trial. I think that’s the number one, or the main advantage, why the survival is so much better in the patients who got to go on a clinical trial.

Monika Sharda: Right. And the other study focuses on clinical trial enrollment. So statistics show that less than 10% of people with cancer participate in clinical trials. For this  study, researchers surveyed 120 doctors and clinical trial research staff and also 150 cancer patients to try and find out why participation is so low. So Dr. Markham, can you tell us briefly what the researchers found?

Dr. Markham: Sure. I think 1 of the things that is striking is that the number of patients who enroll on trials is so low, the percentage. And we know the barriers to clinical trial enrollments do exist. What this study showed actually was that the perceptions of what these barriers are, really differed between the physicians and research staff and the patients. So clearly we didn’t have a great understanding of the barriers on each side.

I’ll give you just a couple of examples. In this study, patients more often than physicians or research staff believed that trials are only available and only for people whose cancer is considered hopeless. We know that’s not reality, but that’s a perception that panned out in the study. Also more patients, more so than physicians or staff, believe that clinical trials don’t help an individual patient. And we know that not to be true. And I think that former study is a really good example of that where in the prior study participating in a clinical trial actually did improve survival. And then a third example is physicians and research staff in the study, more so than patients, were more likely to believe that patients decline a clinical trial due to either language or cultural barriers or due to a lack of understanding about clinical trials.

Monika Sharda: Where do you think these perceptions arise from that people have about clinical trials, just going back to the couple of examples that you gave? For example, people thinking that clinical trials are only used when their disease is hopeless or that they don’t actually help the patients themselves. Where do you think those perceptions stem from?

Dr. Markham: It’s hard to know, but I think communication or lack of communication about trials, or lack of enough communication about clinical trials is really a large part of the problem. I think that clearly this is evidence that we oncologists and cancer researchers maybe haven’t done a great job or as good a job as we should be doing when it comes to educating our patients. I think this study demonstrates that we do have a lot of room to improve on the patient education piece.

Monika Sharda: Do you have any thoughts on some specific ways that people with cancer and their family can work together with doctors to communicate better about clinical trials?

Dr. Markham: I think the more education on the cancer or various topics that patients want to bring up in the exam room, the more sort of preparation work a patient and a caregiver can do in advance of the visit the better. Coming to an appointment with a list of questions about trials for example can really help to guide a conversation. I think that it’s also a good idea to bring somebody with you to an appointment and this holds true for other reasons, including listening in and having some extra set of ears there to hear important parts of a discussion about a cancer diagnosis or prognosis or treatment. But really helping to sort of prompt questions about clinical trials may be useful.

I think for doctors, a good way to open up this conversation is just with open-ended questions. Some of the things I like to ask my patients are, “What do you know about clinical trials?” or, “What would you like to know about clinical trials?” And this is really a good way for me as a physician to gauge the level of understanding of a trial at the outset. And I can gauge whether there’s any perceptions or misperceptions that I can help to clarify. And it’s a great launching pad for a discussion about clinical trials.

Monika Sharda: Dr. Agarwal, did you have anything to add about this study?

Dr. Agarwal: I think I agree with everything Dr. Markham just said. In my practice, I spend a significant amount of time when I see a patient for the first time who has come to establish care in my clinic, on just orienting them on clinical trials regardless of whether they are currently eligible for the trial or we have a trial for them or not. I just talk to them about the clinical trials. And that is a theme in my practice. Even a nurse practitioner and nurses, the more our patients hear about clinical trials, I think more amenable they will be or they are, in our experience, to accept enrollment on a clinical trial down the line. But as Dr. Markham said, it’s not only 1 doctor or 1 nurse or 1 nurse practitioner. I think it has to be a more holistic approach educating at different levels. All the websites as we discussed as we know of from Cancer.Net, NCI, ClinicalTrials.gov. All those websites have great information on clinical trial availability of a clinical trial for a given disease condition or given stage of a disease. By doing all of those our patients can be made aware of all those websites other than the orientation in the clinic. So I think this has to be a global approach and which ultimately will lead to increased awareness and increased participation of our patients on clinical trials.

Monika Sharda: Thanks. And I appreciate you sharing some resources with our listeners of where they can learn more about clinical trials so they can be prepared to have these conversations with their health care team. And just a quick note for our listeners, you can learn more about clinical trials on Cancer.Net by visiting cancer.net/clinicaltrials. And there’s also a couple of other resources that Dr. Agarwal mentioned. Dr. Markham, did you want to add any other resources?

Dr. Markham: Sure. So Cancer.Net is definitely a great resource. And it’s written in a way that is easily understandable. The other 2 that I would mention are the American Cancer Society’s website and the National Comprehensive Cancer Network or NCCN. And both of those have very good information about clinical trials in general. ClinicalTrials.gov, as Dr. Agarwal mentioned, also does and can be a very useful tool at finding a specific clinical trial for a specific condition.

Monika Sharda: Great. Well, thank you both for taking the time to distill these studies and the takeaways for people with cancer and their loved ones. Is there anything else that you would like to note about either of these studies or about clinical trials in general that we haven’t already touched on?

Dr. Markham: Yeah, I was going to say I think I would just add that I commend the researchers who did these studies and are getting their work published. I think it’s important that we improve access to clinical trials as much as possible. And these two studies help to work in that direction.

Dr. Agarwal: I agree, 100%.

Monika Sharda: Great. Well, thank you both again for your time.

ASCO: Thank you Dr. Agarwal and Dr. Markham.

You can find more research from recent scientific meetings at www.cancer.net. And if this podcast was useful, please take a minute to subscribe, rate, and review the show on Apple Podcasts or Google Play.

This Cancer.Net podcast is part of the ASCO Podcast Network, a collection of 9 programs covering a range of educational and scientific content offering insight into the world of cancer care. You can find all 9 shows, including this one, at podcast.asco.org.

Cancer.Net is supported by Conquer Cancer, the ASCO Foundation, which funds breakthrough research for every type of cancer, helping patients everywhere. To help fund Cancer.Net and programs like it, donate at conquer.org/support.

About the Chemo

This podcast was originally published by BBC You, Me & the Big C on March 15, 2018 here.

The #youmebigc team, Rachael, Deborah and Lauren give you their personal guide to all things chemo. They are joined by chemotherapy nurse Kate Lewis from The Royal Marsden who shares her advice and expertise.

Chemo Class with Madelieine Kuiper

This podcast was originally published by Kappa Kappa Cancer on February 13, 2019, here.

Why can’t I have sushi or manicures while on chemo? Erin’s lifeline for meds and frantic midnight emails during her treatment, her Nurse Practitioner Maddie Kuiper, visits the studio to walk us through a typical chemo session, how chemo meds vary and when you should/shouldn’t freak out and call your nurse.

Immunotherapy in the Elderly

This blog was originally published by Cancer Today by Emma Yasinki here.

Immune checkpoint inhibitors can be effective treatments for elderly people with some types of advanced cancer, but more information is needed on their risks and benefits in this group.

​Photo by graffoto8​ / iStock / Getty Images Plus

CHECKPOINT INHIBITORS, a type of immunotherapy drug, help spur the immune system to kill cancer cells. These drugs can be effective treatments for some patients who otherwise would have few options.

Beginning in 2011, with the approval by the U.S. Food and Drug Administration of the first checkpoint inhibitor, seven of these immunotherapy drugs have come onto the market for treatment of various cancer types.

Enthusiasm for these drugs is widespread, including among elderly patients with advanced cancer. Now, some frail elderly patients who might previously have opted out of chemotherapy are choosing immunotherapy in hopes of achieving a long-term response.

But data on immunotherapy side effects and outcomes are more limited in elderly people than in younger patients. Some doctors worry that all the excitement surrounding checkpoint inhibitors is preventing older patients from getting palliative and hospice care that could be more likely to improve their lives.

Rawad Elias, an oncologist at Hartford Hospital in Connecticut, studies immunotherapy in older patients and presented on the topic at the American Society of Clinical Oncology Annual Meeting in Chicago in June 2019. Cancer Today spoke with Elias about the benefits and risks of checkpoint inhibitors and how their availability may affect treatment decisions for older patients.

Q: Are there common misconceptions among patients and families about checkpoint inhibitors?
A: We’re very excited about [immunotherapy] because it’s an option now other than chemotherapy, [but] it doesn’t work in all cancers. Even in the cancer[s] that it works for, it doesn’t work in all patients. And most patients, in fact, do not respond to checkpoint inhibitors.

We often see patients who … ask us, “OK. How about immunotherapy?” And we’ll have to explain that, unfortunately, in your type of cancer, it doesn’t even work.

Q: What do we know about the efficacy of checkpoint inhibitors in older patients?
A: Unfortunately, older adults are underrepresented in clinical trials. Older adults constitute about 60% of cancer patients, and [in] the clinical trials of checkpoint inhibitors, they [made up] about 40% [of participants]. Also, patients who are enrolled on clinical trials are usually the … fit people with [few] medical complications. So we don’t really understand the clinical profile of these drugs in the real-world population.

We did some work in the past looking … if the efficacy of the checkpoint inhibitors is similar across age groups. We published that in the Journal for ImmunoTherapy of Cancer based on [an] age cutoff of 65. The efficacy of checkpoint inhibitors was considerable in younger and older adults. What we don’t know about, though, is what’s the impact of frailty on these medications? And does that make patients more prone to toxicity? Does it make the efficacy of the drug less?

Q: What are the special considerations older patients need to take into account when considering checkpoint inhibitor therapy?
A: What we don’t know about … is the impact of low-grade toxicity or any toxicity on older adults. We tend to call things like fatigue or a little bit of nausea “low-grade” toxicity, but we don’t know the impact of this low-grade toxicity on an 80-year-old person who already has trouble getting out of the house.

When it comes to older patients with an advanced cancer, this is a really critical thing to discuss: What’s your quality of life during this period of time, and what matters most to you as a person? The goal is not to go and treat the cancer. The goal is to treat you as a person. And it’s only you as a patient who gets to determine: What does that mean?

For example, [one of my patients], even though therapy could have been an option for her, she’s a frail older adult. We talked about [the fact that] the impact of treating her with immunotherapy would be potentially more fatigue and coming to the doctor’s office [more frequently]—coming in once every two weeks or once every four weeks … getting bloodwork, waiting in the waiting room to see the doctor and then getting the infusion, then going back home, then coming back again. So the question is: Does that make sense to you? My patient … decided that doesn’t make sense to her based on what we think … [immunotherapy] is going to achieve.

Q: Why are some people concerned that the increasing popularity of checkpoint inhibitors could hinder access to palliative and end-of-life care?
A: Unfortunately, when we’re treating cancer patients, we’re treating a very hard disease and even small things get us excited. In the hype or the excitement about checkpoint inhibitors, many may skip that conversation [about risks and alternatives like palliative care] and go straight to, “Let’s start you on checkpoint inhibitors and see what happens.” And what’s happening in most patients is that they do not respond, and we forget about palliative care which we know, for sure, makes people have a better quality of life, keeps them outside the hospital, keeps them at home. This is not to say older adults should not be treated, but to say that there are concerns about these drugs. They do not work for everyone.​ ​​

Emma Yasinski​ is a Florida-based freelance science and medical journalist.​

Focusing on Proton Therapy

This blog was originally published by Cancer Today by Sue Rochman here.

Proton therapy, an alternative to standard radiation therapy, is safe and effective. But evidence is lacking that it’s always a better option than standard radiation, and some insurers balk at the higher price tag.

Photo by ​​​​gorodenkoff​ / iStock / Getty Images Plus

IN AUGUST 2017, Ha​uli Sioux Warrior Gray noticed a lump in her left breast. Two months later, after having seen three different health care providers, the then 33-year-old mother of two from Yukon, Oklahoma, learned she had stage IIB breast cancer. In November, she started chemotherapy to shrink the 7-centimeter tumo​r in her left breast and kill the cancer cells that had spread to her lymph nodes. In March 2018, she had a mastectomy. When it was time to start radiation, Gray says, her radiation oncologist at the Integris Cancer Institute in Oklahoma City explained that proton therapy would be a better option than standard radiation therapy because “it would save my heart and lungs.”​

Gray’s doctor sent a treatment proposal for proton therapy to her health insurer. The request was denied. “I didn’t know insurance companies did that,” says Gray. Aided by a media consultant brought in by her doctor, Gray used social media and local news outlets to tell her story. Time was ticking—the first of 34 proton therapy radiation treatments that would target her lymph nodes and any breast tissue remaining in her chest wall was scheduled for May 10, just three weeks away. When her insurer wouldn’t budge, the proton therapy center, ProCure, agreed to front the cost. The same day, says Gray, the Indian Health Service, which also provided her with health benefits, called to say they would cover the cost of the treatment. “I was surprised, shocked and happy,” says Gray. “I had been praying and asking God if this is what needed to be done.”

For about a century, radiation therapy has been a mainstay of cancer treatment. Standard radiation systems use photons, or X-rays, to kill cancer cells. Proton therapy uses particles that can be targeted at the tumor more precisely. Studies have shown that proton therapy is safe and effective. Less clear is which patients with which types of cancer should receive it instead of standard radiation. Clinical trials that compare proton and photon therapies are now underway, but enrolling patients hasn’t been easy. And in the years that it takes fo​r the answers to come in, thousands more cancer patients will find themselves in a position similar to Gray’s.

Photons and Protons

Radiation kills a cell by damaging its DNA. The photon beam used in standard radiation therapy travels through normal cells in the body, gets into the cancer cells, and then travels again through normal cells as it comes out the other side of the body. Protons are particles with a different set of physical characteristics. They accelerate and penetrate the skin quickly, explains Steven Lin, a radiation oncologist at the University of Texas MD Anderson Cancer Center in Houston. Then the particles stop at the tumor, where they deposit all their energy at once.

The U.S. Food and Drug Administration (FDA) approved proton radiation as a cancer treatment in 1988. Before the FDA can approve a new cancer drug, clinical trials must show that the treatment is safe and effective for a specific type of cancer. New devices and technologies like proton therapy are held to a different benchmark. They only have to be proved safe and effective overall, not for a specific use. This means “there is no clear indication where proton [therapy] should be the standard treatment,” says Lin. Instead, “every cancer patient who needs radiation is potentially eligible for proton treatment, but not all patients will benefit.”

When there are no specific indications for a treatment’s use, insurance coverage can vary widely. Medicare typically covers the cost of proton therapy, regardless of the type of cancer. But many private insurers do not want to pay for proton therapy when it has not been shown to be more effective than standard radiation therapy and can cost four to 10 times more. A recent study found that two-thirds of patients with private health insurance initially had their requests for proton therapy denied. (On appeal, about 68% of patients initially denied coverage had their treatment approved.)

​Determining the BenefitFor children with cancer, proton therapy is now a routine treatment. “For many pediatric patients, proton therapy offers clear benefits,” says Shannon MacDonald, a radiation oncologist at Massachusetts General Hospital in Boston. When treating children, she explains, “you are treating brain tumors and tumors close to areas that are responsible for future growth.” Before proton therapy was available, some of these children would not have been able to have radiation at all. With proton therapy, she says, they can be treated, and the tissue spared from radiation will continue to grow and develop normally. Proton therapy has also made radiation a possibility for some adults with rare or difficult-to-treat cancers, such as tumors in the central nervous system, brain, head and neck, eye, skull and spine.

In other instances, proton therapy has allowed many patients to avoid some or all of the potential side effects associated with standard radiation therapy, which can include skin problems, pain and swelling, and heart and lung problems. That was the case for Arianne Missimer of Coatesville, Pennsylvania, who was diagnosed in 2015 with a stage III liposarcoma—​a rare cancer that can start in muscle tissue—in her right thigh. The 34-year-old physical therapist, registered dietitian and athlete needed radiation therapy to treat her cancer and was concerned about her potential risk for pain, swelling, weakness and long-term bone damage. Her radiation oncologist explained the difference between photon and proton therapies and then suggested proton therapy at Penn Medicine’s Roberts Proton Therapy Center in Philadelphia. Her insurer was willing to cover it.

A Growing Business

Proton therapy centers are now ​located across the U.S.

​Waiting for Answers

It’s unclear whether proton therapy improves outcomes and reduces side effects in other cancer types, including breast and prostate cancer. The National Cancer Institute (NCI) and the Patient-Centered Outcomes Research Institute (PCORI) have funded seven phase III randomized trials comparing proton therapy and photon therapy in patients with breast, esophageal, liver, lung and prostate cancer and two types of brain tumors, glioblastoma and low-grade glioma. Some of the trials are comparing overall survival; others are looking at reductions in symptoms and side effects.

New Research Sheds Light on Side Effects

When combined with chemotherapy, proton therapy is associated with fewer severe s​ide effects than standard radiation therapy, according to a​ study.

The results of these trials have the potential to inform future treatment guidelines, but finding patients for the studies has been laborious. In 2018, almost two years after it opened, the breast cancer trial had enrolled only 317 of 1,716 patients needed; after five years, the prostate cancer trial, which needs 400 patients, had enrolled only 254. Radiation oncologists point to multiple factors contributing to the slow patient accrual. In some cases, says Lin, doctors may believe proton therapy is better, and they don’t want their patients to participate in a clinical trial where there is a chance they won’t receive the newer approach. In other instances, patients don’t want to take the chance they will be assigned to the treatment arm that doesn’t receive proton therapy.

There is also an insurance barrier. In the major proton therapy trials, insurers are asked to pay for patients’ radiation treatment, whether it’s proton or photon therapy. Justin Bekelman, a radiation oncologist at the Penn Medicine Abramson Cancer Center, says it’s all too common for insurers to say they won’t pay for an unproven treatment when a patient is selected for the proton therapy arm. Bekelman was the lead investigator for the breast cancer trial and a co-lead investigator for the prostate cancer trial.

“Naturally, insurance companies are going to question the value,” says Bekelman. “That’s precisely why we need to run these trials. We want to determine if there are benefits and if there are harms to proton therapy, and in which cancer patients which treatment will be most successful for cancer control and reducing side effects.” But researchers can’t do that if insurers won’t cover that care.

In 2012, the University of Texas MD Anderson Cancer Center launched the NCI-funded clinical trial comparing protons and photons in esophageal cancer, which aimed to enroll 180 patients. Enrollment closed this year with 104. (Another 21 patients enrolled but couldn’t be evaluated because their insurer wouldn’t pay for the proton therapy.) Lin, who is overseeing the study, says some patients declined to enroll when they learned their health insurance covered proton therapy. “We explain to [patients] that the proton therapy is experimental, which is why we are trying to do the study,” he says. “But they say they’ve heard good things about it. Others say, ‘I have money and I don’t want standard treatment. I want the best.’”

It’s easy to understand why a patient who has pored over a proton therapy center’s website might feel that way. In a study published online March 15, 2018, in Radiation Oncology​, researchers analyzed 46 websites of proton therapy centers—half of which w​ere in the U.S. The analysis found that many centers used language that could lead patients to think that choosing proton therapy would give them a better outcome, says the study’s senior author Alexander Louie, a radiation oncologist and epidemiologist at Sunnybrook Health Sciences Centre in Toronto. “Many of the websites made blanket or generic statements that may not be completely supported by evidence but have some credence potentially or theoretically, blurring the line between evidence and advertising,” he says.

“It’s not as easy as saying if proton therapy is good or bad,” adds radiation oncologist Jeffrey Buchsbaum of the NCI’s Radiation Research Program. “Proton therapy is like a vehicle for getting the patient to a better place. And it has to be used properly.” There are certain situations, he notes, in which patients wouldn’t be alive without proton therapy. “But that doesn’t mean it’s necessary for all cancers.”

Proton Therapy Tips

Follow​ these suggestions​ as you consider radiation therapy options.

​Moving Forward

The American Society for Radiation Oncology has developed model policies for insurers that delineate where there is sufficient evidence to support coverage of proton therapy. Insurers also use National Comprehensive Cancer Network treatment guidelines to support or deny a patient’s treatment with proton therapy. To move research forward, investigators are trying to work with hospitals to find ways to make insurers more amenable to covering the cost of treating patients in randomized clinical trials comparing photon therapy and proton therapy. In some cases, this may include reducing the cost of proton therapy to make it more comparable to that of standard radiation therapy. “The issues happening here are partially the result of the complexity of the health care delivery system,” says Buchsbaum.

But for patients, treatment choices must be made now. Missimer believes that proton therapy helped treat her cancer without sacrificing her athleticism. She is an active member of Penn Medicine’s proton center alumni group, which provides support to patients who are currently receiving or are considering proton therapy. She also appears in an advertisement for Penn Medicine’s proton therapy center, and an article about her experience is included on the cancer center’s website.

Missimer’s treatment began with chemotherapy, which she admits slowed her down. But during her proton therapy, which started in July 2015, she joined a ninja gym. And as she recovered from the surgery and additional chemotherapy that followed the radiation, she kept going. In May 2016, Missimer competed in the Philadelphia regional American Ninja Warrior competition. “I lost my brother to cancer,” she says. “He had radiation and had significant complications. The only thing I get is a little stiffness. But as long as I keep moving, my leg is good.”

Gray completed her proton beam treatment in June 2018, about a year after she’d first felt the lump in her breast. Skin damage is a common side effect of both types of radiation therapy. Gray says her doctor told her that her skin did well during the proton therapy. “But if that was well,” she says, “I can’t imagine what worse would be like. My chest looked like burnt hot-dog skin. And I still have a dark scar from the burn that might not ever go away.” After being out of work for a full year, Gray returned to her job as an educational specialist for Native American youth in October 2018, and she slowly started back at the gym. She wears a compression sleeve and a glove to manage lymphedema that developed in her arm—caused by either the surgery or radiation—and deals with nerve pain in her arm and chest. None of it has been easy, but, she says, “my faith has gotten me through.”​ 

Sue Rochman is a contributing editor for Cancer Today.​

The Right Dose

This blog was originally published by Cancer Today by Kate Yandell here.

Researchers want to find out when cancer patients can benefit from receiving lower doses of drugs or radiation, shortening treatment or skipping certain treatments altogether.

​​​

OVER A SPAN OF 15 YEARS, ​Liza Bernstein was diagnosed with three separate primary, early-stage breast cancers. Even though she was treated by the same oncologist throughout, the treatments she received varied with each diagnosis.

​Bernstein, who lives in the Los Angeles area, was first diagnosed with hormone receptor-positive breast cancer in 1994, when she was 29 years old. She recalls that her doctors were pleased to be able to do a lumpectomy, only removing part of the breast, instead of a mastectomy as would once have been standard. However, her surgeon removed about 20 lymph nodes from her armpit, and she received both radiation and chemotherapy.

In the course of receiving her second diagnosis, a hormone receptor-positive cancer in her opposite breast, in 2005, Bernstein underwent a sentinel lymph node biopsy, a less invasive procedure that requires surgeons to remove only a few lymph nodes in areas where the cancer is most likely to have spread.

Bernstein was also able to get testing with a product called Oncotype DX, which measures gene expression in breast tumors and helps estimate the likelihood that chemotherapy will prevent an early-stage, hormone receptor-positive cancer from recurring. The test, released in 2004, helped Bernstein and her oncologist make the difficult decision to skip chemotherapy in 2005, due to little predicted benefit. Bernstein received a lumpectomy, radiation and the hormone therapy tamoxifen. Conversely, when she was diagnosed with another hormone receptor-positive cancer in 2009, genomic tumor testing helped them decide to include chemotherapy, along with a double mastectomy and tamoxifen, in her treatment.

Advances in cancer research can mean making patients’ treatment more onerous and complex. But some of the changes in Bernstein’s breast cancer treatment over the years reflect de-escalation—the process of decreasing the intensity or duration of a treatment, thus reducing side effects and cost, while maintaining the treatment’s effectiveness.

Today, researchers are investigating whether they can identify patients—using genomic tumor testing, imaging of the cancer or other methods—who can receive less intense treatment. Treatment de-escalation aims to spare patients the burden of unnecessary treatments and side effects.

“The key is we want to give people the right treatment that they need without treating them excessively, which just produces too much toxicity,” says Eric Winer, a medical oncologist and chief of the Division of Breast Oncology at the Dana-Farber Cancer Institute in Boston.

Treating the Right Patients

Treatment de-escalation has been successful primarily in cancers where the survival rate is high. “When you have a situation where mortality from a given malignancy is high, then it’s pretty hard to think about backing off [from treatment],” Winer explains.

The effects of treatment can last long after chemotherapy or radiation is completed. For example, 87% of people in the U.S. diagnosed with Hodgkin lymphoma, which until the 1960s was usually fatal, live five years or more. “The issue for this group of people, who are often diagnosed in their 20s and 30s, is that they have a long life ahead of them,” says Peter Johnson, a medical oncologist who specializes in lymphoma at University Hospital Southampton in England. The radiation and chemotherapy typically given for Hodgkin lymphoma can result in serious side effects, including heart disease, second cancers and infertility.

Over time, doctors have adopted techniques for delivering radiotherapy to Hodgkin lymphoma patients that increasingly spare normal tissues from damage, Johnson says. Most recently, researchers have learned that they can perform a form of imaging, called 18F-fluorodeoxyglucose PET, to determine early on whether a patient’s Hodgkin lymphoma is responding to chemotherapy. If the scan indicates a good response, the patient may be able to skip later radiation therapy or receive a less intensive chemotherapy regimen.

“In some ways, it’s a reflection of how successful modern oncology has been that we’re thinking about these things,” Johnson says of the topic of de-escalation.

The rise of genomic testing, among other factors, has contributed to a decline in chemotherapy use for patients with early-stage breast cancer whose disease is driven by hormones. With Oncotype DX and similar tests, patients with hormone receptor-positive, HER2-negative breast cancer can learn how likely they are to benefit from chemotherapy. Their score can help determine whether their drug treatment after surgery should include both chemotherapy and hormone therapy or whether just hormone therapy is enough.

Researchers are investigating de-escalation strategies for patients with early-stage HER2-positive breast cancers as well. These patients are often treated with HER2-targeted therapy and a multidrug chemotherapy regimen. Winer’s research shows that patients with small HER2-positive cancers that have not spread to the lymph nodes can safely use a de-escalated ​chemotherapy regimen that includes just one drug, paclitaxel, alongside targeted therapy.

Challenges of Stepping Back

Despite some successes in de-escalation, it can be easier to intensify treatment than to take treatment away. This is partly because it is difficult to prove that taking away treatment is not going to harm patients—a different statistical challenge than showing that a therapy is significantly better than standard care.

For example, in 2004, researchers discovered that patients with stage III colon cancer lived longer if oxaliplatin was added to their chemotherapy regimen. The additional chemotherapy drug can lead to peripheral neuropathy, and the effects are cumulative as therapy continues. An international consortium of researchers published a study in the New England Journal of Medicine​ on March 29, 2018, pooling the results of six randomized clinical trials that included 12,834 participants. The trials investigated the practice of shortening chemotherapy after surgery from six to three months for these patients.

“We thought with such a large number it would be very easy and we’d get a clear answer, [but] we haven’t got as clear an answer as we thought we would,” says Timothy Iveson, a medical oncologist at University Hospital Southampton who co-authored the study.

The study did not meet pre-specified statistical benchmarks to determine that a shorter period 
of chemotherapy was not worse than standard chemotherapy for the patients in the trial in general. However, the survival difference between patients using shorter versus longer chemotherapy (six months versus three months) was small, Iveson says, and the decrease in side effects with shorter chemotherapy was large. And for some patients, treatment for three months was sufficient. Cancer treatment guidelines now recommend the shorter chemotherapy regimen as an option for certain patients with low-risk stage III colon cancer.

New information about cancer subtypes can also spur de-escalation. But even when it’s clear that de-escalation is necessary, it can take time to settle on the right strategy, as shown by the experience of researchers trying to back off treatment for head and neck cancer caused by the human papillomavirus (HPV). “There’s been an epidemic of oropharyngeal cancers that are related to HPV,” explains Joshua Bauml, a medical oncologist at the Hospital of the University of Pennsylvania in Philadelphia. “These cancers have a much higher cure rate, and that’s wonderful, but the issue is that our treatment paradigm is still based upon older cancers with a different biology.”

Standard treatment for patients with advanced head and neck cancer—originally developed for patients with smoking- and alcohol-associated cancers—involves some combination of surgery, radiation and chemotherapy. But these treatments can cause troubling side effects, including difficulty swallowing, dry mouth, problems with speech and changes in taste.

One approach for reducing toxicity of chemotherapy for these patients was to replace the chemotherapy drug cisplatin with the targeted therapy Erbitux (cetuximab), in an attempt to spare patients the side effects that cisplatin can cause when combined with radiotherapy. However, recent clinical trial res​ults have shown that patients with HPV-positive oropharyngeal cancer treated with Erbitux have shorter survival than those treated with cisplatin and have similar rates of side effects, indicating that this is not a good de-escalation strategy.

Early trials of approaches to reduce doses of radiation ​or chemotherapy for patients with HPV-related oropharyngeal cancer have shown promise, Bauml says. However, he urges clinicians to wait for further data before adopting new protocols for HPV-related oropharyngeal cancer. “If a head and neck cancer metastasizes, it is incurable,” he says. “It’s really essential that when we move towards treatment de-escalation, this is done through robust clinical trials.”

Getting Targeted

The term de-escalation is used most often to describe efforts to reduce harms from old modes of therapy, including surgery, radiation and chemotherapy. But researchers are also working to understand the right doses of medication for patients being treated with newer targeted therapies and immunotherapies.

A study in the July 2018 issue of Cancer, for instance, showed that Sprycel (dasatinib), a type of targeted therapy called a tyrosine kinase inhibitor, is effective at a reduced dose in treating chronic myeloid leukemia (CML). The lower dose appears to cause fewer dangerous side effects, such as buildup of fluid near the lungs, and costs around half as much. Other tyrosine kinase inhibitors have also been shown to be effective in treating CML at reduced doses, says study co-author Hagop Kantarjian, an oncologist who specializes in leukemia at the University of Texas MD Anderson Cancer Center in Houston.

Traditional methods of determining doses for cancer drugs aren’t always ideal for dosing targeted therapies, Kantarjian explains. Clinical trials for chemotherapy ramp up doses in people until the highest dose with acceptable side effects is found, a measure known as maximum tolerated dose. Targeted therapies, in contrast, can be effective at doses much lower than the maximum tolerated dose. Researchers are still trying to find the best strategies for determining dosing of targeted therapies.

Researchers are also investigating whether they can reduce the time that patients are on targeted therapies and immunotherapies. For instance, “there are no clear, specific guidelines on exactly how long to treat patients with immune therapy in cancer,” says Michael Postow, a medical oncologist at Memorial Sloan Kettering Cancer Center in New York City who treats patients with melanoma.

Scientifically, it makes sense that patients who respond to immunotherapy drugs might be able to stop taking them at some point, says Janet Dancey, scientific director of the Canadian Cancer Trials Group and a medical oncologist at Queen’s University in Kingston, Ontario.

Most cancer drugs work by directly killing or inhibiting the growth of cancer cells. In contrast, immunotherapies work by stimulating the immune system to attack cancer. It’s possible that once the immune system has been activated, continued administration of the drugs isn’t necessary.

Dancey’s organization is currently enrolling patients for the STOP-GAP study, a randomized trial looking into whether melanoma patients who have responded to a class of immunotherapy drugs called PD-1 inhibitors can stop treatment or whether they would benefit from staying on treatment indefinitely.

There are multiple reasons to stop treatments, says Postow. “People would want to stop mostly to get their lives back to themselves, for flexibility in travel and work. … And I think the idea of being under treatment is still a reminder that there is something wrong with the patient.”

There are also financial implications: Checkpoint inhibitors have generally debuted with list prices of $150,000 per year or more. And treatment comes with other costs like time taken off from work, Postow says.

Currently, Postow works with his patients to make individual decisions on whether to stay on immunotherapy after all evidence of active cancer disappears or after two years of improvement on the treatment. He hopes further research will make choices easier for patients. “As you can imagine, there is a lot of emotional decision-making around this issue, too, which is reasonable in a setting where we don’t have strong science to specifically guide us,” he says.

A Lower Dose of 
Financial Toxicity

Researchers are​ looking into whether some drugs are just as effective when taken at a reduced​ dose.

​A Shared Decision

Whether patients are considering skipping chemo​therapy or stopping immunotherapy, having thoughtful discussions about benefits and risks of treatments is key. That includes helping patients understand side effects, says Iveson, who studied shortening chemotherapy for colon cancer patients. For instance, rather than telling patients they might experience peripheral neuropathy, doctors should explain this can mean not being able to button a shirt or feel one’s feet.

“The challenging part is that, for both doctors and patients, there’s a tendency to be risk averse,” Winer notes. People don’t like to feel they are leaving potential benefits of treatment on the table. Doctors sometimes underestimate side effects and overestimate treatment benefits, he says, and “nobody wants to be judged as having done something wrong by backing off if there’s a bad outcome.”

For Bernstein, the lengthy decision-making process that came with skipping chemotherapy after her second cancer diagnosis was difficult because there wasn’t a clear-cut answer of what to do, at least until she got the Oncotype DX test results. But she says she ultimately was glad to have had in-depth discussions with her doctor. Despite progress in treatment de-escalation, Bernstein hopes more can be done both to eliminate unnecessary treatment and to treat cancer more effectively.

“Over time there have been strategies that have come into play and have helped, in a sense, to do less harm, but by no means do they do no harm,” Bernstein says. “I want to make that clear.”​ 

Kate Yandell is the digital editor of Cancer Today.

 

LiveHelp Online Chat – Clinical Trials

This resource was originally published by Live Help Cancer here.

 

NCI Information Specialists can answer your questions about cancer, clinical trials, and quitting smoking. LiveHelp is confidential. Our Information Specialists do not provide medical advice and our service is not a substitute for talking with your health professional.

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El servicio de LiveHelp también está disponible en español en el siguiente enlace: https://LiveHelp-es.cancer.gov.

Immunotherapy

This video was originally published by the National Cancer Institute on June 13, 2018, here.

 

Immunotherapy uses the body’s immune system to fight cancer. This animation explains three types of immunotherapy used to treat cancer: nonspecific immune stimulation, T-cell transfer therapy, and immune checkpoint inhibitors. http://www.cancer.gov/immunotherapy

About Clinical Trials Videos

These videos were originally published by the American Cancer Society here.

 

Clinical Trials Videos

Knowing all you can about clinical trials can help you feel better when deciding whether or not to take part in one as part of your cancer treatment. Explore these videos to get answers to basic questions and concerns about clinical trials so that you will be better prepared to discuss this option with your doctor and your family. (ACT videos were produced by Genentech in collaboration with the American Cancer Society.)

Is It Difficult to Participate in a Clinical Trial?

Clinical Trial Mythbusters

Clinical Trial Mythbusters: Is It Difficult to Participate in a Clinical Trial? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

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

Transcript:

Andrew Schorr:

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 questions@patientpower.info. 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?

T.J. Sharpe:

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.

Andrew Schorr:

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?

T.J. Sharpe:

Yes. It’s been five years of treatment followed by now two good years of a clean bill of health.

Andrew Schorr:

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.

T.J. Sharpe:

You’re welcome. It’s my honor to be able to represent all these patients.

Andrew Schorr:
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?

Ken Getz:

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.

Andrew Schorr:

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.

Andy Lee:

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.

Andrew Schorr:

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?

Ken Getz:

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.

Andrew Schorr:

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.

T.J. Sharpe:

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.

Andrew Schorr:

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?

Andy Lee:

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.

Ken Getz:

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.

Andrew Schorr:
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.?

T.J. Sharpe:

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.

Andrew Schorr:

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.

Andy Lee:

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.

Andrew Schorr:

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?

Ken Getz:

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.

Andrew Schorr:

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?

Andy Lee:

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.

Andrew Schorr:

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?

Ken Getz:
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.

Andrew Schorr:

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.

Ken Getz:

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.

Andrew Schorr:
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?

T.J. Sharpe:
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.

Ken Getz:

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.

Andrew Schorr:
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?

Andy Lee:

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.

Andrew Schorr:

Great. I wanted to note for your audience. If you have a question, send it to questions@patientpower.info. 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?

T.J. Sharpe:

Nearly four years. Three-and-a-half years.

Andrew Schorr:

Were there ever times when you said, “I’m done. I want to bail out.” You know.

T.J. Sharpe:

I’ll be very careful how I answer this question for Andy’s sake.

Andy Lee:

It’s okay, T.J., we’re friends.

T.J. Sharpe:

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.

Andrew Schorr:

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?

Andy Lee:

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.

Andrew Schorr:

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.

Andy Lee:

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.

Andrew Schorr:

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.

Ken Getz:
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.

Andrew Schorr:

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.

T.J. Sharpe:

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.

Andrew Schorr:

Right, to be honored. So, Andy, at Merck you’ve established some internet platforms in particular related to keeping people informed, right?

Andy Lee:

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.

Andy Lee:

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.

Andrew Schorr:

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?

Andy Lee:

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.

Andrew Schorr:

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.

Ken Getz:

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.

Andrew Schorr:

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?

Andy Lee:

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.

Andrew Schorr:

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.

Ken Getz:

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.

Andrew Schorr:

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.

Andy Lee:

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.

Andrew Schorr:

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?

T.J. Sharpe:

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.

Andrew Schorr:

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.

Clinical Trial Toolkit

Clinical Trial Mythbusters: How Does Medicare or Medicaid Impact My Ability to Participate in a Clinical Trial?

 

Clinical Trial Mythbusters: How Does Medicare or Medicaid Impact My Ability to Participate in a Clinical Trial? from Patient Empowerment Network on Vimeo.

Downloadable Guide

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?

Transcript:

Laura Levaas:             

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 questions@patientpower.info. 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.

Mark Fleury:              

Hello Laura. Thanks for having me on.

Laura Levaas:             

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.

Mark Fleury:              

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.

Laura Levaas:             

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?

Mark Fleury:              

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.

Laura Levaas:             

Okay. What are some of the barriers around Medicare and Medicaid patients who want to get involved in a clinical trial?

Mark Fleury:              

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.

Laura Levaas:             

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?

Mark Fleury:              

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.

Laura Levaas:             

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.

Mark Fleury:              

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.

Laura Levaas:

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.

Mark Fleury:              

You’re welcome.

Laura Levaas:             

Hi Jeanne.

Jeanne Regnante:        

Hey Laura.

Laura Levaas:             

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?

Jeanne Regnante:        

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.

Laura Levaas:             

And would you say that seniors are also part of this underserved population?

Jeanne Regnante:        

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.

Laura Levaas:             

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 –

Jeanne Regnante:        

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.

Laura Levaas:             

So, then circling it back around to clinical trial participation, how can we connect the dots here?

Jeanne Regnante:        

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.

Laura Levaas:             

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.

Jeanne Regnante:        

I’ll let Mark take that one.

Mark Fleury:

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.

Laura Levaas:             

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 questions@patientpower.info. So, now I would like to talk with Laura McHugh. Are you with us, Laura?

Laura McHugh:          

I am. Thank you so much for having me.

Laura Levaas:             

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?

Laura McHugh:          

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.

Laura Levaas:             

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?

Laura McHugh:          

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.

Laura Levaas:             

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?

Laura McHugh:          

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.

Laura Levaas:             

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?

Laura McHugh:          

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.

Laura Levaas:             

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”?

Laura McHugh:          

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.

Laura Levaas:             

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?

Laura McHugh:          

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.

Laura Levaas:             

This may seem like a silly question, but do folks on those programs get the same care as somebody that has a private insurance?

Laura McHugh:

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.

Laura Levaas:             

Wonderful. And again, just to underline that clear and open communication is important.

Laura McHugh:          

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?”

Laura Levaas:             

Thank you, Laura.

Laura Levaas:             

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?

Mark Fleury:              

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.

Laura Levaas:             

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?

Jeanne Regnante:        

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.

Laura Levaas:             

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 –?

Jeanne Regnante:        

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.

Laura Levaas:             

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?

Laura McHugh:          

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.

Laura Levaas:             

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?

Mark Fleury:              

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.

Jeanne Regnante:        

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.

Laura Levaas:

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?

Mark Fleury:              

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.

Laura Levaas:             

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?

Laura McHugh:          

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.

Laura Levaas:             

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?

Jeanne Regnante:        

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.

Laura Levaas:             

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?

Jeanne Regnante:        

Oh, I heard you say Laura.

Laura Levaas:             

Yeah. Sorry.

Jeanne Regnante:        

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.

Laura Levaas:             

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.”

Mark Fleury:              

Yeah. I’m happy to jump in with a quick answer on that.

Laura Levaas:             

Okay. Thanks Mark.

Mark Fleury:              

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.

Laura Levaas:             

We have one more. Annie B, “I’m on Medicare. Where do I find a clinical trial in my town?”

Mark Fleury:

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.

Laura Levaas:             

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.