Clinical Trial MythBusters: What Is the Value of Diversity in Clinical Trials?

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Is diversity important in clinical trials? Mel Mann, a 20+ year survivor of chronic myelogenous leukemia (CML) and his wife, Cecelia Mann, both think so. In 1995, Mel Mann was told he needed to get his affairs in order, as he was given three years to live. Fortunately, clinical trials extended his life. Mel shares the impact that tomorrow’s medicine today has had on his life. Dr. Richard Schilsky, Senior Vice President and Chief Medical Officer (CMO) of ASCO, unpacks many of the issues that have led to the lack of diversity in clinical trials and initiatives in place that are changing all of this.

This is a Patient Empowerment Network program produced by Patient Power. We thank AbbVie, Inc., Astellas, Celgene Corporation, and Novartis for their support.

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Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. 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.

Andrew Schorr:

And greetings from near San Diego, Carlsbad, California. I'm Andrew Schorr from Patient Power. Welcome to this Patient Empowerment Network program, the next in our series Clinical Trial MythBusters, and this program is so important, discussing what is the value of diversity in clinical trials.  And, believe me, you'll hear it is so critical.  We have to do better, and we're going to discuss that over the next hour. 

I want to thank the companies that have provided educational support through grants to the Patient Empowerment Network.  They have no editorial control, but their support is welcome.  And that is AbbVie Incorporated, Astellas, Celgene and Novartis.  All right.  

We've got a lot to discuss today, and we welcome your questions along the way.  I want to first introduce someone who, like me, has greatly benefited from a clinical trial and believes that they are alive today because of their participation. And so joining us from Atlanta is Mel Mann along with his wife and care partner Cecelia Mann.  Mel, welcome to the Patient Empowerment Network program. 

Mel Mann:
Thank you very much.  

Andrew Schorr:
And we're going to hear more of Mel's story in just a minute.  I want to introduce a very prominent medical expert who joins us.  He is the senior vice president and chief medical officer at really the largest cancer organization, the American Society of Clinical Oncology, and that is Dr. Richard Schilsky. Dr. Schilsky, welcome to our program.   

Dr. Schilsky:
Thank you, Andrew.  Happy to join you.  

Andrew Schorr:
Okay. And are you in the Washington, DC, Virginia area?  

Dr. Schilsky:
That's where our organization is based, in Alexandria, Virginia, yes.  

Andrew Schorr:
Thank you for being with us.  I should mention that Dr. Schilsky has had a lot of experience related to trials. He was the head of hematology/oncology at the University of Chicago, which of course Chicago is a very diverse city, and the University of Chicago does a lot of research.  And he also helped run a big research group that doctors from around the world are part of, and he did that for many years.  So we're going hear more from Dr. Schilsky in just a minute.  But, first, Mel.  So Mel, in the late '90s you were dying, right, of chronic myeloid leukemia, correct? 

Mel Mann:
Yes, I was.  

Andrew Schorr:
Losing weight and being told that there wasn't much to do, right?  

Mel Mann:
Correct, yes.  

Andrew Schorr:
Maybe a transplant.  But you were in Atlanta and you went from doctor to doctor, but somehow you got to MD Anderson, a major research center in Houston.  What did they offer you there?  

Mel Mann:
When I first went out to MD Anderson they said they were going to put me on a clinical trial after clinical trial.  And the first thing they did was increase my dose of interferon, and that was the medication, the standard therapy at the time, and then they tried different combinations of drugs, and eventually I started on different clinical trials.  

Andrew Schorr:
Okay. And, Cecelia, you were there in Atlanta and he was scooting over to Houston, it's not exactly around the corner. Why were you supportive of that?  

Cecelia Mann:
I was supportive of that because that was the last chance that he had to a cure and for survival.  So from the very beginning, whatever type of treatment he needed when he was flying around, whether he was going looking for bone marrow transplants, doing bone marrow drives, and therefore I was supportive of. We had a 5?year?old daughter at the time, and so anything that Mel needed I was there to support him.  

 

Andrew Schorr:
So, Mel, this is a happy story because here we are in 2018 as we do this program and you are with us when many people with CML at the time were not with us that long.  Hopefully, a transplant could be curative, but a lot of people passed away.  You were lucky enough to come back as they were rolling through different trials and there was a new one that opened up for a drug called imatinib mesylate (Gleevec), a pill.  

Mel Mann:
Yes. 

Andrew Schorr:
What happened?

Mel Mann:
Okay. So in the summer of '98 the Phase I Gleevec study opened up, and I went out to MD Anderson, and I was patient number two, and I started taking it at a low dose, and it was effective for me. And eventually they increased the dose and it started changing my leukemia around to eventually I reached what they call a major molecular response.  And that was 20 years ago.  This summer I went over 20 years.  

Andrew Schorr:
Wow. Well, Dr. Schilsky, is that an example of a patient getting, if you will, tomorrow's medicine today, what we hope for?  

Dr. Schilsky:
Absolutely.  And, first of all, it's such a wonderful story, Mel.  It's great to hear you tell it, and it's exactly why we do research, exactly why we do clinical trials, to try to discover the new therapies that people need that will give them the kind of long?term survival and quality of life that you've been experiencing.  It's just—it's wonderful.  

Andrew Schorr:
So, Dr. Schilsky, let's get into the problem.  So, generally, there are many clinical trials that take longer than one would hope to fill, and the FDA and I know scientists have been seeing well, gee, how do we know what we're testing applies to people maybe with different ethnic, gender backgrounds, a variety of situations, and often we can't find people who fit those categories to be in.  What is that—how poorly have we been doing in the past with diversity in trials, and what does that mean for developing new medicines?  

Dr. Schilsky:
Well, we don't do well in almost any dimension. We don't get enough underrepresented minorities in clinical trials.  We don't get enough older people in clinical trials.  You have to remember that 60 percent of cancers occur in people 65 years and older, and yet only about 10 percent of people participating in clinical trials are 65 and older.  So we are having to treat the majority of older people, and I would say the majority of minority people, with data derived from participants in clinical trials who are not like them.  

We need to change that for a whole host of reasons.  It's historically been very challenging, and the problems really sort of boil down into three big areas that I think we can discuss a little bit further.  

First is awareness.  Many people are not aware that clinical trials are even an option for them.  Many people think that a clinical trial is a last resort, and I want to dispel that myth right out of the box.  Clinical trials can be a very good option for patients right from the time of their cancer diagnosis even if it's their very first treatment.  So clinical trials may be a last resort, but they don't have to be, and there are many clinical trials that are appropriate for people right following the initial diagnosis of their cancer.  

So there's the awareness issue, and sometimes, frankly, not even the doctors are aware of what clinical trial options are for their patients. And the one thing we know for sure is that the most influential person as to whether or not a patient goes into a clinical trial is their doctor.  If the doctor does not recommend it, if the doctor is not aware of it, it's not going to happen.  

But then you get into the more technical issues.  There are things, there are rules for clinical trials, because they are research studies.  They are experiments.  There are very well defined rules, most of which are in place to protect the people who are participating in the study.  Some of these rules are called eligibility criteria, and they specify the characteristics of people who can enroll in the study.  Well, historically, they tend to be very rigid and very limiting, and you'll often hear people talk about how the only people who can get into clinical trials are Olympic athletes.  That may be the case, but it's not Olympic athletes that we're treating in the clinic every day, so we need to make our clinical trials more representative so that they're more applicable to the typical person that a doctor sees in their office.  

And then there are the logistical or operational issues of the clinical trial.  The clinical trial can be very burdensome.  Mel just described how he had to travel from his home in Atlanta to Houston to participate in a clinical trial.  Not everybody can afford to do that.  Not everybody can take time away from work, time away from home.  And the clinical trial requires not only that you travel sometimes but that you travel on a specific schedule because of the requirements of the trial.  

So all of these are issues that are—can limit participation in trials, and many of them are magnified in minority populations or in populations that don't have the economic resources to be able to meet the requirements of the trial.  

Andrew Schorr:
Right.  Let's talk about that for a minute.  So, Cecelia, you go out in the community and speak to people, and you probably meet some people who maybe are diagnosed with a blood cancer, like you're active with the Leukemia and Lymphoma Society so you may speak to them, and they say even if you brought up about a trial, they say, hey, Cecelia, I'm working two jobs. Or my husband or spouse is working two jobs, and we've got two kids, three kids.  How could we ever participate?  We just can't get away or we don't have the family support or whatever. Those are real issues, right?  

Cecelia Mann:
True.  True, those are real issues, and I try to direct them towards resources that Mel and I found out about along the way.  The Leukemia and Lymphoma Society, they have resources to help with travel, and American Cancer Society has resources that help with the hotel and lodging. And there are a number of other different types of funds that can be assessed to make that a possibility.  

But you're right, Andrew.  I had one young lady at a health fair and a second cancer had popped up, and she was coming there to get information, and she was saying that they were saying it wasn't too much they could do, and I brought up MD Anderson. And she immediately said, I can't go out there, I have to go to work, and she turned around rough.  And so when they listen to me then always glad to tell them about it and let them know there is an option and that clinical trials work, and I point to Mel, my husband, as a success story also.  

Andrew Schorr:
So, Dr. Schilsky, you talked about physician awareness.  It's also about more physicians participating in trials at I think what you call the community level.  So, in other words, MD Anderson and where you work, at the University of Chicago, those are big city centers and where they are in Atlanta there's Emory and some other mainly centers, but what about out in the hinterland, if you will?  Can somebody who lives there diagnosed with a cancer, how do they have access to a trial that their doctor knows about and maybe that's more close to home, if you will? 

 

Dr. Schilsky:
Right.  So, of course, we know that anybody with cancer prefers to be treated in their community, and most are.  So one of the goals is to be sure that oncologists practicing in all sorts of community settings have access to clinical trials.  Now, one of the ways that happens is that for more than 50 years now the National Cancer Institute has actually been operating and funding a community?based clinical trials network.  It used to be called the CCOP program.  That's an acronym that we don't have to go into.  They've recently changed the name.  It's now called NCORP program, but—that stands for, I think, the National Community Oncology Research Program.  

But the point is that the program, which is in most but not all of the states in the United States, funds community oncologists to participate in NCI?sponsored clinical trials, and there are at least 65 or 70 such clinical facilities around the country right now.  So in those medical practices patients can find those clinical trials in their communities without having to travel.  

There are also other community?based networks that are active participants in cancer clinical trials.  So I think at the end of the day the critical thing for patients, and this is sometimes easily forgotten because you're so, you know, your thinking and your time and your emotion are all taken up in dealing with the cancer diagnosis. It's really important, though, to ask the doctor, do they have access to clinical trials.  Do they have a clinical trial that might be appropriate for you? And if not you might want to consider where else you could go, hopefully still relatively nearby to get access to a clinical trial.  

Andrew Schorr:
Mel, so for you, you went around to some doctors who were not aware of anything new to do for you, right?  And that's still true in so many areas of cancer.  Now, what do you say to patients about speaking up because Dr. Schilsky just referred to it, people are terrified.  They really just want the doctor to have the answer.  What do you tell people so that they maybe advocate for themselves?  

Mel Mann:
Okay. So if you're looking for a clinical trial and I'm out at, like you say, a health fair, we have a—The Leukemia & Lymphoma Society has something called the clinical trial support center, and they have nurses who work early in the morning till late at night.  And you call them up and you tell them about your illness, and they check the availability for what clinical trials are out there, and then they narrow it down to what you actually qualify for, and then they take into consideration your finances and other issues.  

And then you're left with a number of possible clinical trials that you have, and you can take that back to the doctor and you can discuss that with him.  So that's one of the things I talk with them about.  

Andrew Schorr:
I could mention, now this is really more broadly across cancer, too.  So there are breast cancer groups, there are lung cancer groups, and I would just make a pitch to find out, is there a local chapter or national number for you, for the cancer you or a loved one been diagnosed with, and that's the question.  

Mel Mann:
Yes. 

Andrew Schorr:
Say, look, A, I don't want to feel I'm alone, and, B, how do I get—how do I get connected with what could be lifesaving or life?extending treatments for me and that I could discuss with my doctor.  And understanding—and then, boy, if there are obstacles like financial issues, logistical issues, travel issue, is there support for that.  

Mel Mann:
Yes. 

Andrew Schorr:
So let's go back to the inclusion/exclusion or eligibility issue you spoke about, Dr. Schilsky, because, you know, somebody who has cancer may also have heart problems or diabetes or some other issue.  Maybe they previously had another cancer, and so for the companies developing new drugs they may be happy with narrow inclusion criteria because they don't want to have anything get in the way, some previous thing you've had, to affect their ability to have a new drug go on the market. 

So what kind of work is going on between government and the drug manufacturers so that the criteria, not so tight, but you can still get legitimate scientific answers?  

Dr. Schilsky:
Right.  So, as you alluded to, Andrew, there are good reasons that there are eligibility criteria. One of them is to protect the patients in the study from circumstances that would increase their risk of participating in the study.  Another is because the companies or whoever is sponsoring the study wants to be able to isolate the specific effect of what they're studying without having a lot of confounding factors that could muddy the water and makes it difficult to interpret the results.  But that said, the bad thing about all that is that the results of the study might not be applicable to the majority of people who could benefit from the treatment because they weren't included in the study to begin with.  

So one of the things that my organizations has been working on very hard over the last couple of years now, and we've been doing this collaboratively with people from the Food and Drug Administration and the National Cancer Institute and a lot of clinical experts and a patient advocacy group, Friends of Cancer Research, is to try to expand or broaden or simplify some of these eligibility criteria that tend to keep people off of trials and in particular tend to keep minority populations off of clinical trials.  

So, for example, it's not unusual for someone who has a new diagnosis of cancer to have previously had some kind of cancer earlier in their lives.  So we might see a patient who has lung cancer who 15 years ago had a diagnosis of prostate cancer.  Well, for that lung cancer patient to go on a trial that has the typical inclusion and exclusion criteria that doesn't allow for this previous malignancy, they would be excluded even if they had been cured of that prostate cancer 15 years ago. 

We also see, and you mention what we call in the medical profession comorbidity.  So if someone's got cancer and they also have heart disease, they also have diabetes, high blood pressure, anything that affects the functioning of your normal organs, can also exclude people from participating in trials, and there are certain limits that we feel can be expanded and still allow the treatment to be given safely.  

So just about a year ago now we came out with a set of recommendations for how eligibility criteria can be modified to make clinical trials more inclusive.  And now just recently, I'm really pleased to say, the National Cancer Institute expanded their sort of template protocol document that many investigators follow to incorporate our recommendations, so now their standard protocol includes these broader inclusion and exclusion criteria.  And the FDA now is working on what they call guidance documents to advise commercial companies that are running clinical trials to do just the same thing.  So we are very optimistic now that we've got this ball rolling.  We're going to be removing these obstacles, and we're going to be able to have more inclusive and diverse population of patients who participate in cancer clinical trials.  

Andrew Schorr:
Great leadership.  I hope it works great, and we'll be happy to support you.  So, Mel and Cecelia, let's talk about the money part of it a little bit. So you were making trips to Houston, Mel, Cecelia was home with a 5?year?old, and so admittedly there may be hardships, financial hardships, being away from family if you have to go to a trial somewhere else, checking back.  What do you say to people when they say, well, I'm just going to go with the traditional stuff.  It's close to home.  In other words, if there can be programs that can help them it still takes courage, if you will.  So what would you say to people about investing in their life, if you will?  

Mel Mann:
Well, you know what the standard, what the current treatment is and the outcome of that, so if you want to have a different outcome then you have to try something new which is probably going to be a new drug.  So you have to weigh that with the cost and the travel.  Some people may not have the support, the caregiver support to go a long distance, so you have to take that into consideration.  As far as the Leukemia and Lymphoma Society they do have certain funds where they can help with travel pay, co?pay, insurance premiums, that could help alleviate some of it.  So there's a lot of stuff that's involved, and it's an individual decision.  

Andrew Schorr:
So we'll just make a comment, though.  So, many people have a church or synagogue, friends, neighbors even if they're living alone, but yet people are sometimes hesitant to ask for help.  And I would say speak up.  People do want to help you.  

Dr. Schilsky, let's talk about another reality of trials. There's a history certainly and some fear still in the black community of whether they were tested on, without their knowledge even, going back years and the general thought, you've heard it through your career, I'm sure, people say, well, I don't want to be a guinea pig for a couple reasons.  One is we don't know if it's going to work.  And second of all if there are different arms of a trial I don't know if I'm going—I'm going to go to all this trouble and expense, I don't know if I'm going to get the good stuff.  So maybe you could speak about that a little bit.  First of all, the fears of being experimented on, and then also about whether you will get what could be a breakthrough.  

Dr. Schilsky:
Yeah. Well, for sure, you know, there is this sort of sordid history of inappropriate experimentation on people, and clinical trials are a form of experimentation.  They are a form of research.  There's no doubt about that.  But clinical trials these days are highly regulated, overseen by independent groups that include patients and clinical experts that come together in committees called IRBs, Institutional Review Boards, and they evaluate on both the risks and the benefits to patients who participate in clinical trials.  

They make sure that the trial has an appropriate consent process associated with it, that it's explained in plain language to patients, so I think these days a lot of those concerns no longer exist.  And I hope that people can get beyond the history that led to some of those concerns.  The—sorry, I lost a train of thought on the rest of your question.  

Andrew Schorr:
The issue about are you going to get the good stuff.  

Dr. Schilsky:
Oh, yes.  So a couple points there.  One point I want to make clearly is that in most cases cancer clinical trials do not include a placebo or an inactive treatment.  That's not always the case, but it's true most of the time.  So patients are always going to get at least the standard of care treatment, and of course the standard of care is what is at that time known to be the best available treatment.  

The whole point of doing the research is to determine if the new thing is better, and, of course, we always hope it will be.  It's not always better, but sometimes it is, as in Mel's experience.  And I think this has to be clearly laid out to patients.  They have to clearly understand why the research is being done. In many trials nowadays even if the patient is assigned to get the standard of care treatment there still may be an option to get the new treatment at a later point.  So if the standard of care doesn't work many times there's still the opportunity to get the new treatment following the standard of care treatment.  

So the trial really boils down to not standard versus new but new versus standard followed by new.  So eventually everybody may have a chance to get the new treatment.  That's not always the case, but I think the key—my key take?home, in a sense, is that we're doing the research because we think and we hope the new treatment is better, but we have to do the research to prove that.  And everybody in a clinical trial I think can be assured that they're going to get, at the very least, the best available standard treatment.  

Andrew Schorr:
Mel, when you signed the papers to be in a trial, and you probably shared them with Cecelia, especially back in the late '90s and I participated in one trial in 2000 and another in 2011, there's a lot of paperwork, things in bold face written by lawyers.  I didn't always understand it.  What propelled you beyond that?  Was it just that, oh, my god, if I don't get something I'm going to die?  Or how did you two deal with the paperwork and feel comfortable signing on the dotted line?  

Mel Mann:
Well, I saw a lot of hope in the paperwork.  For example, one trial I was on was peginterferon, and I had been taking interferon every day, injecting myself, and I had to keep it refrigerated and when I travelled it made it difficult.  So with peg I can take one shot a week, so that would make the cancer journey easier.  It may not make me live longer, but it will improve my quality of life, so I saw my quality of life improving with that clinical trial.  And I looked at the paperwork, and I went through it, and I felt comfortable with it.  

Andrew Schorr:
And how about you, Cecelia?  I mean, your husband says, well, I'm going to be in a trial and I've got to sign all these papers.  Did you say at any point, wait a minute, that's scary?  

Cecelia Mann:
Well, no, I didn't.  I didn't because with Mel, he had three years to find a marrow match, and he was at the end of year two and no match in sight.  And so when he had the opportunity to go out to MD Anderson and be on a clinical trial or several, I was okay with that.  I was okay with that.  And I looked at it as actually being a blessing.  And it turned out to be, and we're grateful.  

But I would say to anyone else who is contemplating a trial and that person and their caregiver, their spouse, to just educate yourself, and get as much information as you can, ask as many questions as you can, but please don't just throw it away out of hand.  It's definitely worth considering.  

Andrew Schorr:
Dr. Schilsky, so we have more than 50 million people with a Hispanic background in the United States, and even if many people are speaking English they may speak Spanish at home.  And then when you are diagnosed with a cancer there's a whole new language of stuff that comes into play that even if you're fluent in English it may not be either what you easily understand or even aligns—what's being asked of you aligns with your cultural background.  Okay?  So how, beyond, let's say, the African?American community, when you look at the Hispanic community, how do we encourage participation there and get over some of these cultural or language nuances, if you will?  

Dr. Schilsky:
Yeah.  So it's much the same thing in the sense that the same information has to be conveyed but it may have different meaning and different interpretations in different ethnic and cultural groups.  Most clinical trials now will have a consent form that is fully translated into Spanish.  But, of course, there are many different languages on the globe.  When I was practicing at the University of Chicago for many years on the south side of Chicago, we had Polish?speaking people, we had Russian?speaking people, we had people—Chinese?speaking people.  

So the requirements actually are that there must be a consent form, at least some reversion of which is translated into the first language of the patient.  So if you're a native Spanish speaker, a native Chinese speaker, you have to have, be able to see a consent form written in that language, and generally speaking you have to have your native language interpreter present in the room to help you go through the consent form and respond to your questions.  And that person has to be someone who is independent from the research team so they can give you the straight answer and not be influenced by any member of the research team.  So I think all of that certainly helps.  

But, again, what helps a lot more is to have members of the care team who look like the patient.  So we have problems with diversity in our profession as well.  We have very few African?American oncologists.  We have more Spanish?speaking oncologists, but again we have few Asian oncologists.  So we need to do a better job of improving the diversity of our profession, improving the diversity of the care teams.  We need nursing staff and research staff and other people who work with our patients who represent them and gain their trust, who look like them, who talk their language.  And I think that will go a long way toward making people feel more comfortable about participating in clinical trials.  

Andrew Schorr:
I was at a conference last week and I heard some of the patient experience, people from different drug companies talking about how they were trying to simplify their forms, because I know in 2000 when I entered a Phase II trial there were all kinds of black boxes, you could die, you could this, everything in the kitchen sink was in it.  I'm still here, and I think because of the trial, and most of the side effects I didn't have or they were definitely handled extremely well.  

So right now, where are we, Dr. Schilsky, with participation? And why is it important?  In other words, in this age of personalized medicine why do we need more black people in certain trials?  Like, I know in multiple myeloma, one of the areas we cover, there's a higher incidence in the black population, right, but yet few black people are in the trials for myeloma drugs.  Or maybe there are differences with Asian populations or other populations. So is it that you can't really get a clear scientific answer on the differences?  Is that it?  

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. 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.

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