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Does the Clinical Trial Process Need an Extreme Makeover?

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Published on July 6, 2018

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A number of patients have hesitations about entering clinical trials: Will I be a guinea pig? Will I as a patient be lost in the clinical trial system? Can I select my own arm in a trial? The questions are endless and, left unanswered, contribute to the barriers to trial enrollment. In this MythBusters program, we examine barriers patients may face and discuss the safety and quality of clinical trial care. Jim Omel, a retired-physician-turned-patient-advocate living with multiple myeloma, shares his experience in clinical trials and how he learned about his vulnerabilities as a cancer patient. Also joining the discussion is Dr. Mike Thompson, Medical Director for the Early Phase Cancer Research Program at the Aurora Research Institute and an active clinical researcher developing new treatments. Does the clinical trial process need a makeover? Watch now to find out more.

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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Transcript | Does the Clinical Trial Process Need an Extreme Makeover?

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.

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.

I want to thank the financial sponsors for this program who provided assistance to the Patient Empowerment Network.  They are Celgene Corporation, Astellas and Novartis. They have no editorial control, so what happens in the next hour is what we say, the questions you ask, what we hear from our experts who are joining us.  

If you have a question, send it in to [email protected]  Again, if you have a question, send it in to [email protected], and our wonderful producer Tamara will take a look at it, forward it to me, and as we can over the next hour we'll be discussing questions you have already sent in. And we'll have a very inspiring, I think, and provocative dialogue between our experts. 

So let's meet them.  I want to take you to Grand Island, Nebraska, where my dear friend Jim Omel is there.  He's a retired now family practice physician.  And, Jim, for years you've been a myeloma patient.  When were you diagnosed with myeloma, and what's happened along the way?  You're taking regular treatment now, I think, some treatment for the bone complications.  How are you doing, and when were you diagnosed?  

Jim Omel:

Oh, absolutely.  Without trials our treatment wouldn't change.  When I had a full evaluation at Arkansas they suggested that I join their trial, and I did, and at the end of that trial was a tandem transplant.  And I got to thinking and reading, and I didn't really want to get that extent of treatment.  I had a single transplant, and I dropped out of the trial.  And that's one of the things that I would certainly tell our listeners, that they can stop a trial at any time.  They're not bound to it.  

Ever since then, Andrew, I've had the good fortune of having fairly responsive myeloma, and when I had my treatments they responded to standard therapy.  I certainly would have rejoined another trial if necessary, but I was fortunate that it responded the way it did.  

Let's skip over to Milwaukee, Wisconsin, where we're joined by Dr. Mike Thomson, who is very involved in research, and Mike has been very involved in all sorts of programs related to education.  So, Mike, first of all, welcome to the program, and tell us a little bit about your involvement both locally in research and in education of other physicians nationwide and worldwide.  

I have been on the NCI Myeloma Steering Committee.  I'm currently on the NCI lymphoma steering committee.  I helped organize the ASCO 2016 meeting.  I was the Chair of Education.  As of June, I'm one of the editors for cancer.net around myeloma, so taking over from Paul Richardson who did that.  So I'll have about three years doing that and probably asking people like Jim for help to provide educational materials for people.  

And in the world of myeloma, I've created the MMSM or Multiple Myeloma Social Media hashtag to have Twitter chats, which I know some people don't think are the optimal form of communication, but it is a way to get information out from experts and some opportunity for patients to ask questions.  So I've been highly involved in social media, highly involved in the NCI and NCORP for increasing access to clinical trials in the community.  

And right now I am in the middle of an NCI designated clinical trial called EAA172 for multiple myeloma, which has gone through ECOG Executive Committee, the NCI Myeloma Steering Committee, and now we're discussing with the companies and with Ctap how to bring that forward.  And I think that's—one of the things is how much effort it takes to bring some of these trials from concept to activation.  

So whatever it is, ask your questions, [email protected]  I'm just going to share a little personal story for a second, because I'm very passionate about it, and I wanted to mention it.  And this is part of our Clinical Trials MythBusters series, and we have previous programs on Patient Power with lung cancer experts, experts in other conditions about the clinical trial process, so look that up on patientpower.info. There will be a replay of today's program and also a downloadable guide with highlights that you can share, talk about it with your doctor, with other patients, with people you know and for your review.  Okay.  

So now my own story.  I was diagnosed with chronic lymphocytic leukemia, the most common adult leukemia, in 1996—terrified, had no idea what it was.  Didn't know anything about what a trial was, didn't know what the treatments were.  Quite frankly, thought I'd be dead like within a week.  I didn't know.  

And so you start getting educated, and eventually that led to me connecting with academic medicine specialists and ultimately suggestion at the appropriate time of being in a Phase II clinical trial.  I didn't know what the phases were, we may talk about that along the way, and it was 2,000 miles from my house.  So I traveled a number of times to be in that trial, and I had my local oncologist collaborating on that.  

And the end result was I had a 17-year remission. I had treatment again for chronic lymphocytic leukemia.  It wasn't until last year, 17 years.  And I got the combination of medicines 10 years before that combination was approved. So I'm a believer.  

The second thing I'd say about trials was I was in a second trial along the way, and I had deep vein thrombosis, blockages in the veins in my legs, for a blood thinner trial. And by being observed in that trial, that led to them discovering a second cancer which was at work related to those clots, myelofibrosis, and I was observed, so I liked the attention.  It had nothing to do with what they were testing.  It had to do with the observation you get.  

So, again, I love the attention of being in a trial.  It may give you access to tomorrow's medicine today, but there are things that may be broken. So, Jim, let's start with that. Jim, what has been some of the frustration points for you the way the process has been today?  

The—it may seem like a minor point, but it's not. Patients need to be the center of them. We need to help patients understand what their contribution is to a trial.  For instance, hardly ever does a patient hear how their outcome, what they did during a trial improved the final outcome of a trial.  The patient needs to be centered.  

If we get the trial to a point where some of the questions are pretty obviously answered, rather than continuing to recruit patients just to be statistically valid, I think trials should close sooner. I think they should be more focused on getting patient care without necessarily the scientific question.  I'm not a radical.  I'm certainly a fan of trials.  We wouldn't be where we're at without trials, but I think they should just become more patient-centered and patient-friendly. 

Andrew Schorr:

Okay.  Now, Mike, Dr. Thomson, so we know we can't have new drugs approved by the FDA unless there are trials, Phase I, Phase II for sure, and often, typically, Phase III and sometimes even monitoring after a drug has been approved. I think you call those Phase IV trials. But from where you sit having been around this a long time what are some of your frustrations?  What would you like to see be improved?  

And increasingly both the NCI and others are getting not only patients but community physicians who will say I don't really care about this question here.  And we don't think that it will fly and won't accrue, and we know a lot of trials don't complete accrual, so therefore patients are wasted, if you will, because we won't have the information, we won't be able to answer questions.  So I agree. 

There are so many things get to involved it's hard to break them all down, but part of the issue is answering a clinically meaningful question.  I think the meaning should be patient-centered.  Within those questions you can ask scientific questions that are imbedded in what are sometimes called secondary imports or co?relative studies.  But I just last week was talking to some pharmaceutical leaders, and I said, you have to design a trial to answer a question people care about, and that's patients and physicians.  

Because sometimes the trials are designed to get FDA approval, and they're comparator arm if it's a randomized study, is an arm that we don't think is the current standard of care, and we have to do them in countries where they don't have as many therapies and they don't have as much access, so they'll get them done. But then when they're approved in the U.S. we don't know what to do with the trial, because it's not a question we're asking. So that's important.  And I think if more studies are done not to get FDA approval but to go on pathways and to ask, what are the clinical branch points for decision?making, I think that's when you'll start getting good trials.  

There are a number of other issues around the pragmatics.  So there's this NCI Match study, tons of people screened, very few people on the matched drugs, and they switched over to a strategy more like an ASCO TAPUR, where they waited for people that already had testing and then the people that had already kind of pre?screened couldn't get evaluated for the study. And many, many more people went on study.  The imaging and other things in the middle were not as rigorous as a usual clinical trial. It rolled quickly, and I think the point is you're looking for big end points.  

Where you have to sort of go back to the classical, randomized, Phase III large study is when you're trying to make incremental improvements, so, for instance, breast cancer where the cure rate or progression?free survival rate may be in the 90-something percentile rate, or even CML or other things where we're doing so well you'd need a lot of patients and probably a standard design.  

But in many other areas you can do a variety of different techniques—Bayesian analysis, continuous reassessment models. And one thing Jim mentioned was stopping for futility or if there's an obvious benefit, and that is done but probably not as often as it should be.  And the designs using what are called interim analyses or futility analysis with data safety monitoring boards or DSMBs, probably could be more robust. There could be more of them.  I think people are afraid to do them, because they do slow the trial down, they slow accrual, and that has to do with stuff both within the trial as well as extrinsic to it.  So there are a number of barriers and issues, but I think Jim's pinpointed them as well.  

So, say, I understand the standard therapy, and you're testing it maybe against that, but I want to get the good stuff, because I'm really hopeful.  I want to be a believer.  So could you just describe where we are with randomization, because that's a concern people have? 

And when it comes to randomization, Andrew, there's many, there are many trials that absolutely lack equipoise.  And I'm afraid that scientists often use equipoise. 

Let me give you an example.  There was a trial in which patients had the choice of three oral drugs in one arm versus a stem cell transplant in another arm. Now, think about that.  Think of the insurance ramifications.  Think of the fact that it takes almost a year to really totally recover from a stem cell transplant, versus taking three oral drugs. How can anyone say that there's equipoise in a trial like that?  So how can you pattern your life with the flip of the coin or a computer randomizing you into one of those arms?  

Where are we with more trials being available or having an aspect of it, like testing, closer to home?  

There are exceptions of course.  We're doing a surgical trial or a radiation trial that has to be at one site or sometimes a Phase I trial with just a lot of blood monitoring, very intensive, they can only be done at a few sites. But in general I completely agree that we should try to have the drugs available to people in the community they live in, because that's where their social networks are, right? So that's where their family is. They can stay at home.  They don't have to just go into a hotel.  They don't have to pay for travel, and I think it's better for everyone.  

And for companies, I've been trying to tell them that it's more generalizable to the reality of where cancer patients are. So 85 percent of cancer patients are in the community setting and are treated there, and drugs should be accessible to them there.  So, you know, both the using the CCOP mechanism or NCCCP, and now we have the NCI Community and College Research Program or NCORP.  The whole idea is to increase that access to community sites. So this has been going on a long time. 

I think there were budget cuts, and so the U.S. and the way we've established our cancer budgets has been to decrease access at least NCI trials and usually need some of those NCI trials to support the research infrastructure to do other studies.  So I think part of that, you know, a lot of these things you follow the money. And if there was more money for community research sites, you could hire more research staff to get these things done.  

But I think we need to get them done in the community, because we know if you do early phase studies and they look promising in highly selective patients, then when you expand them and put them in the community you go from efficacy to effectiveness, and the effectiveness isn't there because the patients are different.  So there are all these things with real?world data and comparative effectiveness research at ASCO's cancer link trying to get at some of that not on study to just try to get the data.  

But we need to have access to people, and the way to make drugs cheaper, make them develop faster and answer more questions, both scientific and patient?oriented, is to get more people on trial.  There's a big example for immunotherapy drugs where there are so many immunotherapy drugs and trials there are not enough patients to get it done.  So we're going to enrolling in trials which don't complete, or we're not going to be able to answer these questions, so it's going to stall and move it out the process of moving faster.  In myeloma, we move very fast, but we need to do this in other areas too.  

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.