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How Can Clinical Trials Be Improved to Enhance the Patient Experience?

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Published on August 10, 2018

How can clinical trials be improved to enhance the patient experience? Patient Advocate Jim Omel discusses the design of clinical trials, how they could be modified to be more “patient-friendly”, and what a more patient-centered approach looks like. Leading expert Dr. Mike Thompson, from Aurora Research Institute, also explains what goes into creating a clinical trial, areas with room for change and potential issues, and the value of answering clinically meaningful questions. Additionally, Patient Power co-founder and CLL patient Andrew Schorr shares his experience participating in two clinical trials, the discovery of a second cancer and achieving a 17-year remission from an innovative treatment offered in a trial years before it was approved. 

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


Transcript | How Can Clinical Trials Be Improved to Enhance the Patient Experience?

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:

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?  

Jim Omel:

Well, I think one of the main things, Andrew, is that clinical trials tend to be designed to answer scientific questions.  I think what they should do is be patient friendly.  I think they should be designed to help patients.  If you ask any researcher, what is the purpose of the scientific trial, clinical trial, they will say, to answer a question.  If you ask a patient they'll think the purpose of the trial is to help patients.

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?  

Dr. Thompson:

I agree a lot with Jim. I think another word to put on it is pragmatic trials.  So I've been on a number of advisory committees, NCI investigator-initiated studies and pharma-directed studies, and when you have an advisory group with a bunch of academics they often think about the theories, and they think about what would be interesting to know.  

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 too 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.  

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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