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Dr West

Is the system of clinical trials fair for patients? Do you believe in it?

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I just watched the movie “Dallas Buyers Club”, about the challenges of getting new drugs for HIV/AIRS just as the first effective treatments were being identified in early clinical trials. It portrays the DEA, FDA, and medical establishment as essentially obstructing the process of patients getting access to life-sustaining treatments, and it led me to wonder whether patients and caregivers feel that the system of novel agents being tested in clinical trials is fair or whether the medical establishment today seems to be protecting people from themselves in unwanted ways.  The movie takes its title from a legal workaround that facilitated people buying non-FDA approved drugs from outside sources outside of clinical trials, learning on the fly in a way that seems far more nimble than the sclerotic, formal trial system being practiced by the doctors.

In truth, the movie doesn’t make physicians clearly evil, as they debate the merits of placebo-controlled trials in order to determine whether treatments are safe and effective.  AZT is shown as having potential efficacy but also leading to severe, dangerous side effects in early testing as a single agent at a higher dose than used in later combinations, though I think the overall sense from the movie is that the people pursuing treatments based on collective experience (pre-social media sharing, mind you) were outpacing the pace of progress in the clinical trials system, with its ties to “big pharma” corporations that are suggested as pulling the strings of the puppets in the FDA. 

As someone who runs clinical trials and considers them the building blocks of everything that has come to be defined as our best treatments for cancer, I needed to step back to ask whether people see the clinical trial system as obstructionist. We have the same debates today about trial design and how to provide access to promising drugs, especially in patients with a poor prognosis and few or no compelling standard treatment options.  We want to offer good treatments but not unwittingly harm patients by giving treatments that may be toxic but not formally evaluating outcomes and recognizing the problems as early as possible.  The movie also includes patients on placebo-controlled trials, banding today, pooling their medications and splitting them to provide the best chance that everyone gets some medication, short-circuiting the trials process and ability to determine whether the group receiving study drug did better or worse than those on the placebo.  Of course, placebos remain a dreaded and maligned element of the clinical trials system, even though they sometimes help identify when patients are better off NOT receiving the study drug.

So I ask you the following questions:

1) Do you believe in the clinical trials system? Do you believe that, in the big picture, it serves its goal of identifying drugs that are safe and beneficial? 

2) Is it too restrictive, either in terms of which patients are permitted to enroll, where trials are available, or both?

3) Do you have clear suggestions of how the system should be changed, bearing in mind that our goals need to be to protect patients from insufficiently tested and potentially harmful treatments as well as to deliver new beneficial ones?

4) If treatments are potentially available “off-protocol”, such as a drug for kidney cancer being written for in a group of patients with lung cancer, do patients and caregivers recognize an incentive to conduct the study to clarify the value of the treatment, or would you favor getting the drug by any means necessary and circumventing the trials system?

5) Do you see ways in which social media, efforts like PatientsLikeMe.com and other self-aggregating patient groups might accelerate research by working along with physician scientists and the pharma/biotech industry trying to test novel agents?

We’re going to cover these topics at an upcoming hour long “#LCSM tweet chat” on Thursday, 12/5 at 8 PM Eastern, 5 PM Pacific. I hope you’ll be inclined to join us, or just leave your thoughts here. 

I would say that these issues are clearly very timely, but the movie I just saw reminds me that many of these central questions explore the same themes that  the medical/patient communities have been struggling with for decades.  


5 Responses to Is the system of clinical trials fair for patients? Do you believe in it?

  • costica says:

    I found extremely hard to get in contact with someone running the trial. Phone? Useless, you talk with a call-centre operator who will simply repeat, as many times as needed: “please refer to your doctor”. If your doctor has no idea about any trial, too bad. On many occasions the information on clinicaltrials.gov is simply wrong. Right now for instance there is a trial which appears as “enrolling” in a country, while the trial has been long suspended in that country.

    As unfair as it may seem at first, I still believe that the clinical trials are the only way of making a difference between effective drugs and myths/hoaxes.

    Yes, I find it too restrictive. Why wouldn’t be possible for a patient in a country to participate in a trial run by doctors in another country without traveling? It’s easier to send drugs by courier rather than requiring a patient to travel in another country every three weeks. Of course, this involves cooperation between the doctors in the two countries. I’m speaking here about phase-3 trials, when there is no issue about establishing the optimal dose.

    I definitely believe that social media/internet will be a way through which patients will find trials and those who run trials will find patients, especially when we talk about drugs targeted on particular mutations. It would make sense for me that, before one opens a trial in some location, he will make sure first that there will be patients willing to enroll. And he could do that quite inexpensively.

  • neilb says:

    I don’t know if I’ll be able to make it to the session, but I am in the process of (finally!) completing scholarly work that proposes major changes to the clinical trials system. I come at this from the unique position of being both a policy analyst and trials participant. It is my hope to get feedback here and in other places when this draft is available (in 2-3 weeks). This comment is probably designed to put a little pressure on myself to finish the paper!–Neil

  • Ann says:

    Those are good questions, and I am looking forward to seeing responses. Historical documents like “Dallas Buyers Club” remind us of a time before patients were empowered to participate actively and encouraged (Cancer Grace being a perfect example) to educate themselves in order to increase their odds. I lived through those times, and was deeply involved in advocating for people with AIDS in the 80’s. Before protease inhibitors, before “the cocktail” that has completely changed the paradigm, all we could do as caregivers and patients was watch the sand run through the hourglass. The administrative overlay of the traditional trial structure, while perhaps understandable from a research standpoint, was very hard to navigate and endure as thousands of our friends and loved ones died so quickly and so miserably. The cliche, “desperate times call for desperate measures” applied to us in those days, and while we were certainly shrill and sometimes wrong, we continued to ask THE question and receive little response: “DO THESE DRUGS PROLONG LIVES?” So we went looking for answers ourselves, and in the process found that we could learn the science, talk the language, and apply the knowledge to try to solve what seemed an impossible problem. The historical perspective is important, because those efforts adjusted forever the wall that once existed between researcher and patient.

  • ssflxl says:

    I have been looking for clinical trials for a while and yes, they are very restrictive. you literally have to be healthy, without any other problems, before you can participate. that is a big concern of mine. if you get too sick, you won’t get into a trial, even if it’s a local one.
    Most of us don’t get any help looking for a trial – if there isn’t one in your hospital, then you are out of luck and you need to do all the work. Clinicaltrials.gov is not always up to date – someone has to update the info!!
    I hope for the day when we can do easy videovisits. why can’t we get the trial drug in our local hospital and follow the exact protocol – why must we travel to the trial site? that is just due to paperwork
    the amount of solid evidence you need seems to be a very high bar. I think in oncology, you have some desperate patients who have tried many lines of treatment already, so you do need to lower the bar a little.
    the time it takes for trials to the time the drug is approved is a very long time. I wonder if there is anyway that Phase 1,2 and 3 can be all done at once or pretty consecutively, so time is not wasted.
    All in all, getting into a trial requires hard work on the part of patient or family. you really have to advocate for yourself. needless to say, not all oncologist are familiar with the latest so the patient must do all the lifting.

    ssflxl

  • karenb says:

    In my opinion, communication is the biggest obstacle in getting into the clinical trials. I have been trying since my first visit to Moffitt on October 13th. I have stage 4 nsclc with mets to the spine & ribs so far. The first trial was for Med14736. I had the lung biopsy, brain MRI, CAT scan, etc done. Then the wait began. The lung biopsy came back inconclusive so they tried another sample they had taken. This also came back inconclusive so I was rejected. They want PDL-1 positive people. Weeks go by without being able to talk to people and finally I was told I was a candidate for another trial, a combination of Tremelimumab and MEDI4736. Because they let so many weeks go by I now have to do another lung biopsy, brain MRI,CAT scan, etc. I realize each company sponsoring the drug wants their own but it would seem that most of this testing previously could of been used instead of starting over. Waiting is the enemy and communication would greatly relive all the horrible anxiety one feels daily.

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