December 5 #LCSM Chat Topic – Is the System of Clinical Trials Fair For Patients?
By Dr. H. Jack West
IS THE SYSTEM OF CLINICAL TRIALS FAIR FOR PATIENTS? DO YOU BELIEVE IN IT?
I just watched the movie “Dallas Buyers Club”, about the challenges of getting new drugs for HIV/AIDS 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?
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.