Scientific Data Versus Anecdotes: What Role Should They Play in FDA Decisions
(Thursday, June 30, 2022)
In an ideal world, regulatory policies and laws are based on unquestionable data from high quality scientific research but should there be other criteria or a standard for weighing unconventional data when the scientific data is not sufficient? There are numerous instances where claims of benefits by patient were not supported by the clinical trial data, putting FDA in a tough situation of deciding to approve a product based on perception of benefit rather than the measured efficacy from clinical trials. In many ways, one can argue that surrogate endpoints create a perception of benefit that may not lead to clinical efficacy. This argument has been made by the opponents for the accelerated approval pathway which allows products to be approved based on improvements in a surrogate marker without clear evidence of a direct clinical benefit. The accelerated approval pathway was successfully used initially for developing treatments for HIV in the 90’s, and then for numerous oncology drugs in the last 20+ years. Almost all the drugs approved under the accelerated approval pathway ended up yielding clinical benefits in the long run establishing a scientific standard for approval based on these endpoints. However, there have been cases recently where even the surrogate endpoints were not clear, but the strong claims of benefits made by the patients, which could be argued to be anecdotal, pushed FDA to approve the products anyway. This was the case of DMD drugs from Sarepta and Alzheimer’s drug by Biogen, and now may be true for the Amylyx’s ALS drug as well. And this is not the end and this needs to be formalized to avoid the kind of speculation and uncertainty that surrounded each of the subjective decisions made by the FDA. Can the patient perspectives be treated as real world data with acceptable deficiencies expected there of? There is a strong movement to use real world data (RWD) and evidence based on it for regulatory purposes. True “Real World Data” is full of anecdotes and conjectures. FDA has been open to using RWD and has authored a few guidance documents on its expectations. There is also a significant movement to create and use endpoints based on patient opinion of benefit, the Patient Focused Drug Development (PFDD), which intends to define rules for using information that 20 years ago would have been considered anecdotal. As the boundaries readjust between conventional scientific data and “new forms of data”, we need creative strategies to use both kinds of data together to foster well-defined and consistent policies enacted via transparent regulations based on them. There are a lot of dots to connect. It is happening, slowly but surely.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC