Does Empowering Patients Mean “Better” Regulation of Drugs by FDA?
​[Posted on: Thursday, July 27, 2017]

This week FDA announced several initiatives aimed at greater patient engagement in regulatory processes that could greatly affect future approval decisions and other regulatory actions by the Agency. Involving patients in regulatory processes humanizes the regulatory process by educating consumers about how the FDA works. At the same time, it provides the FDA reviewers first-hand patients’ perspective regarding any decisions they make about a given regulated product. Patient groups have been actively involved in lobbying FDA since the early nineties when the AIDS activities successfully influenced many regulatory innovations at the Agency to get the first anti-HIV drugs approved. In the recent past, patient groups have played an increasingly important role in influencing approval decisions for drugs intended for cancers, rare diseases and unconventional indications. Most of the announcements this week have been in the works for several years. The patient advocates have been on Advisory Meetings since the beginning of such committees. The new Patient Engagement Advisory Committee contains patient advocates with long history of interacting with FDA for various diseases. The first meeting of the committee will be to discuss patient perspectives on clinical trials, not a new topic but a new way to share information that may be more interesting to the patients. Another announcement was about the expanded access program. FDA has always supported the expanded access program approving more than 99% of requests for expanded access to investigational drugs. However, expanded access does not guarantee that the company who owns the investigational product will provide the drug to the patient. About half of the times, companies refuse to provide drugs on compassionate basis due to the concerns about the negative effect on ongoing trials and/or expense of providing drugs at no cost to the patient. Hence the “Right to Try” laws were passed in a few states to encourage companies to part with expensive investigational drugs. FDA’s newest initiative, “The Expanded Access Navigator” (EAN) is an effort to consolidate all information regarding expanded access program in one place. It does not list the drugs available for expanded access since technically all drugs in clinical trials are available for expanded access but it gives links to the compassionate use web pages of several companies where one can find their policies about such donations. So, if you know the company who manufactures the investigational drug you want, and that company is listed on EAN, you can find the policies that the company has to provide its drugs under expanded access program. At this time, this is a very redundant website which patients and doctors may find to be tedious to use. Word of mouth and Google searches may still be the dominant easy sources of information to patients.  Patient engagement is useful but let’s make sure these efforts have real impact and not become merely a public relations exercise for an Agency beleaguered with bad public opinion. Patient engagement is the mantra of the times where companies have successfully used patient advocates to make a public case in support of new drugs. Patient perspectives are already heard by the Agency, although mostly in support of the companies it regulates. By having some patients on its side, perhaps the FDA is trying to balance this area of public opinion.