Not Your Father's FDA

“This is not your father’s FDA.” That was the conclusion I drew from NORD’s Rare Diseases and Orphan Products Breakthrough Summit, a two-day conference that included presentations by Commissioner Gottlieb, and the directors of CDER, CBER, and CDRH, as well as by leaders at the review division level.

I was also able to chat with some patient groups and some companies about their interactions with FDA.Everyone I spoke with was impressed by the agency’s commitment to science-based decision making and its enthusiastic embrace of medical progress. Every FDA speaker touched on at least one of these themes: collaboration, urgency, transparency, inclusion, and adaptability. To pick just one example: it was clear that patient-focused drug development is alive and not just a matter of the FDA “going through the motions.” The agency is “all in” on integrating feedback from patients on endpoints and methodologies, as well as on benefits and risks.Regarding the agency’s growing openness and willingness to evolve its positions, I have been advising clients that FDA will discuss any question with companies and patient groups, as long as it is presented to the agency respectfully, is based on solid science, and reflects serious preparation to maximize the value of the interaction. Both patient groups and companies confirmed this. Some told me of instances where the FDA came to them and said: based on results so far, let’s see if we can work together to accelerate the timeframe so patients can benefit sooner. This is not to say that patient groups and companies are always treated as well as they might like; but the positive responses far outweighed the negative ones, and far more so than in the past.We are at a point where medical progress is breathtaking and FDA sees the opportunity, as well as the challenge, to move forward on therapies that were unthinkable a decade ago. CBER Director, Dr. Peter Marks, observed that “gene therapy is finally the reality in the United States.” Another FDA speaker spoke of the agency’s role as a partner in the acceleration of knowledge. Talking to the patients and companies, it was clear that things are speeding up in a very promising way and FDA’s role is central.Because the conference was about medical products and treatments for diseases and conditions, I cannot report directly on whether the same spirit can be found at CFSAN and DVM, as was evident for CDER, CBER, and CDRH. However, I believe it is. The process of assuring a safe food supply has been completely upended from a decade ago and FDA is now fully aligned on the great shift from reactive responses to prevention of problems. The commitment and enthusiasm is real. With regard to CVM, Ladd Wiley, the Alliance’s executive director, and I have been briefed a number of times. We always come back amazed at the exciting and underappreciated work being done by the dedicated staff at the center.There was nothing wrong with “your father’s FDA,” but the agency has done a lot of growing since then. We usually focus on the impact of program expansions, but I realize now we should be saying more about the agency’s maturation in both process and attitude. It reflects extraordinary progress in becoming a 21st Century regulatory agency.

Editorial note: The Analysis and Commentary Section is written by Steven Grossman, Deputy Executive Director of the Alliance for a Stronger FDA.