Q&A - August 04, 2025
Dear FDA Matters subscribers–—“Q&A Friday” is now “Q&A Monday,” a subscriber-first feature of FDA Matters. It is on the record, so subscribers (including reporters) can use and circulate the Q&A immediately upon receipt.
Subscribers are encouraged to submit questions about any aspect of FDA’s mission, responsibilities, programs, and policies to: sgrossman@fdamatters.com. I will try to respond to questions in a way that makes all of us a little smarter about FDA.
Have a great week, Steven
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Recent columns:
Needed: A National Agenda for Advancing Therapies for Chronic Progressive Diseases (July 31, 2025). This is a particularly important column. Please read it and forward it to others in your network.
Food User Fees: The Good, The Bad...and the Possibilities (July 14, 2025)
Recent Linked-In and Other Exchanges:
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This week’s Q&A Monday features these questions:
· What is FDA’s most pressing top-down need over the next few months?
· Does USDA’s plans to move more staff to regional centers have any consequences for FDA?
· Why did you choose to write about the need for better understanding and tools to evaluate chronic progressive diseases that are severely disabling or fatal?
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Q: What is FDA’s most pressing top-down need over the next few months?
A: Stability of leadership is the agency’s most pressing top-down need. (Note: The most pressing bottom-up need is to decrease staff turn-over, improve morale, and re-establish efficient workflow. Stability of leadership would help.)
On several occasions I have talked about how Commissioner Makary (and then subsequently Dr. Prasad) was an “outside critic who would have to go through a transition to take on the role of inside government decisionmaker.” Commissioner Makary is still in the middle of this--testing different approaches to problem-solving and leadership. That’s understandable and not meant as a criticism.
Dr. Prasad didn’t have the months to think about his new role--as Dr. Makary did; nor was it a good idea to give him even more responsibilities (chief medical and scientific officer) upon arrival. He apparently overturned several staff recommendations despite having criticized his predecessor, Peter Marks, for doing it at all. Likewise, as I examined in my last column (Needed: A National Agenda for Advancing Therapies for Chronic Progressive Diseases), he made several missteps with his handling of Sarepta. I would like to think that reflected his inexperience in the role rather than any hostility toward cell and gene therapy.
At this point, the FDA’s top leadership is thin, especially given the agency’s span of responsibilities and how many members of the team are new to their roles and new to working within the government.
One way to stabilize the leadership situation quickly would be to fill some of the vacant senior positions with individuals already at the agency and with experience in running it. Not acting appointments, but rather permanent ones. This should help stabilize things and, also, should help improve morale among career civil servants.
Q: Does USDA’s plans to move more staff to regional centers have any consequences for FDA?
A: I don’t think so, but it is a matter of concern and something to be watched (e.g., traditionally Congress has played a role and is, at least momentarily, inserting itself into this one). I have been told that 90% of USDA staff are already outside of the DC area. So, the alleged rationale of moving the agency closer to its constituents seems bogus.
Further, this type of action is often undertaken with the hope of shrinking the workforce because a lot of people will resign or retire instead of moving. About 15,000 USDA employees have already left (early retirements, RIF’s, etc.), so it would seem that forcing a few more departures will not have much of an impact.
I am curious if any FDA Matters subscribers can offer more insight into this topic.
Q: Why did you choose to write about the need for better understanding and tools to evaluate chronic progressive diseases that are severely disabling or fatal?
A: For those that missed it: Needed: A National Agenda for Advancing Therapies for Chronic Progressive Diseases. It is one of my most important columns.
FDA’s support has been an essential feature of forty years of progress in stimulating new understanding of rare diseases and the approval of countless drugs to treat and sometimes cure rare diseases.
Yet, within the agency there is a constant tug-of-war about how to address the subset of patients with progressive debilitating and fatal diseases. Compassion and exceptions to the rules are doled out unevenly and, I suspect, inconsistently. Some sense of the agency’s approach; some guidance to patients and industry; and clarity on how, as a class, treatments are evaluated for these diseases. It doesn’t seem too much to ask, even if it requires confronting some difficult issues.
The cause of patients with debilitating and fatal diseases has been taken up by libertarian-oriented organizations that see it as way to move toward “anything goes” drug approvals. FDA needs to have a strong, pro-patient view on how to help patients with these mostly rare diseases without opening the floodgates for a free-for-all.