Q&A - June 26, 2025
Dear FDA Matters subscribers–
Introducing Q&A Friday, a new feature for subscribers.
Each Friday’s Q&A will go to subscribers first, then it will be posted on the website about a week later. Like all FDA Matters analysis (including everything on the website) it will be on the record, so subscribers (including reporters) can use and circulate the Q&A immediately.
To maximize the value of this effort, please send your toughest 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 the agency.
Steven
Q: Do the efforts to dramatically shorten the approval process risk a thalidomide-type situation with no place for a Frances Kelsey to express concerns?
A: There are many FDA programs that work to shorten biopharmaceutical and medtech review times. Commissioner Makary has just announced another one, the National Priorities Voucher Initiative (NPV), that aims to create a significantly faster pathway by focusing on rolling reviews, enhanced communications with the agency, and rapid implementation of AI systems.
Usually, FDA, companies, and the public are aware--well in advance and long before the NDA filing--that the FDA’s review for approval is likely to generate deeper discussions. The change to a two-month accelerated review would still have to account for earlier, as well as contemporary public critiques from study investigators, review boards, clinicians, investment analysts, mandatory SEC disclosures, and FDA reviewers.
For new chemical entities, there should be additional critique from an FDA advisory committee, although it is unclear how advisory committee review fits into the timeframes for the recently announced NPV initiative.
We only know in retrospect that Dr. Kelsey’s was correct in blocking the approval of thalidomide in 1960. So, I can’t say that a latter-day “Francis Kelsey” wouldn’t “win some and lose some” when concerns are being raised prospectively. However, unlike 1960[1], there would be plenty of opportunities for her to voice her concerns publicly and stimulate additional evaluation of the data.
For additional relevant information about FDA’s decisionmaking process, I would recommend the last section (“FDA Is Not So Easy to Influence”) in my earlier column here.
Q: What is the starting point of the next round of negotiations on medical products user fees?[2]
A: The current user fee agreements will expire on September 30, 2027. Hearings start this summer. That gives negotiators about 15 months to reach agreements, which will then be debated by Congress in early 2027.
Based on HHS Secretary Kennedy’s persistent criticisms of FDA/industry relationships, echoed to a degree by Commissioner Makary, I have assumed the Administration will insist on a different structure that it will claim does a better job of insulating the FDA from industry influence.
Commissioner Makary seems to have softened his stance recently, talking a lot about working with industry and supporting a budget request that implies continued user fees. This may be an acknowledgement that FDA cannot sustain itself on appropriations alone. Also, as Commissioner, he may have a greater appreciation of why medical advances are dependent on the cooperation of the agency, patients, and industry.
Negotiators will need to account for the strength and proven durability of the current user fee structure. Improvements are possible, but some new approaches may look good at first glance but may not prove better in practice.
Old and proven structures, new sensitivities....it’s not yet clear how that will play out.
Q: With regard to the new National Priorities Voucher initiative (NPV), you liked some aspects but “worry about diluting the focus on unmet medical needs.” Why does this concern you?
A: Existing accelerated review programs are gated by a requirement that the products being reviewed address an unmet medical need. It is less clear whether that would be the case under the NPV. As described by FDA:
“The FDA Commissioner will use specific criteria to make the vouchers available to companies that are aligned with the national health priorities of:
Addressing a health crisis in the U.S.
Delivering more innovative cures for the American people.
Addressing unmet public health needs.
Increasing domestic drug manufacturing as a national security issue.”
The program is being created with noble intent. Nonetheless, it seems to me that a speedy FDA review is not an appropriate lever for rewarding activities that are not related to safety, efficacy, or unmet medical need.
Reshoring manufacturing plants or committing to limit price increases are worthwhile national priorities, but neither has anything to do with approvals. I might soften on this when more details become available on the NPV, but at least initially I find it troubling.
Note: Thanks to everyone who submitted this week’s questions.
HPS Group, my consulting firm, is accepting new clients and projects. While FDA Matters will always be a work in progress (new columns and new features coming), I can now turn my attention to the re-launch of my consulting firm, HPS Group.
Let’s chat about whether we can help...if I can’t offer you what you need, I have worked with a vast number of people I can recommend. You can reach me at sgrossman@hpsgroup.com or at 301-257-9660.
Have a great weekend,
Steven
Tobacco user fees are excluded because they are permanent, i.e., not re-negotiated every five years and because the industry/FDA relationship in tobacco is completely different than in medical products.