Sen. Cassidy’s FDA Report
Senator Cassidy, the Chair of the HELP Committee, released a report titled “Building the FDA of the Future”. In it, he outlines improvements to FDA to ensure that safe and effective products get to market, and ideally patients, faster. Senator Cassidy, in his opening, echoes something I had the pleasure of hearing Dr. Hamburg say repeatedly, when I worked with her: It’s not innovation if it doesn’t work.
TLDR: Some good ideas are outlined but if you’re reading this, you probably already know everything in the report.
Evidence Generation
The primary way sponsors generate evidence to support the claims regarding their products is through clinical trials. This is particularly important for novel drugs and biologics, and less important for generics and low risk devices.
Traditionally, FDA has required two randomized controlled clinical trials to prove a drug/biologic works. This is not law, and FDA has the authority to be flexible on the types of data required. Just this week, FDA announced, via a NEJM article, that it would stop requiring two studies for any and all applications – one RCT will suffice.
However, other trial designs, like using historical controls, can be used, particularly in small populations. The industry, including FDA and NIH, have not fully embraced decentralized clinical trials, single IRBs, simplified contracts and other administrative practices that could streamline trials. Senator Cassidy rightly points out the need for these. In leveraging both NAMs, as recommended in the report, and digital tools, FDA must make clear what data are acceptable.
The report also addresses the fact that Phase 1 trials are increasingly moving overseas. The report suggests a pilot program for a notification process for Phase 1 trials. However, this notification process already exists. It is unclear why it isn’t working; my advice to anyone who asks is that a GAO study could help us understand the problems with implementation.
Rare Diseases
Developing products for rare diseases is difficult for a number of reasons, not the least of which is the inherently small population on which to test treatments. The report points out the seriousness of clinical holds, and recommends that they be used judiciously. The report also discusses the plausible mechanism pathway and recommends that Congress can do more to support these efforts.
Of note, on Monday (Feb 23) HHS is expected to announce draft guidance on the plausible mechanism pathway.
Medical Devices
The report notes the innovation in medical device development, and raises concerns about the data required for some sponsors submitting a de novo. The report also outlines the challenges that come with iterative devices, particularly those that use/are software – as anyone with a computer or phone knows, software is updated regularly. There is no well-tailored path for approval of these products, however the report does not recommend one, nor does it suggest that the Senator is drafting a proposal. It does give a nod to the pre-cert program which was not a success, and wrongly implies that codifying the pathway would ensure success.
Lastly, as it pertains to medical products, the report raises concerns about regulating devices that use AI and clinical decision support software being overly regulated. The latter was addressed in the 21st Century Cures Act, which the report suggests is not being implemented appropriately; the former is addressed only at the highest levels.
Conclusion
The report will be useful for those seeking to address the identified problems, however, there are few specifics and no legislative language. Stakeholders, including Congress, have their work cut out for them.