Mike's Blog

The FDA is unique in that although the agency is under HHS, its budget is contained in the Agriculture appropriations bill (bear in mind that ‘food’ is the ‘F’ in their name). In addition, during government shutdowns, certain FDA activities can continue to operate (like the Postal Service) in part because the agency receives money from industry for their reviews due to the Prescription Drug User Fee Act (aka PDUFA). However, to continue in this manner, PDUFA needs to be reauthorized by Congress before its current authority expires on September 30, 2027. The FDA has been holding meetings with relevant stakeholders since 2025. As expected, there is no shortage of suggestions and stakeholders are not shy about voicing opinions. In this vein, the release of Roadmap for FDA Modernization 12 Bipartisan Ideas to Help Patients and Strengthen American Leadership makes for interesting reading, particularly because of the overlap in its recommendations with the CTSA program and NCATS. The document was released by the Stanford Mussallem Center for Biodesign and is well-written with practical recommendations.  
 

Many of the recommendations address drug development and regulatory bottlenecks, especially preclinical IND-enabling activities (such as what we heard from FDA at the recent CTSA annual meeting) as well as chemistry and manufacturing controls. One recommendation specifically addresses the qualification process of new approach methodologies (NAMs), specifically human tissue chips and organoids. Flexibility for evolving endpoints (due to changing science) as well as the use of natural history studies as external controls for rare disease trials are discussed. One novel innovation is the suggestion to establish accredited centers for personalized and individualized therapies. The idea is that since these types of therapies are less attractive to the commercial sector, a ‘qualified center’ could utilize validated therapeutic platforms (such as a gene editing mechanism that would target a patient’s unique gene sequence). With regard to platform technologies, another suggestion is to allow developers to reuse data on shared platforms as opposed to the current situation where competitors as well as nonprofits and academic centers are prohibited from accessing what is considered proprietary information.  
 

Another idea is to develop a process for the FDA to share scientific and safety issues beyond the specific sponsor that identified the issue with others working in the same pharmacological space. Some long-standing suggestions are mentioned such as more routine rolling reviews, enhanced consistency with complete response letters, and a call to rely on trusted international peer regulators’ reviews to avoid unnecessary additional clinical trials. Finally, there is the suggestion to make the rare pediatric disease priority review vouchers permanent (currently those vouchers sunset in 2029).
 

There is much to digest in this report, but the bottom line is that if any of these recommendations are considered as part of the PDUFA reauthorization, then academic medical centers and CTSAs will have the opportunity to play a larger role in therapeutic development efforts; many of the capabilities needed to operate in that space already reside within the CTSA institutions and partnerships, especially as we (hopefully) transition to an era of more personalized interventions.  
 

What I’m reading: Out of Pocket, Out of Reach, a STAT series (up to part 5 as of this writing). This series is a no-holds-barred, unabashedly critical review of the evolving unaffordability of health care. Drug prices and hospital costs are major drivers, but the longer-term implications go beyond health care itself and the growing volume of medical bankruptcies. Specifically, employers as the source of health care insurance (which some regard as embedded in an appendix with Moses coming down from the mount) are reconsidering even offering health care insurance at all. Alternative approaches for accessing and paying for health care may gradually be sought by the business community with significant implications for state and national politics along with all of us as both providers and recipients of health care services.  
 

And it happened all the time that the compromise between two perfectly rational alternatives was something that made no sense at all.
-Neal Stephenson, Anathem