Editor’s Note: The following is testimony delivered by Lowell Schiller to the U.S. Senate Committee on Health, Education, Labor and Pensions on Oct. 29, 2025. More information about the hearing can be found here.
Key Points
To realize the promise of new medical technologies and product development tools, the Food and Drug Administration must maintain a modern regulatory ecosystem that enables innovative products to reach patients efficiently without compromising scientific rigor. FDA has the opportunity to modernize its approaches in key respects:
- Facilitating more use of advanced evidence generation techniques, like innovative trial designs and sophisticated uses of real-world data—for example, by addressing issues around data formatting and FDA’s review of patient data, expanding the use of external controls, and updating regulations to better accommodate decentralized and community-based trials.
- Advancing innovation for rare diseases, which together affect an estimated 30 million Americans, by standardizing review approaches and facilitating tools that enable more scalable development, including streamlined review when multiple products leverage a common technology, or when a drug can be individualized for patients in the course of care.
- Updating internal processes, including by: (1) building on existing efforts to leverage AI and other advanced computing tools in product reviews, with refinement and appropriate guardrails, and (2) strengthening confidence in the accelerated approval pathway through procedural transparency and leveraging more real-world data to improve follow-through on confirmatory studies, which would help address challenges like retaining study participants.
- Updating regulations to make operating in the U.S. more globally competitive, particularly in areas where we’ve seen the most off-shoring, like manufacturing and early-phase clinical research—for example, by modernizing manufacturing regulations to facilitate the development of domestic advanced manufacturing facilities, and streamlining requirements for early-phase research while maintaining protections for human subjects.
Full testimony is available here. The Schaeffer Center’s recent white paper on FDA modernization is here.
