Session XI — Federal Perspectives
The FDA Is Moving Faster Than You Think — and NIH Is Rewriting How Science Gets Done
Five senior federal officials on structural shifts in the regulatory and funding environment.
One Trial Is Now the Default
- FDA formalized in NEJM (Feb 2026): one adequate, well-controlled trial + confirmatory evidence is the basis for marketing authorization
- Commissioner’s National Priority Voucher: median approval time 54.5 days (vs. 10-month PDUFA clock)
- CDER 100% compliant with all PDUFA deadlines as of April 2026
- Real-time clinical trials announced: FDA receives safety/efficacy data continuously via EMR
Plausible Mechanism Framework
- Regulatory pathway for bespoke therapies (e.g., custom CRISPR for CPS1 deficiency — “baby KJ”)
- No population-level trial required if: known molecular abnormality, characterized natural history, confirmed target engagement
- Recent examples: Avlayah (Hunter syndrome), Loargys (Arginase 1 deficiency), Zycubo (Menkes disease)
BMD Surrogate Endpoint
- Dec 2025: total hip BMD qualified as validated surrogate for osteoporosis approval
- SABRE Project: 52 RCTs, 160,000+ patients, over a decade
- Fracture trial: 10K-15K patients, 2-5 years → BMD trial: hundreds of patients, ~2 years
- Opens door to many new osteoporosis drugs
Devices & AI (CDRH)
- RAPID program (April 2026): CMS sits at FDA table during IDE → national coverage 60-90 days after authorization (vs. ~1 year)
- Home as Health Care Hub: >400 OTC devices; TEMPO pilot for unapproved digital health
- Agentic AI: foundational models outpacing draft guidance; benchmarks being defined with NIST, ONC
NIH Pivot to Human-Based Science
- Funding opportunities exclusive to animal models will no longer be issued
- COMPLEMENT program (Common Fund): 7 technology development centers for human-based models
- Standardized Organoid Modeling Center ($87M; Frederick National Laboratory)
- Cross-agency real-world data network: NIH + CMS + VA + CDC
Cross-Agency Fluency
- RAPID (FDA + CMS), COMPLEMENT (NIH + FDA + EPA), real-world data (NIH + CMS + VA + CDC)
- “One government” posture — substantively new in scope and formality
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