White Paper: FDA’s Shifting Goalposts—The Human and Economic Cost of Post Hoc Regulatory Rigidity in Biologics (2025–2026)
Executive Summary
The regulatory landscape for biological products in the United States has undergone a fundamental transformation between April 2025 and February 2026. Under new leadership at the FDA and CBER, the agency has moved away from the "regulatory flexibility" that characterized the 2022–2024 era, opting instead for a posture of "radical transparency" and a strict adherence to randomized, controlled trials (RCTs) over surrogate endpoints and single-arm studies. This paper quantifies the impact of these delays—estimating billions in additional development costs and irreversible clinical decline for thousands of children—and demonstrates that many of today’s standard-of-care gene therapies would have failed to achieve approval under the current regime.
1. The Post Hoc Standard: Reversing Precedent
Between 2022 and 2024, the FDA successfully utilized surrogate endpoints (e.g., micro-dystrophin, NfL) and single-arm trials to grant early access to breakthroughs in ALS, DMD, and oncology. However, correspondence from April 2025 to February 2026 reveals a systematic rejection of these same evidentiary markers.
READ FULL WHITE PAPER HERE
