Regulators Remain Consistent On Endpoints While Raising The Bar On Evidence Quality

In this segment of Cell & Gene Live, “2026 Regulatory Roadmap,” Cell & Gene's Chief Editor, Erin Harris, Wilson Bryan, M.D., former director of FDA’s Office of Tissues and Advanced Therapies (OTAT) and Daniela Drago, Ph.D., RAC, Partner at NDA Partners emphasized that regulatory expectations for functional endpoints, biomarkers, and real-world evidence have remained largely consistent over time, with the greatest weight still placed on rigorously designed trials using bias-resistant functional endpoints and well-validated biomarkers tied to clinical benefit.

Real-world evidence and synthetic or modeled controls can strengthen a package when they are pre-specified, methodologically sound, and used alongside multiple complementary control approaches, reinforcing conclusions rather than substituting for robust clinical data.