Ultrarare diseases need a fast lane: biomarker endpoints could help — a Perspective

For ultrarare diseases, biomarker-based approvals could be a powerful mechanism for delivering the “accelerated” in accelerated approval. With a window opening at FDA for reimagining regulatory standards for ultrarare diseases, I would argue that wide-scale use of biomarker-based surrogate endpoints should be on the table for consideration, though achieving this would require a paradigm shift.

FDA’s new leadership has assured the rare disease community that it intends to employ fit-for-purpose evidence standards that don’t force orphan therapies into a regulatory paradigm built for common conditions. ...