FDA’s new CBER and CDER heads back adaptive framework for rare disease therapies

Newly appointed permanent directors of FDA’s CBER and CDER, Karim Mikhail and Michael Davis, indicated they will continue the agency’s push for an adaptive regulatory framework aimed at rare disease therapies.

The emphasis on adaptability, rather than mere flexibility, reflects concerns from the rare‑disease community that lower standards could hinder patient access. BIO President John Crowley voiced this view at a recent town hall, stressing the need for elevated regulatory science.

Both directors expressed optimism that the FDA can implement a truly adapted framework, signaling ongoing support for innovative treatments in the rare‑disease space. The upcoming Senate confirmation of commissioner nominee Heidi Overton may influence the pace of these efforts.