FDA launches Rare Disease Framework to embed patient input early in drug development

FDA's Center for Biologics Research and Evaluation (CBER) director Karim Mikhail announced a new Rare Disease Framework Initiative aimed at moving patient perspectives from an optional, late‑stage element to a required part of regulatory submissions.

Currently, patient input is typically gathered at listening sessions or advisory committee meetings that occur near the end of development, which Mikhail says is “way too late” because clinical plans are already set. The new framework will ask sponsors to involve patients, experts and regulators from the earliest phases, including when companies seek permission to begin human trials.

By integrating patient feedback early, the FDA hopes to shape trial designs and endpoints that better reflect real‑world needs, potentially accelerating development of therapies for rare diseases. The initiative was unveiled last month and signals a shift toward more collaborative, patient‑centered drug development.