US FDA External Controls Guidance Does Little To Advance The Cause, Especially In Rare Diseases

US FDA External Controls Guidance Does Little To Advance The Cause, Especially In Rare Diseases – Comments

16 Jun 2023

Industry and patient groups take issue with draft guidance’s skeptical tone on utility and reliability of externally controlled studies, with some saying the guidance ignores special considerations inherent in studying rare diseases. Recommendations on access to patient-level data for external control arms also are problematic and likely infeasible, stakeholders say.

Road obstruction — Stakeholders would like the FDA to take a less obstructionist approach to use of externally controlled trials. • Source: Shutterstock

The US Food and Drug Administration recent draft guidance on externally controlled trials does little to advance the use of such approaches, ignores challenges inherent with studying rare disease therapies, and imposes potentially unrealistic expectations for data access, stakeholders say.

In comments to the docket, biopharma manufacturers, data companies and patient organizations find much to fault with the February draft...