FDA is establishing a Rare Disease Endpoint Advancement (RDEA) Pilot Program to support novel efficacy endpoint development for drugs that treat rare diseases. The RDEA Pilot Program fulfills a commitment under PDUFA VII.

The RDEA Pilot Program is designed to:

  • Seek to advance rare disease drug development programs by providing a mechanism for sponsors to collaborate with FDA throughout the efficacy endpoint development process.
  • Promote innovation and evolving science by sharing learnings on novel endpoint development through FDA presentations, guidance documents, public workshops, and a public-facing website.
  • Develop FDA staff capacity to enable and facilitate the development and use of novel endpoints to evaluate the efficacy of rare disease therapies.

Read more here.