Drugs

Rare Disease Endpoint Advancement Pilot

David McFarland

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FDA is establishing a Rare Disease Endpoint Advancement (RDEA) pilot program that it committed to under the PDUFA 7 reauthorization. An agency announcement says the program will support novel efficacy endpoint development for drugs that treat rare diseases.

FDA says the RDEA pilot is intended to:

  • seek to advance rare disease drug development programs by providing a mechanism for sponsors to collaborate with the agency throughout the efficacy endpoint development process;
  • promote innovation and evolving science by sharing learnings on novel endpoint development through FDA presentations, guidances, public workshops, and a public-facing Web site; and
  • develop FDA staff capacity to enable and facilitate the development and use of novel endpoints to evaluate the efficacy of rare disease therapies.

The announcement includes general program information and details on requests for program funding.

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