PDUFA VII: US FDA Will Offer Additional Meetings To Boost Rare Disease Endpoint Development

If they reach a public disclosure agreement, a handful of sponsors will be admitted to a new pilot program that allows four meetings with the FDA on rare disease endpoint issues.

drug development pipeline — The RDEA pilot program will offer sponsors additional help from the FDA on novel rare disease endpoints. • Source: Alamy

Rare disease drug sponsors could gain additional access to US Food and Drug Administration officials as part of an relatively small endpoint development pilot program beginning in fiscal year 2023.

The FDA is expected to establish the Rare Disease Endpoint Advancement (RDEA) pilot program, which is intended to increase FDA-sponsor communications and help develop novel endpoints for rare disease clinical

“Current mechanisms for sponsors of rare disease drug development programs to collaborate with FDA are not structured to provide repeated, intensive interactions with the agency,” the FDA and industry wrote in the

PDUFA VII: US FDA Will Offer Additional Meetings To Boost Rare Disease Endpoint Development

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