Drugs

2-Day Workshop on Rare Disease Endpoints

FDA and the Duke-Margolis Center for Health Policy have jointly announced a 6/7-8 public workshop on the agency’s Rare Disease Endpoint Advancement (RDEA) Pilot Program and novel endpoint development for rare disease drug development. The workshop will discuss:

The pilot program, including application process and required elements for a RDEA proposal
Scientific/technical issues associated with developing rare disease study endpoints
Lessons learned from previous user fee meeting programs that can be applied to the pilot

“The lack of regulatory precedent, small trial populations, and/or limited understanding of disease natural history associated with rare diseases creates unique challenges when determining the appropriate efficacy endpoint(s) for clinical trials intended to evaluate the effectiveness of rare disease therapies,” an FDA notice says. “Well-developed efficacy endpoints, especially those that could apply to other rare diseases with similar manifestations, can help drive the general advancement of rare disease drug development.”

FDA says that specific questions about applications will not be addressed at the workshop, but case studies will be presented as learning examples.

For more information and to register for this workshop, please visit the Duke-Margolis Center for Health Policy RDEA public workshop Web page.

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Gupta Raises Concerns About Overton’s Independence, Ideology

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