Mission: Cure joins FDA's Rare Disease Endpoint Advancement Pilot Program as the agency calls for putting the patient's voice at the center of rare disease drug development

NEW YORK, Oct. 27, 2026 /PRNewswire/ -- Mission: Cure (https://mission-cure.org), a patient-led nonprofit working to accelerate treatments for chronic pancreatitis and recurrent acute pancreatitis, today announced that the U.S. Food and Drug Administration (FDA) has accepted its proposal for the Rare Disease Endpoint Advancement (RDEA) Pilot Program, which selects no more than three proposals per year. Mission: Cure will work with FDA experts to develop a patient-reported endpoint that measures pancreatitis flares, an outcome patients helped identify as essential to measure in pancreatitis trials.

The news comes as the FDA gathers public input on its new Rare Disease Framework Initiative ahead of an Oct. 28 public meeting. In the initiative's white paper, the FDA identifies limited natural history data and a lack of reliable clinical outcome assessments as key challenges in rare disease drug development. Mission: Cure's RDEA work is designed to address both.

The RDEA program is run jointly by the FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER). It supports the development of novel efficacy endpoints for rare disease treatments. Mission: Cure is participating as the sponsor of a natural history study in which the endpoint will be studied.

"The FDA is asking patients what counts as meaningful progress. In pancreatitis, the answer has been consistent for years: fewer flares," said Megan Golden, Mission: Cure co-founder and CEO. "We are turning that answer into a measure drug developers can use, and showing that a patient organization can lead the scientific work, not just inform it."

A growing number of therapies are entering clinical development, but there is still no FDA-approved therapy for chronic pancreatitis or recurrent acute pancreatitis that addresses the underlying pancreatic disease. Without an established way to measure flares, each company must develop and justify its own endpoint, adding cost, time, and regulatory risk before a trial can begin.

The endpoint will initially be studied in hereditary pancreatitis and is designed to apply across chronic pancreatitis and recurrent acute pancreatitis. The FDA may share what is learned through guidance, public workshops, and its website, so the knowledge can benefit any company developing a pancreatitis treatment. Mission: Cure invites drug developers and investigators planning pancreatitis trials to engage early in their planning.

More on the RDEA Pilot Program: https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program 

More on the Rare Disease Framework Initiative: https://www.fda.gov/industry/fda-rare-disease-innovation-hub/rare-disease-framework 

About Mission: Cure

Mission: Cure is a patient-led 501(c)(3) nonprofit co-founded in 2017 by Megan Golden and her brother Eric Golden, a pancreatitis patient. It acts as a scientific catalyst and drug development partner, working with companies, researchers, clinicians, and patients to move promising therapies for chronic pancreatitis and recurrent acute pancreatitis into and through clinical trials. Learn more at https://mission-cure.org. See therapies in development in its drug development pipeline: https://mission-cure.org/clinical-trials-and-strategy/our-progress-toward-a-cure/drug-development-pipeline/ 

Media contact
Joshua Henderson, Chief Cure Strategy Officer, Mission: Cure
425688@email4pr.com
+1 (401) 375-2873

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SOURCE Mission: Cure