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Gus Bilirakis Leads Letter to FDA Calling for Improvements in Review of Rare Disease Therapies

U.S. Sens. Amy Klobuchar, D-Minn., and Roger Wicker, R-Miss., and U.S. Reps. Gus Bilirakis, R-Fla., and Doris Matsui, D-Calif., the co-chairs of the bipartisan bicameral Rare Disease Congressional Caucus, led a group of lawmakers in calling on the Food and Drug Administration (FDA) to bring more reliability and consistency to the process of reviewing rare disease therapies.

While there have been improvements in facilitating rare disease products, patients and advocates have identified that FDA divisions and offices can differ in how they treat applications. The lawmakers are urging the FDA to improve reliability in how it reviews applications for rare disease therapies.

“We recognize and appreciate the actions FDA has taken over the years to address the challenges associated with developing treatments for orphan diseases and conditions, including through the Office of Orphan Products Development and the recent establishment of the Accelerating Rare disease Cures (ARC) program. However, across the agency there remains significant uneven application of rare disease policies, guidance, and expertise, even, at times, for the same product application,” wrote the lawmakers. “We urge the convening of an FDA task force including leaders from all divisions, offices, and centers which process rare disease therapy applications to fully examine areas of policy and procedural inconsistency and shortcomings, as well as best practices, and provide concrete recommendations to the administration and Congress for improvement.”

This letter was also signed by 20 other members of Congress from both sides of the aisle, including U.S. Sen. Rick Scott, R-Fla., and U.S. Rep. Maria Elvira Salazar, R-Fla.


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