Klobuchar, Wicker, Bilirakis, Matsui Lead Colleagues in Urging the FDA to Improve Reliability in Reviewing Applications for Rare Disease Therapies

Press Release

"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. 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."

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