Drugs

Congress Urges More FDA Attention to Rare Diseases

David McFarland

— 1 min read
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A bipartisan group of U.S. Senators and House members are urging FDA to step up its regulatory activities to improve rare disease product development and reviews. In a 5/2 letter, the lawmakers ask FDA to “establish an agency task force to, first, identify areas of strength and areas that challenge the efficient but appropriate review of therapies and products developed for patients living with a rare disease and, second, for that task force to publicly issue specific recommendations to achieve greater alignment across the FDA.

The letter asks that the task force issue a public report by 12/2024 that will:

  • Identify specific areas of both concordance and discordance involving rare disease policies across review divisions, offices, and centers through a timely and objective assessment of these disparities, including root cause analyses;
  • Review how FDA advisory committees have addressed rare disease applications and identify examples of inconsistent processes and policies;
  • Review the implementation of the Accelerated Approval pathway for rare disease therapeutic products across medical product centers, including when novel surrogate endpoints were proposed and what the outcome was; and
  • Review gaps in guidance specific to small population development (ultra-rare diseases) and offer recommendations for how the agency can address the unique challenges in these populations with new guidance and where new authorities would be required. 

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