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Rare Disease Advocates Lobby US Congress For FDA Center of Excellence

 
• By Derrick Gingery

Number one ask is for congressional pressure to centralize rare disease expertise at the agency, but advocates also are preparing legislation that would mandate the new center.

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Rare disease advocates also want increased funding for the FDA Office of Orphan Products Development's clinical trial and natural history grant programs. • Source: File photo

Rare disease advocates want Congress to push the US Food and Drug Administration to create a Rare Disease Center of Excellence but have not ruled out forcing the reorganization, if necessary.

As part of Rare Disease Week activities, patients and other stakeholders lobbied lawmakers on 27 February to sign a letter encouraging the FDA to use authority from the 2016 21st Century Cures Act to create a center of excellence that would centralize its rare disease expertise and elevate the program to report directly to FDA Commissioner Stephen Hahn

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