NORD urges Congress to support development of treatments for rare pediatric diseases



NORD has written letters to two key Congressional committees urging permanent authorization of a program designed to spur innovation in development of pediatric rare disease therapies. More than 100 rare disease patient organizations signed NORD’s letters, which were sent to the chairs and ranking members of the House Committee on Energy & Commerce and the Senate Committee on Health, Education, Labor, and Pensions.

The Rare Pediatric Disease Priority Review Voucher Program was established a few years ago and is set to expire in 2016. It provides vouchers to biopharmaceutical companies that develop treatments for pediatric rare diseases. The vouchers, which guarantee a six-month priority review by FDA of another product, may be used or sold to another company.

The program is widely perceived to be successful in encouraging research on pediatric rare diseases. NORD is leading the charge to have it permanently authorized.

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