Rare Disease Supporters Suggest Permanent PRV Program, Even If It May Concern FDA
Non-profit biotech founder says permanent pediatric rare disease priority review voucher program will boost incentive, but FDA is concerned about resource drain vouchers cause.
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Researchers find no significant difference in number of rare pediatric disease treatments starting clinical development before or after incentive was created, but advocates aren't ready to give up on the program yet.
Drug sponsors tell US GAO that orphan incentives don't draw them to the space, and several stakeholders say high prices and scientific discovery are the reason for the sector's growth.
Medicines Development for Global Health is the first nonprofit to earn a priority review voucher for bringing a neglected tropical disease therapy to US approval. Project carried on research from the Wyeth era.