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Rare Pediatric Disease Designations May Be Unavailable After July As Program Renewal Remains Uncertain

 
• By Derrick Gingery

Without new legislation, US FDA said that designations, which are required to receive priority review vouchers, cannot be assured if requested after July. While the window for designations formally closes at the end of September when the authorization of the program expires, the agency is telling sponsors that it needs 60 days before that to review any requests. If a product does get a designation, it would need to secure approval by 30 September 2022 to gain a priority review voucher.

Priority Review Voucher
Stakeholders are pushing for congressional reauthorization of the rare pediatric disease priority review voucher program before it sunsets. • Source: Illustration

Aspiring sponsors have only a few weeks left to request a rare pediatric disease designation from the US Food and Drug Administration as the related priority review voucher program nears its sunset.

The rare pediatric disease priority review voucher program will end on 1 October absent renewal by Congress. However, sponsors who receive a rare pediatric disease designation prior to that date...

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