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US FDA Still Reviewing Rare Pediatric Disease Designation Requests Despite Program Lapse

 
• By Sarah Karlin-Smith

The agency’s actions may signal its optimism about near-term reauthorization despite the PRV program and other bills aimed at tackling rare and childhood diseases not making the December 2024 government spending bill.

one lone Zebra standing in a field.
The FDA told the Pink Sheet that it continues granting rare pediatric disease designations, even though those issued after 20 December 2024 will not qualify a product for a priority review voucher. (Shutterstock)
Key Takeaways
  • The FDA continues to review requests and award rare pediatric designations, even though its congressional authority to give a rare pediatric disease priority review voucher upon approval lapsed.
  • There is optimism the rare pediatric disease PRV program and other health bills that were cut from a December 2024 spending bill at the last minute could pass early in 2025.
  • FDA law experts said reviewing designation requests likely does not add much extra work or effort to the FDA while the program is not authorized.

The US Food and Drug Administration continues to review requests for rare pediatric designations, even though the agency no longer can award a priority review voucher to the products upon...

The agency’s commitment to continue granting rare pediatric disease designations to drug or biologic applications may suggest it is optimistic about the potential for Congress to reauthorize the voucher program,...

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