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Rare, Pediatric Drug Development and Reimbursement Boosted In Shaky US Spending Deal

 
• By Sarah Karlin-Smith

A Catalyst fix, a pediatric rare disease voucher reauthorization that should prevent future workload imbalances for FDA, and a boost for out-of-state coverage in Medicaid are among Congress’s year end commitments to pediatric and rare disease patients that now seem in doubt.

Little girl at doctor's appointment.
Rare and pediatric disease drug development and coverage may get a late 2024 boost from Congress. (Shutterstock)
Key Takeaways
  • A legislative fix for the Catalyst court decision codifying and retroactively applying the FDA's interpretation of orphan drug designations was included in the 17 December continuing resolution to fund the US government through March.
  • Opposition from House Republicans and the incoming Trump administration may still kill what appeared to be a bipartisan, bicameral compromise.
  • The bill also would reauthorize the rare pediatric disease priority review voucher program, add new requirements for combo pediatric cancer drug studies, and ease out-of-state access to care for children in Medicaid.

Rare and pediatric disease drug sponsors and the US Food and Drug Administration could resolve several ongoing rare and pediatric drug development issues in a compromise bill to fund the...

The continuing resolution negotiated by bipartisan leaders in the Senate and House of Representatives must be cleared by 20 December to avert a government shutdown....

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