🧒 Introduction: Why Focus on Pediatric Rare Diseases?
Pediatric rare diseases often involve life-threatening conditions with few treatment options. In 2012, the U.S. introduced the Rare Pediatric Disease Priority Review Voucher (PRV) program to incentivize innovation in this space.
🇺🇸 What is the PRV Program?
A PRV is awarded upon FDA approval and allows the sponsor to expedite another drug’s review. These vouchers were transferable and sometimes sold for up to $350 million, offering crucial capital for biotech startups.
✅ Achievements and Impact
– 53 vouchers awarded across 39 diseases
– Over half issued in the last 4 years
– Key examples: SMA (Spinraza), Batten disease, and sickle cell anemia
❌ Why Was the Program Terminated?
– Expired on Sept 30, 2024, due to a sunset clause
– No Congressional renewal despite support
– Criticisms: Favoring big pharma and voucher resales undermining intent
🔄 Current Efforts to Revive It
– Proposals include price caps and restrictions to smaller companies
– Alternative supports: grants, tax credits, EMA collaboration
– Bipartisan lawmakers and patient groups are pushing for renewal
📝 Next
In the next volume, we’ll analyze Japan’s rare and pediatric disease policy and how it compares with the U.S.
