Congress Reauthorizes Rare Pediatric Disease Priority Review Voucher Program Through 2029

The Consolidated Appropriations Act of 2026, signed into law on February 3, has reauthorized the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program, restoring a key regulatory and commercial incentive for sponsors developing therapies for rare pediatric diseases. Section 6604 of the law extends the Food and Drug Administration's authority to issue priority review vouchers through September 30, 2029, halting a period of legal and commercial uncertainty after the program lapsed at the end of 2024.

The legislative provisions were incorporated from the Mikaela Naylon Give Kids a Chance Act into the broader budget package that ended a partial government shutdown earlier this month. The reauthorization marks an important milestone in the bipartisan congressional effort to support treatments for rare pediatric diseases.

Under the program structure, sponsors can request the FDA to designate a drug as an RPD drug, which must treat a serious or life-threatening condition that primarily affects patients from birth to 18 years of age and has a prevalence of fewer than 20,000 people in the United States. Upon FDA approval of a designated RPD drug, a sponsor receives an RPD PRV.

A voucher allows the holder to secure priority review — an approximately six-month FDA review — for a different new drug application, accelerating traditional approval timelines by several months. Crucially, these RPD PRVs are fully transferable and marketable, allowing sponsors to sell them to third parties, often generating substantial nondilutive funding.

The secondary market for these RPD PRVs has seen transactions in the hundreds of millions of dollars. Recent sales of RPD PRVs include Jazz Pharmaceuticals at $200 million in January 2026, Abeona Therapeutics at $155 million in June 2025, Zevra Therapeutics at $150 million in April 2025, Acadia Pharmaceuticals at $150 million in December 2024, PTC Therapeutics at $150 million in November 2024, and Ipsen at $158 million in August 2024.

The newly enacted statute includes reporting provisions that set the stage for continued assessments of the program's impact. Section 6604 requires the Government Accountability Office to study and submit a report to Congress on the effectiveness of awarding rare pediatric disease priority vouchers in the development of products that treat or prevent rare pediatric diseases not later than five years after the enactment of this provision. The GAO analysis on this program is likely to be submitted after the current program authority extension is set to expire.

The reauthorization provides renewed clarity and strategic opportunity for companies developing therapies in an area often challenged by small patient populations and extended development timelines. The reinstatement effectively restores a regulatory incentive structure that began in 2012 and entered a sunset period before lapsing at the end of 2024.

Next week marks Rare Disease Week on Capitol Hill, reflecting the continuing dedicated focus on how to address unmet rare disease patient needs. The current Prescription Drug User Fee Act program is set to expire on September 30, 2027. Rare disease issues have been a prominent part of prior PDUFA reauthorizations and PDUFA VIII may follow suit.