On February 3, 2026, the Consolidated Appropriations Act, 2026 (P.L. 119-75) became law and marked an important milestone in the bipartisan congressional effort to reauthorize the rare pediatric disease priority review voucher program. Section 6604 of this law extends the Food and Drug Administration’s (FDA) authority to issue priority review vouchers to encourage treatments for rare pediatric diseases through September 30, 2029.
While the extension of this authority provides certainty to this program for the next few years, newly enacted reporting provisions included with the extension also set the stage for continued assessments of the impact of this program. Section 6604 requires the Government Accountability Office (GAO) 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. Notably, this means the GAO analysis on this program is likely to be submitted after the current program authority extension is now set to expire.
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 (PDUFA) program is set to expire next year on September 30, 2027. Rare disease issues have been a prominent part of prior PDUFA reauthorizations and PDUFA VIII may follow suit. Policymakers perpetually examine how to spur the timely development of safe and effective therapies for rare disease patients as they strive to optimize a drug development paradigm that reflects unique development challenges, urgent patient needs and advances in innovation. In the interim, the recent extension of the rare pediatric disease priority review voucher authority underscores Capitol Hill’s continued engagement on these issues.
