FDA recently announced the issuance of the first voucher awarded under its restored Rare Pediatric Disease (RPD) Priority Review Voucher Program for a drug that was approved after the program’s previous sunset. The Consolidated Appropriations Act, signed into law on February 3, 2026, reintroduced voucher availability for RPD-designated drugs and created a safety net for designations made when vouchers were not immediately available.
The previous iteration of the program had been allowed to sunset in late 2024 without a clear congressional plan for reintroduction, which had put the future of the program in doubt. While the new legislation still leaves some uncertainty for the long-term future of the program, authorizing FDA to grant these vouchers only until September 30, 2029, sunset provisions and renewal negotiations have been longstanding features of this program.
The reintroduction will be felt most for rare disease developers—often smaller biotechnology companies serving inherently small patient populations—who can benefit from these programs, including by selling the vouchers to other drug developers. FDA also operates similar voucher programs targeting other public health priorities where Congress has granted FDA authority to do so, such as for tropical diseases.
To qualify for an RPD voucher, a developer’s drug candidate and marketing application must independently meet stringent substantive and procedural criteria. Careful and advanced planning are therefore essential to ensure that a voucher will be granted on approval.
Drug Designation
To be eligible for RPD designation the drug candidate must be for the prevention or treatment of a rare pediatric disease, meaning a serious or life-threatening disease impacting fewer than 200,000 people in the United States that primarily affects individuals aged from birth to 18 years.
Application Eligibility
To qualify as a “rare pediatric disease product application,” an NDA or BLA must:
- Be for a drug or biological product for the prevention or treatment of a rare pediatric disease;
- Not contain an active moiety that has previously been approved;
- Be submitted as a 505(b)(1) NDA or a 351(a) BLA;
- Be deemed eligible for priority review (i.e., the drug is for a serious condition that would provide a significant improvement in safety or effectiveness of the treatment, diagnosis, or prevention of a serious disease);
- Rely on clinical data from pediatric studies; and
- Not seek approval for an adult indication in the original rare pediatric disease product application.
There are also several expectations from FDA, including with respect to the format, content, and timing of RPD-related submissions, that it has set forth in a 2019 draft guidance. CDER’s 2026 guidance agenda notes that it will also release a new guidance document in CY 2026 covering its priority review voucher programs, which may include updates applicable to the RPD program and which we expect will provide useful transparency on FDA’s implementation and policies for these programs going forward.
The high market demand for these vouchers underscores the continued effectiveness of the program as a robust development incentive, even as FDA experiments with other initiatives and voucher programs that are also intended to shorten drug review periods, such as the Commissioner’s National Priority Review Pilot Program (which issues nontransferable vouchers and is also not targeted specifically toward rare or pediatric programs).
While the long-term availability of RPD vouchers may hinge on Congress’s renewal, their value may also depend on FDA’s approaches to other development incentives that, if not managed carefully, could erode the effectiveness of existing programs.
Sponsors developing drugs or biologics in this space that may qualify for a voucher should pay close attention to FDA’s requirements, even if and as they continue to evolve, to ensure an RPD voucher is issued on approval. We will continue to monitor developments in this space.