An expiring FDA program designed to incentivize research in pediatric rare disease may see new life. The Pediatric Rare Disease Priority Review Voucher (RPD PRV) provides sponsors of granted new marketing applications for drugs and biologics intended to treat a rare pediatric disease with a priority review voucher to expedite the FDA’s review of future marketing applications. The PRV is fully transferrable to other drug sponsors, creating a potentially significant revenue source for companies engaged in rare disease research. This program is distinct from other priority review programs, such as the highly publicized – but narrower – Commissioner’s National Priority Review program (CNPV), which does not allow transfer of the PRV.
The RPD PRV program sunsetted by statute in December 2024, and numerous attempts by Congress to reauthorize it have been unsuccessful. However, the “Mikaela Naylon Give Kids a Chance Act,” a recent bill incorporated into the 2026 Continuing Appropriations Act, would reauthorize the RPD PRV program and extend voucher awards through 2029.
Rare Pediatric Disease Priority Review Voucher: Requirements and Timing
Despite the sunset of the program, the current statute authorizes the FDA to grant requests for a RPD PRV for any approved rare disease product application until Sept. 30, 2026, provided that the sponsor of the application received pediatric rare disease designation prior to Dec. 20, 2024. Applications that were not designated or approved prior to the December 2024 cutoff are ineligible for the RPD PRV.
The requirements under the current statute, as further developed through FDA guidance, are as follows:
- Designation Requirements. Sponsors may request rare pediatric disease designation for a drug or biologic intended to prevent or treat a rare pediatric disease. A “rare pediatric disease” under the statute (1) is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals from birth to 18 years under standard treatment for the disease, and (2) is “rare” in the United States (impacting fewer than 200,000 people in the United States under the Orphan Drug Act).
- Designation Timing. The FDA recommends submitting a complete rare pediatric disease designation request at the same time (within two weeks) as submitting requests for fast-track or orphan drug designation – or, if only requesting rare pediatric disease designation, no later than the FDA’s filing of the product application.
- RPD PRV Request and Eligibility. Designation alone does not confer a voucher. To receive a voucher at approval, the application must qualify as a “rare pediatric disease product application,” which requires the application is:
- submitted as a new drug application under section 505(b)(1) or a biologics license application under section 351(a) of the Public Health Service Act;
- for the prevention or treatment of a rare pediatric disease; and
- the first approved application for the active moiety (drugs) or active ingredient (biologics) for that disease.
The FDA issues its decision on an RPD PRV request at the same time as the agency issues approval of the marketing application for the product.
The RPD PRV itself, once issued, provides an expedited review for future applications, reducing review time from the typical 10 months to six months. That four-month acceleration has proven to be valuable to drug sponsors. A niche market for PRVs has emerged whereby, typically, larger drug companies are buying PRVs from smaller sponsors of rare disease products, although this is not always the case. Media reports indicate that the most recent PRV was sold for $200 million, setting a new high for PRV value. A report by the Rare Disease Company Coalition estimates that approximately 200 rare disease therapies are currently impacted by the sunset, putting approximately $4 billion in total PRV value at risk for biotech companies.
Current Status of the RPD PRV Program
Congress previously enacted a short-term extension that moved the program’s sunset for rare pediatric disease designations from Sept. 30, 2024, to Dec. 20, 2024, via the Continuing Appropriations and Extensions Act, 2025 (H.R. 9747, Public Law No. 118-83). The statutory deadline by which a designated product must be approved to receive a PRV remains Sept. 30, 2026, unchanged by the short-term measure. The FDA reflected the new designation deadline on its website.
Reauthorization Effort: Mikaela Naylon Give Kids a Chance Act/Consolidated Appropriations Act, 2026
The RPD PRV-reauthorization provisions of the bill, the Mikaela Naylon Give Kids a Chance Act (H.R. 1262), are included in the Consolidated Appropriations Act, 2026 (H.R. 7148). On Jan. 30, 2026, the Senate passed the Consolidated Appropriations Act, 2026, which now heads back to the House for further action. The bill would give the FDA authority to award RPD PRVs through Sept. 30, 2029. The act revises timing of the PRV user fee payment to be due upon submission of the application that uses the voucher.
The act also directs the Government Accountability Office to re-evaluate the RPD PRV program effectiveness, the specific approved indications for each approved rare disease product application and whether each met an unmet need, the value of any PRV sale or transfer, the timing of PRV use, sponsor information for those granted PRVs, and how the PRV program impacts FDA resource allocation and workload. The GAO previously conducted a similar study under the current version of the statute, but the pending bill requests a follow-up study upon reauthorization.
How RPD PRV Differs From the CNPV
Subsequent to the statutory sunset of the RPD PRV program, the FDA launched a new and very different PRV program. The Commissioner’s National Priority Review program has been highly publicized in recent months and presents a valuable option to qualifying sponsors. The CNPV program is an FDA commissioner-initiated program launched in June 2025 without a specific statutory authority (unlike the RPD PRV program), and without formal rulemaking/comment or published regulations. The program was announced by the FDA via press communication. It is uncertain whether the FDA might also revive the RPD PRV program without Congressional authority if Congress is unable to enact final passage.
The CNPV program is focused on drug and biologics applications that advance identified U.S. national priorities (e.g., major public‑health crises, transformative cures, onshoring/supply resilience, affordability). There are important differences that sponsors should note and therefore should consider evaluating each opportunity independently. These differences include:
- Purpose and timing. The CNPV program aims for a highly compressed FDA application review timeline (one to two months) via a high‑touch, multidisciplinary “tumor board‑style” review of the same application. The CNPV program also provides sponsors with enhanced communications with the agency during development and review. By contrast, the statutorily-created RPD PRV program only provides expedited review of a future application. The RPD PRV statute also does not itself create a distinct internal FDA review council or process for the qualifying application.
- Transparency and criteria. The FDA made statements explaining that the agency intends voucher selection under the CNPV program to weigh national priority alignment alongside feasibility, public health impact, application readiness, and known risks/uncertainties. The agency emphasizes that voucher selection does not alter standards for approval or scientific conclusions, according to the staff manual guide. There are no statutory criteria and no guarantee that a product that aligns with the priorities of the program will be selected. CNPV grants are at the agency’s discretion and are made in publicly-announced batches on the FDA’s website. In contrast, the RPD PRV program has clearer, explicit written criteria that confers a PRV to all applications that meet the statutory criteria. The FDA retains authority to make scientific decisions based on whether the product is for a rare pediatric disease or whether the product is the same as a previously-approved moiety or active ingredient (all of which might be subject to internal agency appeal processes and legal challenge on an Administrative Procedures Act basis or otherwise). However, the RPD PRV statute appears to generally require the agency to grant vouchers to all qualifying rare disease product applications.
- Operational requirements. The FDA updated staff guidance for the CNPV pilot to include pre-submission requirements, such as providing domestic facility and bioresearch monitoring site information at least 60 days before the complete application to enable timely inspections under the compressed review schedules. The FDA also indicates that review divisions can request limited extensions during the CNPV process via the delegated official if necessary. In contrast, applications for products that qualify for an RPD PRV are typically reviewed under standard agency procedures for drug and biologic applications, unless the application qualifies for other expedited programs in addition to the RPD PRV.
Practical Implications for Sponsors
- RPD PRV Planning. For products intended for a rare pediatric disease that have not yet been designated, the designation window has closed. For designated products, sponsors should plan to time the application submission well before the Sept. 30, 2026, approval deadline to secure a voucher. An application that will not be finally approved until after Sept. 30, 2026, will not be eligible for an RPD PRV unless reauthorization efforts are successful.
- Early FDA Engagement. If reauthorized, sponsors with early-stage programs should proactively engage the FDA to align development plans to ensure the indication and product are eligible for designation and an eventual RPD PRV. Sponsors should ensure disease prevalence estimates are well-supported and transparent, as the FDA may scrutinize methodology and data sources. When applicable, sponsors may need to evaluate patient registries or engage third parties to research and publish scientific support for prevalence estimates.
- PRV Value. If the RPD PRV program is reauthorized, the impact of PRV value will be significant for start-up and rare disease companies as well as larger pharmaceutical companies that have also sold off PRVs to gain access to quick cash needed for marketing and development programs. Sponsors should consider whether PRVs will continue to increase in value, or whether the latest high-value PRVs took into account their current scarcity in light of the currently sunsetted program. In any case, PRV value will be a critical part of especially smaller company valuation in considering potential investments in, or acquisitions of, PRV-eligible businesses.
McGuireWoods’ Life Sciences Industry Team continues to monitor congressional and regulatory developments related to pediatric drug and biologic development, as well as FDA priority review programs. For questions about related topics, contact the authors or a member of the team.