It Lives (for now)!: Congress Revives Pediatric Priority Review Voucher Program with the Give Kids a Chance Act of 2025

There are officially signs of life for FDA’s rare pediatric disease priority review voucher (RPD PRV) program!  On September 17, the House Committee on Energy and Commerce marked up the Give Kids a Chance Act of 2025 (H.R. 1262), legislation that would reauthorize FDA’s RPD PRV program and strengthen the agency’s authority in several related areas.  The bipartisan bill originally was a piece of a broader healthcare package that nearly passed late last year but was sidelined in the closing days of the 118^th Congress.  The current iteration of this bill has languished for over seven months since being introduced in the House of Representatives and referred to the House Committee on Energy and Commerce on February 12. Notably, the bill has accumulated 285 cosponsors, which amounts to over 65% of the total number of voting members of the Chamber, which indicates substantial support despite this inaction.

At the heart of the bill is the revival of the RPD PRV program, which expired in part in December 2024; this date marked the deadline by which a drug product could be designated as treating a rare pediatric disease.  Looming now is the second deadline date in the law: the sunset to receive an RPD PRV, with eligible applications needing to be approved by September 30, 2026.  Originally created in 2012, the program was designed to incentivize the development of treatments for rare pediatric diseases by awarding transferable (i.e., saleable) vouchers that allow faster FDA review of future products.  To date, 56 vouchers have been issued across roughly 40 rare conditions—including sickle cell disease, AADC deficiency, spinal muscular atrophy, Duchenne muscular dystrophy, and Friedreich’s ataxia—creating significant financial incentives to bring much-needed therapies to patients that might not otherwise have been developed.  As we previously blogged, an analysis from National Organization for Rare Disorders found that among the 39 rare pediatric diseases for which vouchers had been awarded at that time, only three had any FDA-approved products on the market before the program’s enactment, suggesting that the incentive has been very effective.  If enacted, the program would be extended through September 30, 2029.  Notably, the bill as currently drafted would do away with the two-part deadline (first for designation and then for approval) as it currently exists, replacing it with a single deadline date after which FDA cannot award vouchers.

There was enthusiasm late last year when the committee unanimously advanced a similar bill in 2024 prior to the December 2024 expiration of the first deadline date in the law, reflecting the bipartisan appeal of incentivizing pediatric rare disease research.  However, given the lack of further action and even less up until this point in 2025, patient advocacy groups have had to champion reauthorization.  See, for example, this call to action by the EveryLife Foundation for Rare Diseases (“EveryLife Foundation Asks Advocates to Urge Your Member of Congress to Support the Reauthorization of the Rare Pediatric Disease PRV Program”).  Patient advocates, including HPM’s own James Valentine, have echoed and amplified this sentiment.  In his remarks to the Rare Disease Congressional Caucus Briefing last week, Mr. Valentine stated that “the Rare Pediatric Disease Priority Review Voucher program [and] patient-focused drug development initiatives—all reflect bipartisan recognition that rare disease drug development is different, and that patients must have a central voice.”

Beyond RPD PRVs, the Give Kids a Chance Act would expand FDA’s oversight and clarify certain of its regulatory authorities:

• Pediatric cancer trials:  FDA could require pediatric studies of combination therapies of certain molecularly targeted cancer drugs when the drugs are owned by the same company or are generics.
• Enforcement authority:  FDA could take enforcement action against drugmakers that fail to meet pediatric study obligations under PREA, aligning these requirements with other post-market study mandates.
• Orphan drug exclusivity:  The bill would codify FDA’s interpretation of orphan drug exclusivity (i.e., seven-year market exclusivity for orphan drugs prevents the approval of the same drug for the “same approved use or indication within such rare disease or condition,” as opposed to the previous and more general “same disease or condition.”).  The bill also defines the term “approved use or indication.” This would narrow the scope of exclusivity so that it applies only to the indication rather than the entire designated disease (as previously discussed here).
• Organ transplant system:  The legislation authorizes FDA to collect registration fees from Organ Procurement and Transplantation Network members—a public-private partnership that manages the U.S. organ donation and transplantation system—to fund transplant operations.
• Global presence:  FDA would be permitted to establish an office in a country that is a party to the Abraham Accords (i.e., Israel and four Arab nations: the United Arab Emirates, Bahrain, Sudan, and Morocco). This provision is intended to enhance supply chain security and leverage innovation in the region, according to a cosponsor.

The bill’s markup signals fresh momentum for legislation that stalled at the end of 2024, when broader negotiations were derailed.  For rare disease product developers, the bill offers the continuation of a powerful incentive that patients and their families, drug developers, and other stakeholders have been pleading for. Given this momentum from a wide variety of stakeholders, including the numerous cosponsors, we can only hope that the provisions beyond RPD PRV reauthorization do not create further roadblocks to getting this program back on the books as soon as possible.

With the RPD PRV program’s sunset roughly a year away, a failure to reauthorize—and soon—may force many companies to make the difficult choice of rushing to submit their NDA/BLA in the coming months or early 2026.  Rushed submissions could lead to a wave of applications that do not have the benefit of full clinical or CMC datasets or vetting with FDA (e.g., via multiple interactions post-Phase 3).  In turn, this could create a high-risk situation in which 2026 may be marked by more applications receiving Complete Response Letters, which is not an efficient use of industry or FDA resources.

In the long term, it would be a tragedy for patients who might not benefit from therapies that would otherwise have been developed if the appropriate incentive was in place. We will never know the cost of the uncertainty this has caused, including, at minimum, delays in getting such therapies to patients suffering from serious or life-threatening diseases who need them. FDA has been doing its part to encourage the continuation of the program, primarily by continuing to grant rare pediatric disease designations past the December 2024 deadline. Now it is Congress’s turn to act, and quickly; the clock is ticking.