This bill would reauthorize an existing program which allows drug companies to receive a Priority Review Voucher (PRV) in order to have an expedited approval process for a treatment that would benefit pediatric rare disease patients. This is important to our community because we want the companies working on new treatments to have every tool available to get those treatments approved as quickly as possible for our community.
This legislation has broad bi-partisan support, but did not make it through the legislative process in 2024, expiring on December 20, 2024, despite a five-year reauthorization passing the House with bipartisan support.
12/1/2025 Update
From the EveryLife Foundation
The EveryLife Foundation for Rare Diseases applauds the House of Representatives for passing the Give Kids a Chance Act (H.R. 1262), legislation that includes policies that will spur progress in therapy development for rare diseases, most notably, the reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program. Today’s milestone comes as a result of community-driven advocacy and dedication from rare disease patients, families, organizations, and others who shared their stories and used their voices to ensure Congress acted.
Rare Pediatric Priority Review Voucher Program Reauthorization
The Give Kids a Chance Act includes a 5-year renewal of the Rare Pediatric Disease Priority Review Voucher Program, which lapsed in December 2024. We are especially grateful to the original bill leads, Representatives McCaul (TX-10), Dingell (MI-6), Bilirakis (FL-12), Matsui (CA-7), Schrier (WA-8), Harshbarger (TN-1), Castor (FL-14), Kelly (PA-16), Crenshaw (TX-2), Trahan (MA-3), and Weber (TX-14) for their leadership and steadfast commitment to restoring the PRV Program’s authorization.
The PRV Program has led to life-altering product approvals to treat over 40 rare diseases, such as Progeria syndrome, Spinal Muscular Atrophy, Sickle Cell Disease, Rett syndrome, and other conditions that previously lacked any FDA-approved treatments.
Retaining Access and Restoring Exclusivity Act (RARE Act)
By passing the Give Kids a Chance Act, Congress also approved language from the RARE Act, clarifying that the Orphan Drug Act’s market exclusivity period should be applied based on the approved use or indication a drug is approved for, rather than applying to all uses within a disease or condition, as a court ruled in a 2021 case. If signed into law, the RARE Act would ensure that incentives to study different segments of a given patient population remain. We are grateful to the RARE Act’s lead sponsors, the House co-chairs of the Rare Disease Congressional Caucus, Representatives Doris Matsui (CA-7) and Gus Bilirakis (FL-12).
Further Action Needed
While we celebrate the House passage of the Give Kids a Chance Act in the 119th Congress, we recognize that this is only the first hurdle. Despite last year’s efforts, the Rare Pediatric Disease (PRV) Program expired in December 2024, leaving one less tool to bring treatments to the rare disease community. The impact of this is seen in clinical trial delays, not by science, but by financial limitations. With only five percent of diseases having an FDA-approved treatment, it is imperative that we continue to advance solutions to treat these devastating diseases, 70% of which start in childhood.
The EveryLife Foundation calls on the Senate to act swiftly and pass the Give Kids a Chance Act (S.932) to ensure that life-saving therapy development remains a resource for innovation and treatment.
To learn more about engaging with your Senators to support reauthorization of the PRV program, and to access shareable resources, please click here
9/19/2025 Update
From Alliance for Regenerative Medicine:
Yesterday, the House Energy and Commerce Committee voted to advance the Give Kids a Chance Act (H.R. 1262) with strong bipartisan support – 47 members of the Committee voted in support of the bill with no opposition. Additionally, 12 Committee members during the markup spoke in strong support of reauthorizing the Rare Pediatric Disease Priority Review Voucher (PRV) Program, which expired last year and is included in the Give Kids a Chance Act.
During the markup several members of Congress noted that the Rare Pediatric Disease PRV Program is a proven tool to spur treatment for children living with rare and devastating diseases, and it does so at no additional costs to taxpayers. It was also noted that there are few policies in Congress that rally as much bipartisan support as Give Kids a Chance Act, and that it passed the House of Representatives in the previous Congress.
To capitalize on this momentum, we need your help! Please contact your representative to urge House Leadership to schedule the Give Kids a Chance Act for a vote on the floor and pass it as part of end-of-year legislation.
If you are unsure of your representative, you can easily do that by entering your home address at this link. To assist you, please review these materials:
9/16/2025 Update
DSF, along with 190 patient organizations, signed onto the letter urging swift reauthorization of the Rare Pediatric Disease PRV Program from the Everylife Foundation. The letter has been delivered to House and Senate leadership. Tomorrow, the House Energy & Commerce Committee will hold a Full Committee markup that includes H.R. 1262, Give Kids a Chance Act of 2025. You can see the final letter at this link.
Since 2012, the Rare Pediatric Disease PRV Program has:
- Enabled 62 new treatments for rare diseases that affect children
- Provided over 40 disease communities with hope, only 4 of which had a treatment before the program’s inception
- Spurred investment in therapy development for hundreds of pediatric diseases that would otherwise be considered financially unviable programs
2/12/2025 Update
the Give Kids a Chance Act, H.R. 1262, was reintroduced by Representatives McCaul (TX) and Dingell (MI). The Give Kids a Chance Act would accelerate pediatric cancer treatments and expand access to life-saving therapies for children battling rare diseases as well as reauthorize the Rare Pediatric Review program. The Creating Hope Act of 2012 established the PRV Program to help expedite the development and promotion of drugs for diseases that disproportionately affect children. Since then, the PRV Program has incentivized the development of 58 new treatment options.