Bilirakis, Barragán, Cammack, Auchincloss, Kean & Mullin Introduce Bipartisan Bill to Permanently Protect Rare Pediatric Disease Drug Development Program -Legislation Provides Long-Term Certainty to Encourage Investment in Lifesaving Treatments for Childr

Bilirakis, Barragán, Cammack, Auchincloss, Kean & Mullin Introduce Bipartisan Bill to Permanently Protect Rare Pediatric Disease Drug Development Program

-Legislation Provides Long-Term Certainty to Encourage Investment in Lifesaving Treatments for Children with Rare Diseases-

 WASHINGTON, D.C. – Earlier today, U.S. Representatives Gus Bilirakis (R-FL), Nanette Barragán (D-CA), Kat Cammack (R-FL), Jake Auchincloss (D-MA), Tom Kean, Jr. (R-NJ) and Kevin Mullin (D-CA) introduced bipartisan legislation to permanently authorize the U.S. Food and Drug Administration’s (FDA) Rare Pediatric Disease Priority Review Voucher (PRV) Program, an important incentive for developing treatments for children with rare and devastating diseases.  Since its creation in 2012, the program has helped bring treatments to market across 47 rare pediatric diseases - only four of which had an FDA-approved treatment before the program existed. The program, currently scheduled to sunset in 2029, awards a transferable priority review voucher to companies that successfully develop and receive FDA approval for qualifying treatments. Because rare-disease drug development can take as long as 15 years, permanent authorization would provide the long-term certainty needed to encourage continued investment in treatments for small patient populations that might otherwise struggle to attract research and development resources.

 “Parents of children battling rare diseases should never have to hear that promising research was abandoned because there wasn’t enough incentive to pursue a treatment,”said Congressman Gus Bilirakis. “Medical innovation is advancing at an incredible pace, but no child should be left behind simply because their disease affects too few patients to attract traditional investment. Drug development takes years, and innovators need certainty when making research decisions today. By making this successful program permanent, our bipartisan bill will encourage continued investment in lifesaving treatments and give hope to families who are desperately waiting for the next breakthrough.”

 “We should not let a rare disease become a death sentence simply because the market is too small,”said Rep. Nanette Barragán. "The Rare Pediatric Disease Priority Review Voucher Program has helped bring treatments to children who face devastating diseases that previously had few or no options. That is why I am joining Congressman Bilirakis in introducing bipartisan legislation to permanently authorize this program and create certainty for researchers and investors who need to make decisions today for families and children who cannot afford to wait.”

 “Every child deserves a chance at life, no matter how rare their disease,” said Congresswoman Kat Cammack. “A rare diagnosis should never mean that a child is left behind in the search for a treatment or cure. The Rare Pediatric Disease Priority Review Voucher Program encourages America’s innovators to take on these devastating diseases and pursue breakthroughs that can give children and their families hope. I’m proud to join this bipartisan effort to make this important program permanent.”

 “Every child deserves a shot at a healthy future,”  said Congressman Mullin. “The Rare Pediatric Disease Priority Review Voucher Program is bringing lifesaving treatments to kids who desperately need them, and we can’t let that progress stall. I’m proud to join this bipartisan effort to make the program permanent and give families hope that more rare pediatric treatments will be developed. When a child’s life is on the line, we can’t afford to wait.”

 "Children with rare cancers need cures just as much as everyone else. Yet, many companies who want to develop those cures can’t find the necessary financing for such a small group of patients. The Rare Pediatric Disease PRV program provides these companies a vital incentive to invest millions of dollars to find cures for only a handful of children. Congress must permanently reauthorize this program to help these kids and give families hope that treatments and therapies will continue to advance for these devastating conditions,”said Congressman Tom Kean, Jr.

 “The medical science to cure pediatric diseases has never been more promising. The vouchers have been an important tool to turn that science into therapies. Congress must not abandon what's working – we should make the vouchers permanent,” said Congressman Jake Auchincloss.

 Although the program does not sunset until 2029, investment decisions are being made now. During the program’s lapse from December 2024 to February 2026, the Rare Disease Company Coalition identified roughly 200 therapies at risk of losing eligibility and reported that investors were pulling back from the space. Permanent authorization would provide greater certainty for continued investment in rare pediatric disease research. The legislation is supported by the National Organization for Rare Disorders (NORD), Rare Foundation (formerly EveryLife Foundation for Rare Diseases), Biotechnology Innovation Organization (BIO), and Rare Disease Company Coalition (RDCC).

 “The Rare Pediatric Disease Priority Review Voucher program is a proven, budget-neutral incentive that spurs innovation and supports the development of new therapies for children living with rare diseases,”said Chris Porter, Chair of the Rare Disease Company Coalition. “During the program's lapse from December 2024 to February 2026, over 200 pipeline assets were at risk of not receiving a priority review voucher, representing more than $4 billion in potential lost reinvestment capacity. Permanently reauthorizing the program will provide the certainty and predictability rare disease innovators need to continue investing into rare disease research and development for patients with few or no existing options. The Rare Disease Company Coalition applauds Representatives Bilirakis, Auchincloss, Barragán, Cammack, Mullin and Kean for their leadership and for taking action to make the PPRV program a permanent part of our nation’s rare disease innovation ecosystem.”

 “For families facing a rare pediatric disease, time isn't a luxury. The Rare Pediatric Disease Priority Review Voucher Program has proven to be one of the most effective tools we have for enabling companies to translate promising science into new treatments. Developing a treatment for a devastating pediatric disease takes years, sometimes decades. Making the PRV permanent gives drug developers and the patient communities counting on them the certainty to make that long-term bet. The RARE Foundation, formerly known as the EveryLife Foundation for Rare Diseases, is grateful to Representatives Bilirakis, Barragán, Cammack, Auchincloss, Kean, and Mullin for introducing the Priority for Pediatric Cures Act so that we can build on the incredible momentum of the first 14 years of the PRV Program, which has enabled over 70 new treatments for pediatric rare diseases,” Annie Kennedy, Chief Mission Officer, Rare Foundation.

 “The Rare Pediatric Disease PRV program provides critical incentives for rare disease research and development at no cost to taxpayers. Permanent reauthorization of this vital program would bring hope to children and families touched by rare disease who have no more time to wait, and stability and confidence for biotech innovators who are leading the way in the discovery of breakthrough treatments,” said BIO President & CEO John F. Crowley. “Previous lapses in the PPRV program have devastated families who have run out of treatment options. We are grateful to Representatives Bilirakis, Barragan, and the bipartisan leadership of all the bill’s cosponsors for making the PRV permanent on behalf of pediatric patients and their families.”

Background

 Roughly one in ten Americans lives with a rare disease. Of the more than 10,000 known rare diseases, approximately 95 percent still have no FDA-approved treatment, and half of all patients diagnosed with a rare disease are children. Because rare diseases affect relatively small patient populations, promising treatments can struggle to attract the investment necessary to support years of costly research and development.

The Rare Pediatric Disease PRV Program helps address that challenge by awarding a priority review voucher following FDA approval of a qualifying treatment. The voucher may be used for another eligible application or transferred to another company. FDA has awarded 63 vouchers to date, with more than 90 percent going to therapies for indications that previously had no approved treatment. Making the program permanent would provide greater certainty for continued investment in treatments and cures for children with rare diseases.