Program Provides Crucial Incentives to Spur the Research and Development of Rare Disease Treatments for Most Vulnerable Population
Washington, D.C. – The Rare Disease Company Coalition (RDCC) is proud to endorse H.R. 7384, The Creating Hope Reauthorization Act of 2024, which would reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program. H.R. 7384 was introduced Thursday by Representatives Michael McCaul (R-TX-10), Gus Bilirakis (R-FL-12), Michael Burgess (R-TX-26), Anna Eshoo (CA-D-16), Nanette Barragán (CA-D-44) and Lori Trahan (MA-D-3).
Half of the 30 million individuals living with a rare disease in the United States are children – and 30 percent of those children won’t live to see their fifth birthday. Pediatric rare disease research is critical to provide hope for these children and their families.
Over the past decade, the program has proven to be an innovative and cost-effective policy that spurs research & development (R&D) in rare pediatric populations. The PRV program enables RDCC members to provide hope to children living with a rare disease while reinvesting critical dollars into additional R&D for rare disease treatments. The program has zero cost to taxpayers, and remains a critical incentive to attract investment into rare disease drug development.
The PRV program is set to expire on September 30, 2024, and without Congressional action, hope for millions of children may vanish.
“It is absolutely critical that Congress reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, which provides a crucial incentive for life science companies to direct research and resources toward drug development for our most vulnerable population – our children,” said Curt Oltmans, Chair of the Rare Disease Company Coalition. “Reauthorization of this program will allow our members to continue discovering, developing, and delivering treatments to children living with a rare disease. The Rare Disease Company Coalition is grateful to champions in the House who are leading the way to reauthorize this life-changing program.”
Rare disease drug development is uniquely challenging, and with those challenges come increased risk. Incentives like the PRV program are crucial to mitigating the financial risks associated with investment in the rare disease space. Without incentives like PRVs, investors may view rare disease drug development as too risky – and for pre commercial companies that rely on capital markets for funding, that could make the difference between bringing a product to market and having to shutter programs. Research shows that rare disease companies are struggling to survive in the current policy and funding ecosystems, and policies like the PRV program are necessary to attract investors that are fundamental to rare disease drug development.
The RDCC applauds the introduction of the Creating Hope Reauthorization Act, and urges Congress to act quickly to reintroduce the Rare Pediatric Disease PRV Program.
###
About the Rare Disease Company Coalition (RDCC): Founded in May 2021, the Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes so that critical innovation can continue and positive changes can be enacted for the rare disease community. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com