Community Support Letter for the Rare Pediatric Disease PRV Program (PDF)

The Honorable Chuck Schumer Majority Leader                                        The Honorable Mike Johnson
Minority Leader                                                                                                     Speaker of the House
United States Senate.                                                                                          United States House of Representatives 
322 Hart Senate Office Building                                                                       568 Cannon House Office Building 
Washington, D.C. 20510                                                                                      Washington, D.C. 20510

The Honorable Mitch McConnell                                                                      The Honorable Hakeem Jeffries 
Minority Leader                                                                                                     Minority Leader
United States Senate                                                                                           United States House of Representatives 
317 Russell Senate Office Building                                                                   2433 Rayburn House Office Building 
Washington, D.C. 20510.                                                                                      Washington, D.C. 20515

Dear Majority Leader Schumer, and Minority Leader McConnell, Speaker Johnson, Minority Leader Jeffries,

As you work to finalize legislation for consideration before the end of the 118^th Congress, the undersigned 213 organizations urge you to pass or include within any larger bill the provisions of the Creating Hope Reauthorization Act (H.R. 7384/S. 4583), which would reauthorize the highly effective Rare Pediatric Disease Priority Review Voucher (PRV) program for at least five years. Reauthorizing the Rare Pediatric Disease PRV program has broad bipartisan support, including approval by the full House of Representatives in September as part of the amended Give Kids a Chance Act (H.R. 3433). The current authorization is set to expire on December 20^th, and a timely, clean and long-term reauthorization is critical to maintaining this important incentive which has effectively spurred drug development to help children living with rare diseases.

Since its creation by Congress in 2012, the Rare Pediatric Disease PRV program has helped bring to market therapies for children affected by 39 rare diseases.¹ Without treatment, many of these diseases lead to death or debilitating illness before the children reach adulthood, and only three of these rare diseases had a safe and effective FDA-approved therapy on the market before the program began.² Additionally, more than half of all Rare Pediatric Disease PRV designations occurred in the last four years,³ showing the program is fostering robust drug development where significant unmet therapeutic needs currently exist.

Developing drugs for rare pediatric diseases often presents unique challenges, including small patient populations and difficulties conducting pediatric clinical trials. The incentive established by the Rare Pediatric Disease PRV program is simple and according to the Congressional Budget Office (CBO) score⁴

would not have any effect on direct federal spending: if a product manufacturer develops an FDA- approved therapy to treat a rare pediatric disease, the company earns a transferable priority review voucher – and the right to a more expeditious FDA review timeline – that can be used for a subsequent product application or can be sold and transferred to another company. If the Rare Pediatric Disease PRV program is not reauthorized by Congress, a key incentive that has effectively helped bring treatments and cures to kids and their families will end. We cannot afford to let this happen.

As noted, a five-year extension of the Rare Pediatric Disease PRV program passed unanimously by the House of Representatives and has garnered significant bipartisan support. It is also widely supported by the rare disease patient community, with nearly 200 patient organizations signing on to a letter of support for the bill this summer. We urge that you build upon the work done to date and pass a timely, clean and long-term reauthorization to ensure promising science can be translated into treatments and hope for children and families affected by rare diseases.

Thank you for considering this request and please don’t hesitate to reach out to Jamie Sullivan at the EveryLife Foundation for Rare Diseases, at jsullivan@everylifefoundation.org and Hayley Mason, Policy Analyst with the National Organization for Rare Disorders, at HMason@rarediseases.org with any questions.

Sincerely,

Cc:       The Honorable Bernie Sanders, Chairman, Senate Committee on Health, Education, Labor and Pensions
The Honorable Bill Cassidy, Ranking Member, Senate Committee on Health, Education, Labor and Pensions
The Honorable Cathy McMorris Rodgers, Chair, House Committee on Energy and Commerce
The Honorable Frank Pallone, Ranking Member, House Committee on Energy and Commerce
The Honorable Robert Casey, Lead Sponsor, Creating Hope Reauthorization Act
The Honorable Markwayne Mullin, Lead Cosponsor, Creating Hope Reauthorization Act The Honorable Sherrod Brown, Lead Cosponsor, Creating Hope Reauthorization Act The Honorable Susan Collins, Lead Cosponsor, Creating Hope Reauthorization Act
The Honorable Michael McCaul, Lead Sponsor, Creating Hope Reauthorization Act The Honorable Anna Eshoo, Lead Cosponsor, Creating Hope Reauthorization Act The Honorable Gus Bilirakis, Lead Cosponsor, Creating Hope Reauthorization Act
The Honorable Nanette Barragan, Lead Cosponsor, Creating Hope Reauthorization Act The Honorable Lori Trahan, Lead Cosponsor, Creating Hope Reauthorization Act
The Honorable Michael Burgess, Lead Cosponsor, Creating Hope Reauthorization Act

¹ See: https://rarediseases.org/wp-content/uploads/2024/05/NORD_PRV-white-paper_FINAL.pdf

² Ibid.

³ Mease, C., Miller, K. L., Fermaglich, L. J., Best, J., Liu, G., & Torjusen, E. (2024). Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development. Orphanet Journal of Rare Diseases. https://link.springer.com/epdf/10.1186/s13023-024-03097 x03097x sharing_token=tVsdcxtCuGoLKGG18G02G_BpE1tBhCbnbw3BuzI2ROyCDnBKl_41BmSn3a_5qrzjgrL XsufvRX0wtQEn ALK9Za3v_5zjNTa3quYxLJ0LC4dnFV94TbHqovQ6Vq5sRWu7_u2v1C7h16jaeLChSswkyx4eSqy_KycTNie1 qfGSM

⁴ https://www.cbo.gov/system/files/2024-09/suspensions_week_of_9_23_2024_1.pdf