December 4, 2024
| The Honorable Chuck Schumer Majority Leader United States Senate 322 Hart Senate Office Building Washington, D.C. 20510 The Honorable Mitch McConnell Minority Leader United States Senate 317 Russell Senate Office Building Washington, D.C. 20510 | The Honorable Mike Johnson Speaker of the House United States House of Representatives 568 Cannon House Office Building Washington, D.C. 20515 The Honorable Hakeem Jeffries Minority Leader United States House of Representatives 2433 Rayburn House Office Building 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 118th 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 20th, 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) score 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,
| EveryLife Foundation for Rare Diseases National Organization for Rare Disorders Acromegaly Community Inc. Adrenal Insufficiency United Advocates for Medically Fragile Kids NC Aimed Alliance Aislinn’s Wish Foundation Alagille Syndrome Alliance Alpha-1 Foundation Alport Syndrome Foundation AMDA American Kidney Fund Angelman Syndrome Foundation APBDRFoundation Aplastic Anemia and MDS International Foundation ASXL Rare Research Endowment Autism Science Foundation Avery’s Hope BARE Inc Barth Syndrome Foundation BDSRA Foundation BPAN WARRIORS Bubba’s Light CA Action Link for Rare Diseases (Cal Rare) CACNA1A Foundation Canavan Foundation CDH International Center for Innovation & Value Research Charcot Marie Tooth Research Foundation Child Neurology Foundation Chondrosarcoma CS Foundation, Inc. Coalition to Cure Calpain 3 Coalition to Cure CHD2 COMBINEDBrain Congenital Hyperinsulinism International Conquer MG Cooley’s Anemia Foundation CSNK2A1 FOUNDATION CTNNB1 Connect and Cure Cure CMD CURE GABA-A Cure GM1 Foundation Cure KCNH1 Foundation Cure Lowe Foundation Cure Mito Foundation Cure Sanfilippo Foundation Cure SMA CureARS CURED Nfp (Campaign Urging Research for Eosinophilic Diseases) CureLGMD2i CureSHANK Cyclic Vomiting Syndrome Association Cystic Fibrosis Research Institute Dana’s Angels Research Trust debra of America Dion Foundation for Children with Rare Diseases Dravet Syndrome Foundation Dreamsickle Kids Foundation, Inc. Dup15q Alliance EB Research Partnership End AxD Endosalpingiosis Foundation INC Eosinophilic & Rare Disease Cooperative Epilepsy Foundation of America Fabry Support & Information Group FAM177A1 RESEARCH FUND Familial Dysautonomia Foundation Family Heart Foundation FD/MAS Alliance Fighting H.A.R.D. Foundation flok Health Fondazione Telethon Foundation for Angelman Syndrome Therapeutics (FAST) Foundation for Prader-Willi Research Foundation to Fight H-abc FRAXA Research Foundation Friedreich’s Ataxia Research Alliance (FARA) GABA-A Alliance Galactosemia Foundation Gaucher Community Alliance GBS|CIDP Foundation International Gene Giraffe Project Global Genes Global Liver Institute Glut1 Deficiency Foundation Haystack Project HCU Network America HD-CARE – Huntington’s Disease Community Advocacy & Education Hemophilia Foundation of Southern California Hermansky-Pudlak Syndrome Network Hope for Hypothalamic Hamartomas Hope in Focus Huntington’s Disease Society of America Hydrocephalus Association HypoPARAthyroidism Association Immune Deficiency Foundation INADcure Foundation Indo US Organization for Rare Diseases (IndoUSrare) International Fibrodysplasia Ossificans Progressiva (FOP) Association International Foundation for CDKL5 Research International Rett Syndrome Foundation International Waldenstrom’s Macroglobulinemia Foundation Jack McGovern Coats’ Disease Foundation Jansen’s Foundation Jordan’s Guardian Angels Juju and Friends CLN2 Warrior Foundation Kabuki Syndrome Foundation KCNQ2 Cure Alliance Koolen-de Vries Syndrome Foundation | Krabbe Connect Krishnan Family Foundation Lambert Eaton LEMS Family Association Lennox-Gastaut Syndrome (LGS) Foundation Leukodystrophy Newborn Screening Action Network LGMD Awareness Foundation, Inc LGMD2D Foundation Li-Fraumeni Syndrome Association (LFS Association) Little Hercules Foundation Lung Transplant Foundation MECP2 Duplication Foundation Mellie J Foundation Mission: Cure Mississippi Metabolics Foundation MitoAction MLD Foundation MSUD Family Support Group MTM-CNM Family Connection Muscular Dystrophy Association Myasthenia Gravis Association Myositis Support and Understanding N=1Collaborative National Alliance for Caregiving National Alliance for PANS/PANDAS Action National Ataxia Foundation National Eosinophilia Myalgia Syndrome Network National Fragile X Foundation National Health Council National Kidney Foundation National MPS Society National Tay-Sachs & Allied Diseases Association NBIA Disorders Association Necrotizing Enterocolitis (NEC) Society Neev Kolte & Brave Ronil Foundation NephCure Noah’s Hope – Hope4Bridget NTM Info & Research, Inc. NW Rare Disease Coalition Ogden CARES Organic Acidemia Association Parent Project Muscular Dystrophy Partnership to Fight Chronic Disease Pathways for Rare and Orphan Solutions Petronille Healthy Society PMD Foundation Pompe Alliance Project Alive PTEN Hamartoma Tumor Syndrome Foundation PWSA | USA – Prader-Willi Syndrome Association Rare New England Rare Trait Hope Fund RareRising Raymond A. Wood Foundation Rett Syndrome Research Trust Sanfilippo Children’s Foundation SANFILIPPO SUD SATB2 Gene Foundation SCAD Alliance SHINE Syndrome Foundation Shwachman-Diamond Syndrome Alliance Inc Sickle cell association of Kentuckiana Sisters Hope Foundation Sleep Consortium SMS Research Foundation Spina Bifida Association Stronger Than Sarcoidosis Superior Mesenteric Artery Syndrome Research Awareness and Support Supporters of Families with Sickle Cell Disease, Inc. SynGAP Research Fund, DBA cureSYNGAP1 Taylor’s Tale Team Telomere The Association for Frontotemporal Degeneration The Bluefield Project to Cure FTD The Bonnell Foundation: Living with cystic fibrosis The Children’s Medical Research Foundation, Inc. The DDX3X Foundation The E.WE Foundation The Global Foundation for Peroxisomal Disorders THE KAT6 FOUNDATION INC The LAM Foundation The Little Legs Big Heart Foundation The Louisa Adelynn Johnson Fund for Complex Disease The MED13L Foundation Inc. The Mended Hearts, Inc. The National Adrenal Diseases Foundation The National PKU Alliance The Oxalosis and Hyperoxaluria Foundation The RYR-1 Foundation The Akari Foundation Tough Genes TSC Alliance U.R. Our Hope Undiagnosed Diseases Network Foundation United Mitochondrial Disease Foundation United MSD Foundation United Ostomy Associations of America, Inc. United Porphyrias Association Uriel E. Owens Sickle Cell Disease Association of the Midwest Vasculitis Foundation Wake Up Narcolepsy, Inc. Wilson Disease Association Wisconsin Rare Disease Alliance Wylder Nation Foundation ZTTK SON-Shine Foundation |
Read more from the original letter here.
