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12/04/2024 | Press release | Archived content

Community Support Letter for the Rare Pediatric Disease PRV Program

Community Support Letter for the Rare Pediatric Disease PRV Program

  • December 4, 2024

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 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) 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 [email protected] and Hayley Mason, Policy Analyst with the National Organization for Rare Disorders, at [email protected] with any questions.

Sincerely,

EveryLife Foundation for Rare Diseases

Canavan Foundation

National Organization for Rare Disorders

CDH International

Acromegaly Community Inc.

Center for Innovation & Value Research

Adrenal Insufficiency United

Charcot Marie Tooth Research Foundation

Advocates for Medically Fragile Kids NC

Child Neurology Foundation

Aimed Alliance

Chondrosarcoma CS Foundation, Inc.

Aislinn's Wish Foundation

Coalition to Cure Calpain 3

Alagille Syndrome Alliance

Coalition to Cure CHD2

Alpha-1 Foundation

COMBINEDBrain

Alport Syndrome Foundation

Congenital Hyperinsulinism International

AMDA

Conquer MG

American Kidney Fund

Cooley's Anemia Foundation

Angelman Syndrome Foundation

CSNK2A1 FOUNDATION

APBDRFoundation

CTNNB1 Connect and Cure

Aplastic Anemia and MDS International Foundation

Cure CMD

ASXL Rare Research Endowment

CURE GABA-A

Autism Science Foundation

Cure GM1 Foundation

Avery's Hope

Cure KCNH1 Foundation

BARE Inc

Cure Lowe Foundation

Barth Syndrome Foundation

Cure Mito Foundation

BDSRA Foundation

Cure Sanfilippo Foundation

BPAN WARRIORS

Cure SMA

Bubba's Light

CureARS

CA Action Link for Rare Diseases (Cal Rare)

CURED Nfp (Campaign Urging Research for Eosinophilic Diseases)

CACNA1A Foundation

CureLGMD2i

CureSHANK

Hydrocephalus Association

Cyclic Vomiting Syndrome Association

HypoPARAthyroidism Association

Cystic Fibrosis Research Institute

Immune Deficiency Foundation

Dana's Angels Research Trust

INADcure Foundation

debra of America

Indo US Organization for Rare Diseases (IndoUSrare)

Dion Foundation for Children with Rare Diseases

International Fibrodysplasia Ossificans Progressiva (FOP) Association

Dravet Syndrome Foundation

International Foundation for CDKL5 Research

Dreamsickle Kids Foundation, Inc.

International Rett Syndrome Foundation

Dup15q Alliance

International Waldenstrom's Macroglobulinemia Foundation

EB Research Partnership

Jack McGovern Coats' Disease Foundation

End AxD

Jansen's Foundation

Endosalpingiosis Foundation INC

Jordan's Guardian Angels

Eosinophilic & Rare Disease Cooperative

Juju and Friends CLN2 Warrior Foundation

Epilepsy Foundation of America

Kabuki Syndrome Foundation

Fabry Support & Information Group

KCNQ2 Cure Alliance

FAM177A1 RESEARCH FUND

Koolen-de Vries Syndrome Foundation

Familial Dysautonomia Foundation

Krabbe Connect

Family Heart Foundation

Krishnan Family Foundation

FD/MAS Alliance

Lambert Eaton LEMS Family Association

Fighting H.A.R.D. Foundation

Lennox-Gastaut Syndrome (LGS) Foundation

flok Health

Leukodystrophy Newborn Screening Action Network

Fondazione Telethon

LGMD Awareness Foundation, Inc

Foundation for Angelman Syndrome Therapeutics (FAST)

LGMD2D Foundation

Foundation for Prader-Willi Research

Li-Fraumeni Syndrome Association (LFS Association)

Foundation to Fight H-abc

Little Hercules Foundation

FRAXA Research Foundation

Lung Transplant Foundation

Friedreich's Ataxia Research Alliance (FARA)

MECP2 Duplication Foundation

GABA-A Alliance

Mellie J Foundation

Galactosemia Foundation

Mission: Cure

Gaucher Community Alliance

Mississippi Metabolics Foundation

GBS|CIDP Foundation International

MitoAction

Gene Giraffe Project

MLD Foundation

Global Genes

MSUD Family Support Group

Global Liver Institute

MTM-CNM Family Connection

Glut1 Deficiency Foundation

Muscular Dystrophy Association

Haystack Project

Myasthenia Gravis Association

HCU Network America

Myositis Support and Understanding

HD-CARE - Huntington's Disease Community Advocacy & Education

N=1Collaborative

Hemophilia Foundation of Southern California

National Alliance for Caregiving

Hermansky-Pudlak Syndrome Network

National Alliance for PANS/PANDAS Action

Hope for Hypothalamic Hamartomas

National Ataxia Foundation

Hope in Focus

National Eosinophilia Myalgia Syndrome Network

Huntington's Disease Society of America

National Fragile X Foundation

National Health Council

Superior Mesenteric Artery Syndrome Research Awareness and Support

National Kidney Foundation

Supporters of Families with Sickle Cell Disease, Inc.

National MPS Society

SynGAP Research Fund, DBA cureSYNGAP1

National Tay-Sachs & Allied Diseases Association

Taylor's Tale

NBIA Disorders Association

Team Telomere

Necrotizing Enterocolitis (NEC) Society

The Association for Frontotemporal Degeneration

Neev Kolte & Brave Ronil Foundation

The Bluefield Project to Cure FTD

NephCure

The Bonnell Foundation: Living with cystic fibrosis

Noah's Hope - Hope4Bridget

The Children's Medical Research Foundation, Inc.

NTM Info & Research, Inc.

The DDX3X Foundation

NW Rare Disease Coalition

The E.WE Foundation

Ogden CARES

The Global Foundation for Peroxisomal Disorders

Organic Acidemia Association

THE KAT6 FOUNDATION INC

Parent Project Muscular Dystrophy

The LAM Foundation

Partnership to Fight Chronic Disease

The Little Legs Big Heart Foundation

Pathways for Rare and Orphan Solutions

The Louisa Adelynn Johnson Fund for Complex Disease

Petronille Healthy Society

The MED13L Foundation Inc.

PMD Foundation

The Mended Hearts, Inc.

Pompe Alliance

The National Adrenal Diseases Foundation

Project Alive

The National PKU Alliance

PTEN Hamartoma Tumor Syndrome Foundation

The Oxalosis and Hyperoxaluria Foundation

PWSA | USA - Prader-Willi Syndrome Association

The RYR-1 Foundation

Rare New England

The Akari Foundation

Rare Trait Hope Fund

Tough Genes

RareRising

TSC Alliance

Raymond A. Wood Foundation

U.R. Our Hope

Rett Syndrome Research Trust

Undiagnosed Diseases Network Foundation

Sanfilippo Children's Foundation

United Mitochondrial Disease Foundation

SANFILIPPO SUD

United MSD Foundation

SATB2 Gene Foundation

United Ostomy Associations of America, Inc.

SCAD Alliance

United Porphyrias Association

SHINE Syndrome Foundation

Uriel E. Owens Sickle Cell Disease Association of the Midwest

Shwachman-Diamond Syndrome Alliance Inc

Vasculitis Foundation

Sickle cell association of Kentuckiana

Wake Up Narcolepsy, Inc.

Sisters Hope Foundation

Wilson Disease Association

Sleep Consortium

Wisconsin Rare Disease Alliance

SMS Research Foundation

Wylder Nation Foundation

Spina Bifida Association

ZTTK SON-Shine Foundation

Stronger Than Sarcoidosis

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