Sending Office: Honorable Andre Carson
Sent By:
Ryan.Shay@mail.house.gov

        Request for Cosponsor(s)

Cosponsors (35): Axne, Beatty, Blunt Rochester, Brooks, S. (IN), Brownley,
Bucshon, Carson, Correa, DeFazio, Deutch, Fitzpatrick, Harder, Hudson, Jackson Lee, Johnson, E.B. (TX), Khanna, King, P. (NY), Luján, Maloney, C. (NY),  Maloney, S. (NY), Moore, Murphy, Norton, Payne Jr., Pocan, Raskin, Rouda,
Ruppersberger, Rush, Rutherford, Soto, Suozzi, Visclosky, Walorski, Welch

Dear Colleague,

Please join Rep. Richard Hudson (NC-R-8) and I and become a cosponsor of the
Rare diseases Advancement, Research, and Education (RARE) Act (H.R. 4228) in the 116th Congress. This bipartisan bill will expand the ability of the National Institutes of Health (NIH) and Centers for Diseases Control and Prevention (CDC)
to study rare diseases by improving treatment, research, and diagnostics of rare diseases through new and existing programs. This legislation has been endorsed by 140 patient groups.

Nearly one in ten Americans live with one or more of the roughly 7,000 known rare diseases. These largely inherited diseases – defined as affecting 200,000 or fewer people – often lack substantive research investments and treatment options. While rare diseases
cross the medical spectrum, individuals with rare diseases face some common challenges. Largely due to their limited patient population size, these individuals may have difficulty obtaining an accurate diagnosis, finding physicians or treatment centers with
expertise in their disease, and ultimately finding appropriate treatment options and cures. While over 450 drugs have been approved for the treatment of rare diseases, millions of Americans suffering from a rare disease have no approved treatment options.

While past Congressional action has helped support research at NIH and CDC, much more work needs to be done to help these agencies improve rare disease awareness, education, research, surveillance, diagnosis, and treatment. As a result, we are introducing
the RARE Act to improve NIH and CDC’s ability to study rare diseases by improving treatment, research, and diagnostics of rare diseases through existing and new programs.

The Rare disease Advancement, Research, and Education (RARE) Act would improve:

•    Research: Enhance Rare Diseases Clinical Research Network/Centers of Excellence

The RARE Act would expand an existing and successful program at NIH: the Rare Diseases Clinical Research Network (RDCRN). The RDCRN’s 21 research “centers of excellence” support the research and clinical trials of over 190 rare diseases and increase
the availability of rare disease information.

•    Data: Create a Rare Disease Surveillance System at the CDC

By creating a new CDC surveillance system, the RARE Act would help fill critical gaps in tracking rare disease epidemiology and data and further help researchers understand commonalities between diseases.

•    Awareness: Increase Health Professionals’ Awareness of Rare Diseases

The RARE Act would increase health care providers’ awareness and knowledge of rare diseases through added engagement from the Agency for Healthcare Research and Quality (AHRQ).

•    Policies: Require an Updated Report from National Academics of Sciences, Engineering and Medicine

The RARE Act would require that the National Academies of Sciences, Engineering, and Medicine update its 2010 report “Rare Diseases and Orphan Products: Accelerating Research and Development” in order to further evaluate rare disease efforts and
make further recommendations to policymakers.

Funding: The RARE Act would authorize “as such sums are necessary” for expanding the Rare Diseases Clinical Research Network (RDCRN) at NIH. The bill would authorize $10 million each fiscal year from FY2020 to FY2024 for the creation of National
Rare Disease or Condition Surveillance System to facilitate rare disease research by tracking diseases at CDC. The bill would also authorize $1 million for the updating of the Rare Diseases and Orphan Products: Accelerating Research and Development report
at the National Academies of Sciences, Engineering, and Medicine.

We hope you will join us as a cosponsor of the RARE Act. Please feel free to review our

one-pager and the
bill text. Please contact Ryan Shay at
ryan.shay@mail.house.gov if you have questions or would like to cosponsor.

Sincerely,
André Carson                                              

Endorsements:

• A Twist of Fate-ATS
• ADNP Kids Research Foundation
• Adrenal Insufficiency United
• All Things Kabuki
• Adult Polyglucosan Body Disease Research Foundation (APBDRF)
• Alagille Syndrome Alliance
• Alport Syndrome Foundation
• ALS Association
• American Behcet’s Disease Association
• American Multiple Endocrine Neoplasia Support
• American Partnership for Eosinophilic Disorders (APFED)
• Amyloidosis Foundation
• Amyloidosis Research Consortium
• Aplastic Anemia and MDS International Foundation
• APS Type 1 Foundation, Inc.
• Association for Creatine Deficiencies
• Association for Frontotemporal Degeneration
• Association of Gastrointestinal Motility Disorders, Inc. (AGMD)
• Autoimmune Hepatitis Association
• Autoinflammatory Alliance
• Batten Disease Support and Research Association
• Bridge the Gap – SYNGAP Education and Research Foundation
• Castleman’s Awareness & Research Effort/Castleman Disease Collaborative Network
• CdLS Foundation
• Central Pain Nerve Center
• Charcot-Marie-Tooth Association
• Children’s Cardiomyopathy Foundation
• Children’s PKU Network
• Children’s Tumor Foundation
• Chloe Barnes Rare Disease Foundation
• Cholangiocarcinoma Foundation
• CLOVES Syndrome Community
• CMTC-OVM
• Cure SMA
• Congenital Hyperinsulinism International
• CureCMT4J/Talia Duff Foundation
• CurePSP
• Cutaneous Lymphoma Foundation
• Dravet Syndrome Foundation
• Dysautonomia Foundation
• Dystonia Advocacy Network
• Family Caregiver Alliance
• Fibrous Dysplasia Foundation
• Foundation Fighting Blindness
• Foundation for Prader-Willi Research
• Friedreich’s Ataxia Research Alliance (FARA)
• Galactosemia Foundation
• GBS|CIDP Foundation International
• Genetic Alliance
• Global Genes – Allies in Rare Disease
• Glut1 Deficiency Foundation
• Gut Check Foundation
• Guthy-Jackson Charitable Foundation
• HCU Network America
• Hermansky-Pudlak Syndrome Network
• Histiocytosis Association
• HSAN1E Society
• Hydrocephalus Association
• Hypersomnia Foundation
• Immune Deficiency Foundation
• Indian Organization for Rare Diseases
• International Fibrodysplasia Ossificans Progressiva (FOP) Association
• International Pemphigus and Pemphigoid Foundation
• International WAGR Syndrome Association
• International Waldenstrom’s Macroglobulinemia Foundation (IWMF)
• KIF1A.ORG
• LAL Solace
• LAM Foundation
• Life Raft Group
• Li-Fraumeni Syndrome Association (LFS Association / LFSA)
• Lung Transplant Foundation
• Lymphangiomatosis & Gorham’s Disease Alliance
• Maple Syrup Urine Disease Family Support Group
• M-CM Network
• MDS Foundation
• MitoAction
• MLD Foundation
• Moebius Syndrome Foundation
• Multiple System Atrophy Coalition
• Myasthenia Gravis Foundation of America
• Myositis Association
• Myotonic Dystrophy Foundation
• National Adrenal Diseases Foundation
• National Foundation for Ectodermal Dysplasias
• National MPS Society
• National Organization for Albinism and Hypopigmentation
• National PKU Alliance
• National PKU News
• National Spasmodic Dysphonia Association
• National Urea Cycle Disorders Foundation
• NBIA Disorders Association
• NephCure Kidney International
• Neurofibromatosis Network
• Neurofibromatosis Northeast
• NGLY1.org
• NTM Info & Research
• Oley Foundation
• Organic Acidemia Association
• Osteogenesis Imperfecta Foundation
• Peter Morley, Patient Advocate
• Phelan-McDermid Syndrome Foundation
• PKD Foundation
• Platelet Disorder Support Association
• Prader-Willi Syndrome Association (USA)
• PRP Alliance, Pulmonary Fibrosis Foundation
• Pulmonary Hypertension Association, PXE International
• Rare and Undiagnosed Network (RUN)
• Rare Disease United Foundation
• RASopathies Network, RYR-1 Foundation
• SADS Foundation, Scleroderma Foundation
• Shwachman-Diamond Syndrome Foundation
• Sofia Sees Hope, Soft Bones, Inc.
• SPAN Parent Advocacy Network
• Spastic Paraplegia Foundation
• Spinal CSF Leak Foundation
• SSADH Association
• Sturge-Weber Foundation
• TargetCancer Foundation
• Tarlov Cyst Disease Foundation
• Team Audrey HLH
• The Desmoid Tumor Research Foundation
• The FPIES Foundation
• The Global Foundation for Peroxisomal Disorders
• The Guthy-Jackson Charitable Foundation
• The Hyper IgM Foundation
• The Jansen’s Foundation
• The LAM Foundation
• The Morgan Leary Vaughan Fund, Inc.
• The Myelin Project
• The Myocarditis Foundation
• The Snyder-Robinson Foundation
• The Transverse Myelitis Association
• The XLH Network, Inc.
• Timothy Syndrome Alliance
• Tuberous Sclerosis Alliance
• United Leukodystrophy Foundation
• US Hereditary Angioedema Association
• Vasculitis Foundation, VHL Alliance
• Wilhelm Foundation – the Undiagnosed
• Williams Syndrome Association.

 

Ryan Shay
Senior Legislative Assistant | Office of Congressman André Carson (IN-07)
2135 Rayburn House Office Building | Washington, D.C. 20515
Phone 202.225.4011 | Carson.House.Gov

Related Legislative Issues Selected legislative information: Consumer Affairs, Family Issues, Government, HealthCare, Science, Technology Related Bill Information Bill Type: H.R. Bill Type: 4228 Special Note:

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