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Congressman Carson Introduces Legislation to Improve Rare Disease Treatments

Feb 28, 2018
Press Release

WASHINGTON, DC - Today, on Rare Disease Day, Congressman André Carson (D-IN) announced his renewed, bipartisan efforts to improve rare disease treatment, awareness, education, research, and diagnosis.

Currently, almost 7,000 rare diseases affect nearly one in 10 Americans nationwide, over half of whom are children. Unfortunately, many rare diseases and conditions, which affect small patient populations fewer than 200,000, are serious, life-threatening, or lack an effective treatment.

“Rare diseases impact patients and families in Indianapolis and across the country, and too often they are unable to find a treatment, a cure, or even an accurate diagnosis,” said Congressman André Carson. “While each patient is unique, Congress can do more to address many of the challenges they face, raise awareness and ultimately help patients get the treatment and care they need.”

Today, Congressman André Carson and Congressman Ryan Costello (R-PA) introduced the Rare disease Advancement, Research, and Education (RARE) Act, which aims to address many of the common challenges faced by rare disease patients and their families.

“Constituents and their families who are affected by rare diseases always emphasize the importance of data and research for medical breakthroughs. We should be doing everything we can to accelerate innovation that could result in more effective treatments, the discovery of cures, and more awareness of rare diseases. I am proud to be a supportive voice in Congress for those facing a rare disease and believe this comprehensive legislation will be a critical step forward in helping patients,” said Rep. Costello, a member of the Rare Disease Caucus.

Separately, Congressman André Carson and Congressman Michael McCaul (R-TX) also led many of their colleagues in a bipartisan letter today. The letter urges Congressional leadership to formally recognize February 28 as Rare Disease Day, in line with legislation introduced last year by Congressmen Carson and McCaul. Rare Disease Day is already celebrated each year by rare disease patients and their families, to share their stories and educate their communities.

Congressmen Carson and Costello’s new legislation, the RARE Act, would:

1.      Rare Diseases Clinical Research Network/Centers of Excellence
The RARE Act would enhance an existing and successful program of the National Institutes of Health (NIH), the Rare Diseases Clinical Research Network. This unique Network is made up of 21 research ‘centers of excellence’ studying rare diseases in an interdisciplinary way, working with patients and others on clinical studies and other research. The bill would increase and extend the program’s funding authorization.


2.      Surveillance of Rare Diseases
The RARE Act would require the Centers for Disease Control (CDC) to create a National Rare Disease or Condition Surveillance System. Modelled off of similar systems for other conditions, this formalized infrastructure would track rare disease data, helping researchers to understand commonalities between diseases.


3.      Health Professionals’ Awareness of Rare Diseases
The RARE Act would require the Agency for Healthcare Research and Quality (AHRQ) to expand and intensify its work to ensure that health professionals are aware of rare disease diagnoses and treatments.


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

The following foundations and organizations have announced their support for the Rare disease Advancement, Research, and Education (RARE) Act:

A Twist of Fate-ATS
ADNP Kids Research Foundation
Adrenal Insufficiency United
Adult Polyglucosan Body Disease Research Foundation
Adult Polyglucosan Body Disease Research Foundation (APBDRF)
Alagille Syndrome Alliance
All Things Kabuki
Alport Syndrome Foundation
ALS Association
American Behcet’s Disease Association
American Multiple Endocrine Neoplasia Support
American Partnership for Eosinophilic Disorders
American Partnership for Eosinophilic Disorders (APFED)
Amyloidosis Foundation
Amyloidosis Research Consortium
Angelman Biomarkers and Outcome Measures Alliance
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
Congenital Hyperinsulinism International
Cure SMA
CureCMT4J/Talia Duff Foundation
Cutaneous Lymphoma Foundation
DC Outreach
Defeat MSA: Defeat Multiple System Atrophy
Dravet Syndrome Foundation
Dysautonomia Foundation
Dystonia Advocacy Network
Dystonia Medical Research Foundation
Family Caregiver Alliance
Family Voices NJ
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
Guthy-Jackson Charitable Foundation
HCU Network America
Hermansky-Pudlak Syndrome Network
Histiocytosis Association
HSAN1E Society
Hydrocephalus Association
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)
Jack McGovern Coats' Disease Foundation
Jonah's Just Begun - Foundation to Cure Sanfilippo Inc.
LAL D Aware
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
Marfan Foundation
M-CM Network
MDS Foundation
MLD Foundation
Moebius Syndrome Foundation
Mucolipidosis Type IV 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 Organization for Rare Disorders (NORD)
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
NTM Info & Research
Oley Foundation
Organic Acidemia Association
Osteogenesis Imperfecta Foundation
Pathways for Rare and Orphan Studies
Phelan-McDermid Syndrome Foundation
PKD Foundation
Platelet Disorder Support Association
Policy Served Rare
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., The U.S. Hypophosphatasia Foundation
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 MSA Awareness Shoe
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