Congressman Carson Introduces Legislation to Improve Rare Disease Treatments
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.
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:
National Organization for Rare Disorders
Adult Polyglucosan Body Disease Research Foundation (APBDRF)
All Things Kabuki
American Partnership for Eosinophilic Disorders
Amyloidosis Research Consortium
Association for Frontotemporal Degeneration
Bridge the Gap - SYNGAP Education and Research Foundation
Castleman’s Awareness & Research Effort/Castleman Disease Collaborative Network
Children’s Cardiomyopathy Foundation
Children's PKU Network
CLOVES Syndrome Community
Dystonia Advocacy Network
Dystonia Medical Research Foundation
GBS|CIDP Foundation International
Global Genes - Allies in Rare Disease
Guthy-Jackson Charitable Foundation
HCU Network America
Indian Organization for Rare Diseases
International WAGR Syndrome Association
LAL D Aware
Life Raft Group
Lung Transplant Foundation
Maple Syrup Urine Disease Family Support Group
Moebius Syndrome Foundation
National Adrenal Diseases Foundation
National MPS Society
National PKU Alliance
National PKU News
National Urea Cycle Disorders Foundation
NBIA Disorders Association
NephCure Kidney International
NTM Info & Research
Organic Acidemia Association
Phelan-McDermid Syndrome Foundation
Policy Served Rare
Pulmonary Hypertension Association
Rare and Undiagnosed Network (RUN)
Rare Disease United Foundation
Sofia Sees Hope
Spastic Paraplegia Foundation
Spinal CSF Leak Foundation
The FPIES Foundation
The Myelin Project
Timothy Syndrome Alliance
Tuberous Sclerosis Alliance
US Hereditary Angioedema Association