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Congressman Ryan Costello

Representing the 6th District of Pennsylvania

On Rare Disease Day, Reps. Costello and Carson Introduce the RARE Act

February 28, 2018
Press Release
Legislation to Support Families Affected by Rare Diseases

Washington, D.C. – Rep. Ryan Costello (R-PA) and Rep. André Carson (D-IN) announced today, on Rare Disease Day, their bipartisan legislation to improve rare disease treatment, awareness, education, research, and diagnosis. While many rare diseases and conditions affect patient populations of fewer than 200,000, almost 7,000 rare diseases affect nearly one in 10 Americans nationwide, over half of whom are children. Many rare diseases and conditions are serious, life-threatening, or lack an effective treatment. Reps. Costello and Carson’s legislation, the Rare Disease Advancement, Research, and Education (RARE) Act, 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,” said Rep. Costello, a member of the Rare Disease Caucus. “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.”

“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 Rep. Carson. “While each patient is unique, Congress can do more to address many of the challenges they face, raising awareness and ultimately helping patients get the treatment and care they need.”

Rep. Costello with constituent advocates from the Rare Disease Legislative Advocates during a trip to Washington, D.C.

Rep. Costello with constituent advocates from the Rare Disease Legislative Advocates during a trip to Washington, D.C.


Reps. Costello and Carson’s legislation, the RARE Act, would:

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.

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.          

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.