Proposed RARE Act Seeks to Address Common Challenges Rare Disease Patients Face

Rare Daily Staff

Two Congressmen have introduced the Rare Disease Advancement, Research, and Education (RARE) Act, a bipartisan bill that seeks to address common challenges faced by rare disease patients and their families.

Rep. André Carson, D-Indiana, and Rep. Ryan Costello (R-PA) introduced the RARE Act (HR 5115) on World Rare Disease Day. It is an effort to expand rare disease treatment, awareness, education, research, and diagnosis.

“Constituents and their families who are affected by rare diseases always emphasize the importance of data and research for medical breakthroughs,” said Rep. Costello, a member of the Rare Disease Caucus. “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.” 

The RARE Act would provide funding to enhance and extend the National Institutes of Health’s Rare Disease Clinical Research Network, which consists of 21 researcher centers of excellence focused on rare disease.

The act would require the Centers for Disease Control to create a National Rare Disease or Condition Surveillance System. The system would be modelled on similar systems for other conditions to track rare disease data and help researchers understand commonalities between diseases.

It would require the Agency for Healthcare Research and Quality to expand and intensify its work to ensure that health professionals are aware of rare disease diagnoses and treatments.

In addition, it 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.

More than 50 rare disease organizations have announced their support for the legislation including the National Organization for Rare Disorders, Genetic Alliance, and Global Genes (publisher of Rare Daily).

“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. 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.”

March 5, 2018
Photo: Rep. Ryan Costello with representatives of the Rare Disease Legislative Advocates on World Rare Disease Day