U.S. Rep. Ryan Costello (R-PA) on Feb. 28 — dubbed worldwide as Rare Disease Day 2018 — symbolically announced a proposed bill to enhance research, surveillance and reporting of rare diseases, which the National Institutes of Health (NIH) estimate affect between 25 million and 30 million Americans, or nearly one in 10 people nationwide.
The bipartisan Rare Disease Advancement, Research, and Education (RARE) Act, H.R. 5115, which Costello and U.S. Rep. Andre Carson (D-IN) officially introduced on Feb. 27, also would support improvements to rare disease treatments, education, federal awareness efforts, and diagnosis, according to a summary provided by Costello’s office, which noted that many rare diseases and conditions are life-threatening and lack successful remedies.
“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 Costello, a member of the bipartisan, bicameral Rare Disease Congressional Caucus, which includes two Republican co-chairs, U.S. Sen. Orrin Hatch (R-UT) and U.S. Rep. Leonard Lance (R-NJ), who serve alongside Democratic co-chairs Sen. Amy Klobuchar (D-MN) and Rep. G.K. Butterfield (D-NC).
Among several provisions in the summary, H.R. 5115 would bolster and extend funding for the Rare Diseases Clinical Research Network, an NIH-funded initiative comprised of 21 disease research groups conducting interdisciplinary study on rare diseases. Additionally, the bill would task the Centers for Disease Control and Prevention with establishing a nationwide surveillance system to track and record data on rare diseases for research use.
Rep. Carson said H.R. 5115 — which also would ramp up federal efforts to alert more health professionals to related diagnoses and treatments — is designed to help those patients who too often “are unable to find a treatment, a cure, or even an accurate diagnosis. 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.”
H.R. 5115 has been referred to the House Energy and Commerce Committee for consideration.