Mobile Menu - OpenMobile Menu - Closed


Bilirakis and Butterfield Introduce Resolution to Celebrate the Success of the Orphan Drug Act

March 19, 2019
Press Release

WASHINGTON, DC – Congressman G. K. Butterfield (NC-01) and Gus Bilirakis (FL-12), co-chairs of the Rare Disease Congressional Caucus have introduced H.Res. 242 which celebrates the Orphan Drug Act and the over 750 therapies for rare diseases approved since the Act’s enactment.  The Congressmen announced this Resolution shortly after Rare Disease Day was celebrated by hundreds of advocates across the country.


The Resolution celebrates the success of the Orphan Drug Act and acknowledges the Act’s continued importance for rare disease patients.  Over 750 new treatments for rare diseases have been brought to market as a result of the Act, whereas there were only 34 treatments prior to the Act’s enactment.


“The Orphan Drug Act has been remarkably successful in spurring therapeutic development for the approximately 25 to 30 million Americans with a rare disease, including many constituents of North Carolina’s First Congressional District,” said Congressman Butterfield. “However, with over 90 percent of rare diseases still without an FDA-approved treatment, there is still much work to be done.  This is why the Orphan Drug Act remains an important law for millions of Americans still seeking their very first treatment.”


“Rare diseases are not a rare problem; they impact the lives of millions of Americans.  We must continue to support successful initiatives, like the Orphan Disease Act, which spur the development of treatments and pave the path to cures for rare diseases.  I look forward to continuing this work in a bipartisan manner building upon our recent success with 21st Century Cures to accelerate the development of innovative treatments and cures for these patients,” said Congressman Bilirakis.


The Resolution underscores the importance of the market-based incentives within the Orphan Drug Act that have encouraged private investment into otherwise economically unviable treatments and cures.  These incentives must remain strong so that they can continue to accelerate the development of therapies for the rare disease community.


H.Res. 242 is strongly supported by the rare disease patient community.  Peter Saltonstall, the President and CEO of the National Organization for Rare Disorders (NORD), added, “We at NORD congratulate Congressmen Butterfield and Bilirakis, as well as their many supporting colleagues, for introducing this resolution.  It’s crucial that we as a community not only celebrate the successes of the Orphan Drug Act, but acknowledge its importance for the millions of Americans with a rare disease still awaiting their very first treatment.”


As Co-Chairmen of the Rare Disease Congressional Caucus, Congressmen Butterfield and Bilirakis are leading the charge in the fight for access to affordable treatments for the rare disease community.