Advocates Cheer Senate Leaders for Introducing Bipartisan Bill to Increase Number of Rare Disease Treatments

News provided by

EveryLife Foundation for Rare Diseases

May 21, 2015, 12:19 ET

NOVATO, Calif., May 21, 2015 /PRNewswire-USNewswire/ -- The EveryLife Foundation for Rare Diseases applauded Sens. Orrin Hatch (R-UT) and Amy Klobuchar (D-MN) today for introducing the Orphan Product Extensions Now Accelerating Cures & Treatments, or OPEN ACT.

Supported by more than 150 rare disease patient advocacy organizations, this bipartisan legislation promises to rapidly bring hundreds of safe, effective, and affordable medicines to rare disease patients by incentivizing drug makers to "repurpose" approved drugs for the treatment of life-threatening rare diseases and pediatric cancers. The introduction of the OPEN ACT in the Senate comes on the same day the House Energy & Commerce Committee overwhelmingly approved the 21^st Century Cures bill (H.R. 6) 51 ayes and 0 noes, which includes the OPEN ACT and a number of other provisions that will help rare disease patients.

The OPEN ACT makes available an additional six months of market exclusivity for the repurposed treatment so long as the sponsor company establishes that the therapy is designated to treat a rare disease and obtains a rare disease indication from the federal Food and Drug Administration (FDA) on the drug label.

"On behalf of the nearly 30 million Americans suffering from rare diseases, we are grateful to Sens. Hatch and Klobuchar for working together to ensure that safe and effective treatments for rare diseases are developed and delivered to those in need," said Julia Jenkins, Executive Director of the EveryLife Foundation. "With 95 percent of rare diseases having no FDA-approved treatment, we are confident the OPEN ACT will lead to many more rare disease therapies at affordable prices, fewer patients using untested and potentially ineffective drugs off–label, and a boost in biotech investment."

The EveryLife Foundation for Rare Diseases is a 501(c)(3) non-profit dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy.

CONTACT: Julia Jenkins, 415-254-5758, [email protected]

SOURCE EveryLife Foundation for Rare Diseases