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Congressman Gus Bilirakis

Representing the 12th District of Florida

Bilirakis’ Legislation Opens the Door to Treatments for Rare Diseases

February 28, 2017
Press Release

Washington, D.C. – In honor of Rare Disease Day today, U.S. Representative Gus Bilirakis (FL-12) introduced the Orphan Product Extensions Now Accelerating Cures and Treatments Act, or the OPEN Act (H.R. 1223), to help open the door to potentially hundreds of new treatments for rare diseases. Bilirakis recently spoke on the U.S. House floor in support of the legislation. Please see below for an excerpt of his remarks.

“Most importantly, the OPEN Act would help make sure those suffering from a rare condition can finally find safe, effective, and affordable medication. I was inspired to write the OPEN Act after meeting with folks who live with rare diseases, like Ashleigh Pike, Candace Lerman, and Kelly Freeman from Florida. The ideas that shaped this legislation came from those who it will help most—rare disease patients. After all, rare diseases are not a rare problem,” said Bilirakis.

There are 7,000 rare diseases affecting 30 million Americans, and yet 95 percent of those conditions have no approved treatment or cure. The problem is that each rare disease impacts a small patient population, leaving little incentive for drug makers to run additional trials and bring major market drugs into rare disease markets. The OPEN Act would provide incentives for drug makers to repurpose mainstream treatments, at mainstream prices, for rare diseases.

Over 150 rare disease patient advocacy groups support the OPEN Act, including the EveryLife Foundation, the RASopathies Network, the Pediatric Cancer Foundation, and more.