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Congressman Gus Bilirakis

Representing the 12th District of Florida

Bilirakis: “OPEN Act has the potential to result in hundreds of new treatments for rare diseases”

March 22, 2017
Press Release

Washington, D.C. – During a congressional committee hearing on streamlining regulations at the Food and Drug Administration (FDA), U.S. Rep. Gus Bilirakis (FL-12) highlighted his bill, the OPEN ACT, to open the door for hundreds of new treatments for rare diseases. Click here for more information on the hearing.  See below for an excerpt of Bilirakis’ questioning.

Bilirakis: “There are about 500 approved rare disease drugs, but 7,000 rare diseases affecting some 30 million Americans.  They’re taking medication off-label, not knowing if their drugs are safe and effective for their conditions, or if it’s the proper dosage, and fighting with their insurance companies on coverage of their medications. Does it make sense to incentivize development for a targeted population when there are clearly defined needs?”

Kay Holcombe, Senior VP of Science Policy, BIO: “Yes.”

Bilirakis: “Much like the BPCA (Best Pharmaceuticals for Children Act), it creates an incentive to run more clinical trials in the rare disease space where 95% of the disease have no FDA approved treatments.  This would bring more approved drugs to these patients.  OPEN ACT has the potential to result in hundreds of new drugs and treatments for individuals with rare diseases.  Over 150 rare disease patient groups support this bill.  OPEN ACT was part of the House passed 21st Century Cures Act, and while it fell out at the 11th hour, unfortunately, I’m going to continue to push for this legislation. It’s a priority for me. Do you have any comments?”

Holcombe: “It’s clear that without the Orphan Drug Act we would not have 500 treatments for rare diseases. It’s also a tragedy that we don’t have 7,500. We believe incentives can work.”  

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 the rare disease market. The OPEN Act would provide incentive 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.