Hatch, Klobuchar propose law to foster development of rare disease treatments

Stock image composite, St. George News

WASHINGTON D.C. — Sens. Orrin Hatch and Amy Klobuchar introduced the Orphan Product Extensions Now Accelerating Cures and Treatments Act of 2015 Thursday. The “OPEN ACT,” as it is being called, would build on the success of the Orphan Drug Act to provide incentives to increase market potential for companies that “repurpose” previously-approved drugs for the treatment of rare diseases. Repurposing drugs is faster, less expensive and presents fewer risks than traditional drug development.

Hatch had this to say about the bill:

Finding legislative ways to help medical innovators treat rare diseases has been a top priority of mine for over thirty years, since Congressman Henry Waxman and I first championed the Orphan Drug Act in 1983.

This bill is a natural next step in expanding that effort to close the gap for rare diseases for which we do not yet have treatments. In addition to increasing the number of rare disease therapies, this legislation will help boost innovation and provide safer options for rare disease patients using drugs off-label.

Complex, rare diseases with small patient populations have limited market potential, so there are few economic incentives to develop new drugs targeting those diseases. The Orphan Drug Act was a successful first step toward addressing this issue and led to numerous new lifesaving technologies. However, while there are 7,000 rare diseases that impact 30 million Americans, 95 percent of these diseases still have no treatments.

“While there are about 7,000 known rare diseases, there are fewer than 500 drugs approved to treat any of those conditions,” Klobuchar said. “This means the only option for many of the 30 million Americans with rare diseases is to use drugs off-label in search of relief. The bipartisan open act will help to increase the number of safe, effective and affordable treatments that are available for people with rare diseases.”

The open act would provide an additional six months of market exclusivity to drugs that are repurposed and approved by the Food and Drug Administration for a new indication for a rare disease or condition. This would apply to currently-approved drugs that are still under patent. The six-month extension would be in addition to other types of exclusivity, such as pediatric or qualified infectious disease product exclusivity.


Read the May 5 letter of support for the open act legislation in the House of Representatives from 155 patient organizations:  20150505 Letter in support of OPEN ACT legislation from 155 patient organizations


Submitted by the offices of Hatch and Klobuchar

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