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Sep. 23, 2016

TOPIC: Advocacy, Press Releases

Senate Passes Short-Term Agreement to Accelerate Treatments for 15 Million Sick Children with Rare Diseases

Posted by Jennifer Huron

Unanimous vote extends the Rare Pediatric Disease Priority Review Voucher Program with important policy changes through December

Washington, D.C., September 23, 2016 – On Thursday, the Senate reached an important compromise to accelerate new treatments to help 15 million sick children with rare diseases.

Led by Senators Robert Casey (D-PA), Johnny Isakson (R-GA), and Lamar Alexander (R-TN), the Senate voted unanimously to extend the Rare Pediatric Disease Priority Review Voucher (PRV) Program until the end of 2016.  The Rare Pediatric Disease PRV, supported by the National Organization for Rare Disorders (NORD), is the Food and Drug Administration’s program that incentivizes the research and development of life-saving treatments for rare pediatric diseases. The program was set to expire at the end of this month.

Before voting to extend the program, Sens. Casey, Isakson, and Alexander had attempted to pass the Advancing Hope Act (S.1878), which would have reauthorized the PRV for six years. While this vote failed, they achieved a critical compromise to extend the program and include all of the policy changes included in S.1878, including an important definition change for “rare pediatric disease.”

“The Advancing Hope Act changes the statutory definition for rare pediatric diseases because it takes into account morbidity and incidence rather than overly relying on prevalence data,” said Peter L. Saltonstall, president and CEO of NORD.  “NORD has pushed for this important change for nearly two years so that all deserving rare pediatric diseases are included.”

The compromise bill has moved over to the House with a vote likely next week.  The Senate Republicans have also included the extension of the PRV program in their Continuing Resolution proposal released yesterday.

“We urge members of the House to join their Senate colleagues and unanimously support this legislation that puts children first and protects our most vulnerable citizens,” added Saltonstall.

NORD applauds Sens. Casey, Isakson, and Alexander, and the hundreds of advocates who joined in advocating for the reauthorization of the Rare Pediatric Disease PRV program.

“With the extension, we will need advocates to join us and fight this battle again in November before the extension expires,” said Saltonstall.

The National Organization for Rare Disorders (NORD) is a 501(c)(3) independent charity and the leading independent advocacy organization representing all patients and families affected by rare diseases.

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