Sep. 30, 2016
Posted by Jennifer Huron
“On behalf of the 15 million children with rare diseases, we thank the President for signing the Advancing Hope Act so that some of our most vulnerable citizens are not overlooked,” said Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), which has supported the bill. “We also thank bipartisan leaders in the House and Senate who came together in the past week to show their commitment to help children with rare diseases.”
The Rare Pediatric Disease PRV program encourages the development of therapies for children with rare diseases. There are an estimated 7,000 rare diseases, which are defined as a disease affecting 200,000 or fewer people. Of the nearly one in ten Americans with a rare disease, more than half are children. Of the 350 most “common” rare diseases, 27 percent result in death before the child’s first birthday.
Despite significant unmet medical need—95 percent of rare diseases still have no treatment (the approximately 500 approved orphan products treat only about 375 rare diseases)—manufacturers face significant obstacles that can hinder the pursuit of rare disease therapies for children, including difficulties associated with conducting clinical trials. To tackle these hurdles, Congress established the Rare Pediatric Disease PRV Program.
For nearly two years, NORD has helped to lead advocacy efforts for the permanent authorization of the program with an update that would include all deserving rare pediatric diseases. Efforts included frequent meetings with Congressional offices, sign-on letters, op-eds, and email campaigns.
“We are grateful to our supporters and NORD’s member organizations who joined us in advocacy efforts and made their voices heard,” said Saltonstall. “With the extension, we will need advocates to join us and fight this battle again in November so that this important program does not go away.”
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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