September 24, 2024
The National Organization for Rare Disorders (NORD) applauds the House of Representatives for passing the Give Kids a Chance Act, a major step toward providing more opportunities for children with rare diseases to access life-changing treatments for their conditions that are safe and effective. The bipartisan, unanimous support in the House shown for this bill is a testament to the importance of advancing rare disease pediatric therapeutic development.
NORD is particularly grateful to Representatives Michael McCaul, Anna Eshoo, Gus Bilirakis, and Doris Matsui for championing critical provisions of the Give Kids a Chance Act, including language to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) program and cement how FDA awards orphan drug exclusivity. NORD, along with our rare disease patient organizations and grassroots advocates, have fought tirelessly to move these critical initiatives forward and while we are pleased with the progress made by the House vote, there is no time to lose.
The Pediatric Rare Disease PRV program is set to expire on September 30th. NORD’s analysis shows that 36 out of the 39 rare disease communities who have benefited from the PRV program had no FDA-approved treatment before its creation in 2012. This program has been a game-changer, and we cannot afford to let it lapse. Therefore, Congress must act swiftly to pass a short-term extension of the PRV program as part of the upcoming continuing resolution and continue to make progress towards a full five-year reauthorization as included in Give Kids a Chance Act.
Every day matters for the more than 15 million children in the U.S. with rare diseases. We need Congress to act quickly so that every child, no matter how rare their condition, has hope for access to a life-changing treatment.
Pam Gavin, CEO, National Organization for Rare Disorders (NORD)
