House Passes Bill to Restore Rare Pediatric PRVs

Rare Daily Staff

The U.S. House of Representatives unanimously passed the Mikaela Naylon Give Kids a Chance Act, a bill that will restore the FDA’s Rare Pediatric Disease Priority Review Voucher program.​

The legislation, designed to accelerate pediatric cancer treatments and expand access to life-saving therapies for children with rare diseases, combines the original Give Kids a Chance Act and the Creating Hope Reauthorization Act.​

Rep. Michael McCaul, R-Texas, introduced the legislation, which is sponsored by Reps. Gus Bilirakis, R-Florida; Debbie Dingell, D-Michigan; Kim Schrier, D-Washington; Diana Harshbarger, R-Tennessee; Kathy Castor, D-Florida; Mike Kelly, R-Pennsylvania; Doris Matsui, D-California; Dan Crenshaw, R-Texas; Lori Trahan, D-Massachusetts; and Randy Weber, R-Texas.​

The bill combines two critical initiatives — the original Give Kids a Chance Act and the Creating Hope Reauthorization Act — to spur drug development for pediatric rare diseases, improve patient outcomes, and close gaps in pediatric drug research.​

Each year, nearly 16,000 children in the United States are diagnosed with cancer. Children comprise as many as half of those living with rare diseases, yet treatment options for children remain extremely limited compared with those for adults.​

The Mikaela Naylon Give Kids a Chance Act would reauthorize the FDA’s Rare Pediatric Disease Priority Review Voucher program — initially created under McCaul’s Creating Hope Act — which allows pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases. Since the program’s creation in 2012, the FDA has awarded more than 60 pediatric priority review vouchers for treatments addressing nearly 40 rare pediatric diseases.​

Additionally, thousands of successful drug combination therapies are now being studied and developed for adults, but not for children. The Mikaela Naylon Give Kids a Chance Act would authorize the FDA to direct companies to conduct pediatric trials of combinations of cancer drugs and other therapies.​

“As a founder of the Childhood Cancer Caucus, I’ve met with countless cancer patients and advocates who have asked me for one thing: to give kids their best chance of beating cancer,” McCaul said. “Today, the House of Representatives shined a beacon of hope for those children and their families by unanimously passing the Mikaela Naylon Give Kids a Chance Act.”