Charleston Leader

U.S. Senators Shelley Moore Capito (R-W.Va.) and Jack Reed (D-R.I.) have introduced the Innovation in Pediatric Drugs Act, aimed at accelerating research and advancing treatments for rare diseases affecting children.

“As I meet with doctors, researchers, and parents, I am often reminded that while children sadly can get adult diseases, they are not in fact small adults. That’s why it is essential medicines be studied specifically for children’s use, especially for rare diseases,” Ranking Member Capito said. “This legislation will help ensure pediatric studies are actually being done on both new and innovative drugs, as well as those older drugs now off-patent.”

“Congress must work together to help address the unmet needs of those affected by rare diseases, particularly children. The Innovation in Pediatric Drugs Act would include children in the drug development process to expand access to safe and effective treatments and therapies for children with rare ailments and appropriately meet their needs. Our bipartisan bill would provide new paths for pediatric rare disease research and development and ensure patients with rare diseases aren’t left behind,” Senator Reed said.

The bipartisan Innovation in Pediatric Drugs Act aims to ensure drugs for rare diseases are studied in children and holds drug companies accountable for completing pediatric study requirements. It seeks to close research gaps created by the increase in “orphan drug” approvals by the U.S. Food and Drug Administration (FDA).

Children metabolize drugs differently than adults, necessitating specific studies for children's use. The act intends to expedite therapies needed by children through changes to the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), which encourage and require the study of drugs in children.

Key provisions of the Innovation in Pediatric Drugs Act include:

- Ensuring that drugs for rare diseases are studied in children: With nearly 7,000 rare diseases lacking appropriate treatments—most affecting children—the act lifts the orphan drug exemption in PREA.

- Providing equal accountability for pediatric study requirements: It grants FDA authority to enforce timely completion of legally required pediatric studies.

- Investing in pediatric studies of older off-patent drugs: It increases funding for BPCA NIH programs to gather better data on older drugs used to treat childhood diseases.

Senators Capito and Reed previously collaborated on passing the Childhood Cancer STAR Act. Companion legislation has been introduced in the U.S. House of Representatives by U.S. Reps. Anna G. Eshoo (D-Calif.) and Michael McCaul (R-Texas).