A bipartisan bill proposed by U.S. Sen. Shelley Moore Capito (R-WV) would encourage innovation in the development of pediatric drugs for rare diseases.
“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,” Sen. Capito said on Aug. 7. “That’s why it is essential medicines be studied specifically for children’s use, especially for rare diseases.
“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,” she said.
The Innovation in Pediatric Drugs Act, S. 4905, which Sen. Capito cosponsored on July 31 alongside bill sponsor U.S. Sen. Jack Reed (D-RI), would ensure drugs for rare diseases are studied in children and drug companies are accountable for completing pediatric study requirements, according to a bill summary provided by the lawmakers.
“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,” said Sen. Reed. “Our bipartisan bill would provide new paths for pediatric rare disease research and development and ensure patients with rare diseases aren’t left behind.”
The measure would make changes to the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), two laws that encourage and require the study of drugs in children.
For instance, while drugs for rare diseases, also known as orphan drugs, are not required to be studied in children, the majority of new drugs approved are orphan drugs, which means they are exempt from pediatric study requirements, the summary says.
S. 4905 would ensure that children with rare diseases can benefit from new and innovative treatments, lifting the orphan drug exemption in PREA.
The bill also would increase funding for a BPCA National Institutes of Health program to conduct studies of off-patent drugs used in children to ensure there is better data about older drugs to treat diseases in children, states the summary.
S. 4905 is the companion bill to the same-named H.R. 6664, introduced in December 2023 by U.S. Reps. Anna G. Eshoo (D-CA) and Michael McCaul (R-TX).