RARE Daily

Bipartisan Legislation Sees to Ensure Rare Disease Therapies Benefit Kids

December 12, 2023

California Democrat Anna Eshoo and Texas Republican Michael McCaul have introduced legislation to ensure rare disease drugs are studied in children and to hold drug companies accountable for completing pediatric study requirements.

The Innovation in Pediatric Drugs Act of 2023 has been introduced and is referred to the House Committee on Energy and Commerce.

Congress first recognized the need to ensure drugs were being studied in children in 1997 when it passed the Best Pharmaceuticals for Children Act, a bill to incentivize the study of off-label uses in pediatric populations by offering companies an additional six months of patent life on their product. Eshoo was the author of that legislation.

Congress later passed Eshoo’s Pediatric Research Equality Act in 2003 which requires drug companies to study on-label indications in children when safe and appropriate.

The proposed legislation will strengthen both laws by requiring drugs for rare diseases to be studied in children. And it will grant the U.S. Food and Drug Administration additional authority to penalize companies that don’t complete required pediatric studies on time.

The legislation also calls for increasing funding for the National Institutes of Health’s Best Pharmaceuticals for Children Act Program to $50 million from $25 million, the first funding increase since 2002, to fund additional clinical trials and train additional personnel to help run trials.

“We don’t know whether a drug will be effective in children if we don’t test for it,” said Eshoo. “We’re entering a golden age of medicine thanks to innovative therapies to treat pediatric cancer and other rare diseases. Children must be included in the drug development process, which is why I’m introducing the Innovation in Pediatric Drugs Act to ensure children can benefit from advancements in new treatments and therapies.”

Stay Connected

Sign up for updates straight to your inbox.