This bill authorizes certain targeted clinical trials involving combinations of drugs to treat pediatric cancer, and renews the Food and Drug Administration’s (FDA’s) authority to award priority review vouchers (PRVs) to sponsors of new products for rare pediatric diseases.

Specifically, the bill modifies requirements relating to molecularly targeted pediatric cancer investigations to permit research on new drugs used in combination with active ingredients that have already been approved and that (1) have been determined to be part of the standard of care for treating a pediatric cancer, or (2) have been approved to treat an adult cancer and are directed at molecular targets for pediatric cancer.

The FDA must issue guidance on the implementation of these provisions and report to Congress on its efforts to ensure implementation. The Government Accountability Office (GAO) must report on the effectiveness of the bill's changes with respect to the development of pediatric cancer drugs.

The bill also renews the FDA’s authority to issue PRVs to sponsors of new products intended to treat rare pediatric diseases through September 30, 2029. This is known as the Rare Pediatric Disease PRV program. The program expired in December 2024.

GAO must report on the effectiveness of the Rare Pediatric Disease PRV program, including to what extent PRVs were successful in promoting drug development and expediting patient access to drugs for the treatment or prevention of rare pediatric diseases.