This bill significantly enhances the framework for developing treatments for pediatric cancer by amending the Federal Food, Drug, and Cosmetic Act. It expands requirements for molecularly targeted pediatric cancer investigations, allowing for studies of new drugs or biological products either alone or in combination with existing therapies. These combinations can include active ingredients that are part of the standard of care for pediatric cancer or adult cancer drugs targeting molecular pathways relevant to pediatric cancer. The investigations must be designed to yield clinically meaningful pediatric data on dosing, safety, and preliminary efficacy, utilizing appropriate formulations for different age groups.

The legislation clarifies that combination investigations are limited to those involving a single new active ingredient or a combination where each ingredient is already approved for adult cancer. The Food and Drug Administration (FDA) is tasked with issuing draft guidance within 12 months of enactment and finalizing it within another 12 months to guide the implementation of these changes. These new requirements will apply to relevant drug applications submitted three years after the bill's enactment, with the Secretary of Health and Human Services reporting on implementation efforts within six years.

Additionally, the bill extends the rare pediatric disease priority review voucher program, pushing its expiration date from December 20, 2024, to September 30, 2030. This extension aims to continue incentivizing the development of drugs for rare pediatric diseases. The Government Accountability Office (GAO) is mandated to conduct studies on the effectiveness of both the new pediatric cancer investigation requirements and the priority review voucher program, submitting reports to Congress within ten and five years, respectively, to assess their impact on drug development and unmet medical needs.