Innovation in Pediatric Drugs Act of 2025
Innovation in Pediatric Drugs Act of 2025
Plain Language Summary
# Summary: Innovation in Pediatric Drugs Act of 2025 **What the Bill Does** This bill would expand the FDA's power to require drug companies to conduct studies on children for rare diseases. Currently, drugs designed for rare conditions (called "orphan drugs") are generally exempt from pediatric study requirements. This bill would remove that exemption, allowing the FDA to mandate that companies test certain rare disease drugs on children if doing so could improve treatment options or provide additional choices within a drug category. **Who It Affects** The bill primarily impacts pharmaceutical companies that develop drugs for rare pediatric diseases and children with rare conditions.
It also affects the FDA, which would gain new regulatory authority and must create guidelines on how the new requirements will work, including when companies can request exemptions or waivers. **Key Provisions** The bill requires the FDA to issue guidance explaining implementation details, including how companies can obtain waivers from pediatric study requirements. It also gives the FDA enforcement power to take action against companies that fail to meet these new study requirements. The change aims to ensure that children with rare diseases have access to better-tested treatment options. **Current Status** The bill is currently in committee (S 705, 119th Congress) and has not yet been voted on by the full Senate.
CRS Official Summary
Innovation in Pediatric Drugs Act of 2025This bill expands the Food and Drug Administration’s (FDA’s) authority with respect to research on rare pediatric diseases, including by permitting the FDA to require pediatric studies on certain orphan drugs and to take enforcement action against drug sponsors that fail to satisfy pediatric study requirements.Specifically, the bill would impose pediatric study requirements on drugs for rare diseases or conditions (i.e., orphan drugs) if the FDA determines that (1) the drug could improve the treatment, diagnosis, or prevention of a disease compared with currently available products for the relevant pediatric population; or (2) there is a need for additional options within the drug’s class or indication. (Under current law, pediatric study requirements generally do not apply to orphan drugs.) The FDA must issue guidance describing how these changes will be implemented, including information on how waivers will be granted. The bill also permits the FDA to take enforcement action against drug sponsors that fail to comply with pediatric study requirements, if such sponsors demonstrated a lack of due diligence in satisfying the requirements.Additionally, the bill authorizes the National Institutes of Health to allot a certain amount of funds for priority pediatric research, including research on drugs with no remaining patents on which pediatric studies are needed.Finally, the Government Accountability Office must report on the bill’s impact on rare disease drug development and on the availability of pediatric information on orphan drugs.
Latest Action
Read twice and referred to the Committee on Health, Education, Labor, and Pensions. (Sponsor introductory remarks on measure: CR S1347)