The "Innovation in Pediatric Drugs Act of 2025" aims to enhance pediatric drug development by amending the Federal Food, Drug, and Cosmetic Act. A significant change is the application of pediatric study requirements to orphan drugs for indications where the Secretary determines a meaningful therapeutic benefit for children could be represented. This expands the scope of required pediatric assessments, which previously largely exempted orphan drugs. The bill also mandates the issuance of guidance and a public meeting to clarify how these new requirements, including deferrals and waivers, will be implemented for orphan drugs.

To ensure compliance, the bill modifies enforcement actions for failures to meet pediatric study requirements. It introduces a due diligence process, requiring the Secretary to issue a noncompliance letter and allow a 45-day response period before determining a lack of due diligence. Enforcement actions under Section 303 will only apply if a lack of due diligence is demonstrated, and unmarketed drugs are exempt from these specific penalties. The FDA will also be required to report annually on compliance with deferral deadlines and list all penalties or settlements related to pediatric study non-compliance.

Furthermore, the legislation provides greater certainty regarding waivers by requiring the Secretary to maintain automatic full waiver lists for adult-related diseases where pediatric studies are impossible, highly impracticable, or meet other waiver criteria. If a disease is on this list, assessments for that condition will be automatically waived. The bill also authorizes the NIH Director to allocate up to one percent of pediatric research funding for specific pediatric studies and mandates a GAO study on the impact of these changes on rare disease drug development and pediatric information availability.