Innovation in Pediatric Drugs Act of 2025
The Innovation in Pediatric Drugs Act of 2025 would update how the federal government oversees pediatric drug studies, with a focus on molecularly targeted pediatric cancer drugs and orphan-drug indications. It tightens enforcement procedures for pediatric study requirements, adds a due-diligence process and timelines, increases transparency about penalties and settlements, and funds pediatric research at NIH. It also creates a pathway to adjust, clarify, and accelerate pediatric studies for orphan drugs, including guidance and public engagement, while preserving a separate, explicit lane of requirements for targeted pediatric cancer products. The overall aim is to better ensure that children’s needs are studied and labeled appropriately, while reducing uncertainty for sponsors and improving accountability.
Key Points
- 1Strengthened, more process-driven enforcement for pediatric study requirements
- 2- Introduces a formal “noncompliance letter” at 270 days and a 45-day response window, with a determination of due diligence before any enforcement action under section 303.
- 3- Adds a limit that enforcement actions cannot be taken for drugs no longer marketed, and creates a transition rule allowing actions only for failures after 180 days from enactment.
- 4Greater FDA transparency and accountability on penalties
- 5- FDA must include penalties, settlements, or payments under section 303 in a public report, listing drug name, sponsor, and amount, and evaluate deadlines tied to deferrals/extensions under PREA-related processes.
- 6Increased funding for pediatric research
- 7- Directs NIH to allocate up to 1% of pediatric-research funding for dedicated pediatric research under this Act from 2026 through 2030.
- 8Expanded and clarified pediatric studies for orphan drugs
- 9- Applies pediatric assessment requirements to orphan-designated drugs only if pediatric assessments would meaningfully benefit patients, with deferrals/waivers applying similarly to other pediatric requirements.
- 10- Requires issuance of draft guidance within 1 year and final guidance within 18 months after a required public meeting, covering how waivers, timing, and rare-disease considerations will work.
- 11- Requires a GAO study within four years to assess the impact on rare-disease drug development and pediatric labeling, including input from industry, patients, and providers.
- 12Certainty and process around waivers
- 13- Establishes an automatic full-waiver list for certain adult-related diseases where pediatric studies are impracticable or would meet waiver criteria, and ties this to the initial pediatric study plan so that listed diseases can be waived automatically if applicable.
- 14Specific note on targeted pediatric cancer drugs
- 15- The bill includes a rule of construction ensuring that its changes do not limit investigations of molecularly targeted pediatric cancer drugs that have orphan designation, preserving any existing or future targeted-cancer study obligations within that framework.