S. 705 — Innovation in Pediatric Drugs Act of 2025

This section revises enforcement procedures under Section 505B(d) of the Federal Food, Drug, and Cosmetic Act (FDCA) (21 U.S.C. 355c(d)) for sponsor failures to submit required pediatric assessments for drugs or biological products with potential pediatric uses (i.e., Pediatric Research Equity Act). Specifically, it (1) requires FDA to issue a noncompliance letter, provide a 45-day response period, and determine lack of due diligence before concluding noncompliance; (2) permits penalties under FDCA Section 303 (21 U.S.C. 333) only upon a lack of due diligence finding; (3) prohibits such penalties for products no longer marketed; (4) makes a conforming amendment to FDCA Section 303(f)(4)(A) (21 U.S.C. 333(f)(4)(A)) to expressly include Section 505B violations; and (5) delays enforcement to failures occurring 180 days or more after enactment.

This section revises the contents of the annual FDA report to Congress on the Pediatric Research Equity Act (PREA)—which requires drug sponsors to submit pediatric assessments with certain new drug and biologics applications, subject to waivers or deferrals (21 U.S.C. 355c)—as specified in Section 508(b) of the Food and Drug Administration Safety and Innovation Act (21 U.S.C. 355c–1(b)), by (1) expanding paragraph (11) to include an evaluation of compliance with deadlines provided for in deferrals and deferral extensions; (2) making conforming punctuation changes to paragraphs (15) and (16); and (3) adding paragraph (17) to list penalties, settlements, or payments under section 303 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 353) for failure to comply with PREA requirements, including for each the name of the drug, sponsor, and amount imposed.

This section revises the funding provision for the pediatric research program to authorize the Director of the National Institutes of Health (NIH) to make available up to 1% of the amount appropriated for pediatric research to each national research institute and national center under title IV of the Public Health Service Act for fiscal years 2026 through 2030. The program supports clinical trials and other research on priority gaps in pediatric therapeutics (i.e., drugs and biological products), including developmental pharmacology and pediatric diseases.

This section amends sec. 505B(k) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355c(k)), which implements the Pediatric Research Equity Act (PREA) requiring pediatric safety and effectiveness assessments for certain new drugs and biological products, to exempt such products for an orphan-designated indication under sec. 526 (21 U.S.C. 360bb) unless the Secretary determines the assessments could yield a meaningful therapeutic benefit over existing therapies (as defined in sec. 505B(c)). Deferrals and waivers under PREA apply to these assessments to the same extent as others. (Thus, orphan drug developers for rare diseases affecting fewer than 200,000 U.S. patients are generally relieved of pediatric study mandates absent FDA benefit determination, balancing rare disease incentives with pediatric data needs.) The amendment applies only to PREA-covered applications submitted on or after the later of (1) 18 months after FDA final guidance issuance under this section or (2) a later regulatory date set by the Secretary. This section further directs FDA to (1) issue draft guidance within one year of enactment on PREA application to orphan drugs (addressing waivers, pediatric plans, rare disease lists under sec. 505B(a)(5)(E) and (b)(2)(E), real-world evidence, and scientific challenges); (2) hold a public meeting within six months of the draft to inform finalization; and (3) finalize guidance within 18 months of the meeting. This section requires the Comptroller General to report to specified congressional committees within four years of the amendment's applicability date on this section's impacts on rare disease drug development and pediatric labeling for orphan indications, including a survey of drug developers, patient and provider input, and study requirement effects. This section includes a rule of construction preserving molecularly targeted pediatric cancer study requirements under sec. 505B(a)(3).