Key Takeaways

• The U.S. Food and Drug Administration (FDA) has requested public comment concerning incentives and obstacles to pediatric drug development. It will hold a public meeting for interested parties on September 15, 2025, and is accepting written comments until September 30, 2025.
• Pharmaceutical sponsors may be particularly interested in comment concerning pediatric exclusivity and the Rare Pediatric Disease Priority Review Voucher program, which began sunsetting on December 20, 2024, and will expire unless Congress extends the program.

The FDA will hold a public interested parties meeting on September 15, 2025, to receive public comment concerning pediatric drug development under the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA).¹ The agency is also accepting written comments until September 30, 2025.²

The BPCA and PREA are complementary statutes that encourage the development of new pediatric drugs. The BPCA was first enacted in 2002 and provides incentives, such as additional market exclusivity, for sponsors who conduct voluntary pediatric studies.³The PREA was enacted the following year and requires pediatric studies for certain new drugs such as new active ingredients and new dosage forms.⁴

The upcoming meeting and public comment are intended to help the FDA better understand the effects of PREA’s requirements and the BPCA’s incentives on pediatric drug development and the practical challenges of conducting pediatric studies.

Sponsors may be particularly interested in comment concerning the BPCA’s incentives. Pediatric exclusivity provides a six-month extension of all unexpired Orange Book-listed patents and FDA regulatory exclusivities in exchange for conducting a voluntary pediatric study.⁵To qualify, the sponsor proposes the pediatric study and, if the FDA agrees, it will issue a written request that authorizes it. After the FDA accepts the study data, the sponsor will receive the six-month extension for all listed drugs products that it owns containing the studied drug.⁶

Sponsors may also be interested in discussion of the Rare Pediatric Disease Priority Review Voucher (PRV) program, which begins to sunset after December 20, 2024, unless Congress extends this program.⁷This program awards a transferable voucher to a sponsor who obtains approval of a new drug that treats a rare pediatric disease. The voucher can be redeemed for priority review of a subsequent application, entitling its holder to FDA review in six months instead of the usual 10 months.⁸

PRV vouchers can be sold or transferred to third parties, who can redeem them to receive priority review for a different drug product, making the vouchers a valuable asset. Unfortunately, Congress has not yet reauthorized the pediatric PRV program past 2024 and the FDA cannot issue new vouchers until it does.⁹

Sponsors developing drugs or biologics for rare pediatric indications or who seek to benefit from these incentives should consider participating in the meeting or submitting a written comment. Stakeholder feedback may influence the FDA’s future policy and new legislation that the agency requests from Congress.

The FDA encourages interested parties to register and submit written comments in advance of the meeting on September 15. Additional information, including the agenda and instructions is available on the FDA’s meeting page at https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/interested-parties-meeting-implementation-best-pharmaceuticals-children-act-and-pediatric-research.

For additional information regarding new developments at the FDA, submitting a written comment to the FDA, and how to qualify for pediatric exclusivity or other regulatory incentives, please contact Wilson Sonsini attorneys Eva F. Yin or Daniel E. Orr.

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^[1]U.S. Food and Drug Administration, Interested Parties Meeting: Implementation of the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act, https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/interested-parties-meeting-implementation-best-pharmaceuticals-children-act-and-pediatric-research-equity-act (last visited: Jul. 17, 2025).

^[2]Id.

^[3]21 U.S.C. § 355a(b).

^[4]21 U.S.C. §§ 360c(a)(1)-(2).

^[5]21 U.S.C. § 355a(b).

^[6]U.S. Food and Drug Administration, Pediatric Drug Development: Regulatory Considerations—Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act Guidance for Industry (May 2023), at pp. 29-30.

^[7]21 U.S.C. § 360ff.

^[8]Id.

^[9]U.S. Food and Drug Administration, Rare Pediatric Disease Designation and Priority Review Voucher Programs (Sept. 27, 2024), https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs.