FDA Rare Pediatric Disease Designation (RPD)
RareMoon has secured over 20% of all FDA Rare Pediatric Designations.
The FDA's Rare Pediatric Disease (RPD) designation is designed to incentivize the development of therapies for serious or life-threatening conditions affecting individuals under the age of 18. Upon product approval, RPD designation qualifies sponsors for a Priority Review Voucher (PRV), a highly valuable, transferable asset that can expedite another pipeline program or be monetized through transfer to another sponsor.
With a nearly 100% success rate, RareMoon excels in securing RPD designations, frequently in tandem with Orphan Drug Designation (ODD) and often during the preclinical stage of a program development. Services include designation eligibility assessment, authoring, review by subject-matter experts, document reconciliation, and submission.
Services Include:
- Feasibility Assessments and regulatory eligibility analysis
- Author, format, and Quality Control (QC)
- Strategic reviews of client-prepared drafts and rationales
- Guidance on FDA engagement strategy and timing
- Submission
- Post-submission support and FDA response preparation
Disclaimer (Updated November 2025)
Under current law, the U.S. FDA's Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program entered its sunset period on December 20, 2024, meaning drugs were required to secure designation (RPD) by that date and approval by September 30, 2026, to remain eligible for the PRV.
By way of update, Congress introduced the Give Kids a Chance Act (H.R. 1262 / S. 932) to reauthorize the RPD PRV program. The bill renews the FDA's authority to award RPD PRVs through 2029 and proposes related updates to pediatric development policy.
Key details include:
- Extension: The bill amends Section 529(b)(5) of the Federal Food, Drug, and Cosmetic Act to extend the FDA's authority to issue RPD PRVs through September 30, 2029.
- Removal of Sunset Language: Section 5(a) of the bill removes the previous statutory sunset date of December 20, 2024, and replaces it with September 30, 2029, effectively continuing the program for five additional years.
- No Two-Step Structure Specified: Unlike current law, the bill does not explicitly restate a two-step deadline (i.e., "designation by X date and approval by Y date"). The amendment eliminates the previous sunset date but does not introduce new separate cutoff points for the two-step structure (designation and approval).
- Current Law Remains in Effect: As of now, the bill has not been enacted. Therefore, the existing deadlines remain active, and RPD designation must be granted by December 20, 2024, and product approval must occur by September 30, 2026, until the new legislation is passed and implemented.
The bill was unanimously advanced by the U.S. House Committee on Energy and Commerce in September 2025 and awaits full Congressional approval.
We will continue to monitor these legislative developments to help clients strategically plan designation timing, pediatric study design, and regulatory submissions under both current and potential reauthorized frameworks.