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 July 2025)
The FDA's Rare Pediatric Disease (RPD) Priority Review Voucher program officially entered its sunset phase on December 20, 2024. Under the current provision, the FDA can no longer award PRVs unless a therapy received RPD designation by that date. Additionally, those designated therapies must be approved by September 30, 2026, to qualify for a voucher. While the FDA still accepts RPD designation requests, only previously designated products remain eligible for PRVs. Legislative efforts to reauthorize the program are ongoing.
As of July 2025, reauthorization efforts are underway for the FDA's Rare Pediatric Disease Priority Review Voucher (PRV) program. Two bipartisan bills (H.R. 1262 – Give Kids a Chance Act of 2025 and S. 932) have been introduced in the House and Senate, respectively, combining provisions from the original Give Kids a Chance Act and the Creating Hope Reauthorization Act, aiming to extend the PRV program by an additional five years.
We will update this page regularly with FDA and Congressional updates regarding the RPD program's status to ensure biotech sponsors and rare disease developers remain informed and prepared to act within appropriate timelines.