Leading the Charge in Securing Rare Pediatric Disease Designations
Our team has secured over 20% of all FDA Rare Pediatric Designations.
The FDA’s Rare Pediatric Disease (RPD) designation is a strategic regulatory tool 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, we excel in securing RPD designations, frequently in tandem with Orphan Drug Designation (ODD) and often during the preclinical stage of a program.
We support Clients at all stages of development — from early strategic assessment through authorship, rigorous review, and regulatory submission — ensuring the pediatric justification and application criteria are precisely aligned with the FDA’s expectations.
Will the FDA’s Rare Pediatric Disease Designation Priority Review Voucher Program Be Renewed?
Disclaimer (Updated April 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 April 2025, bipartisan legislation has been introduced in both the House (H.R. 1262) and Senate (S. 932) to reauthorize the FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program, aiming to extend the program for another five years.
Both bills are currently under congressional review, with strong support from rare disease and pediatric cancer stakeholders urging swift passage.
