The FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program is approaching a significant sunset phase. On September 27, 2024, the FDA announced that, under the Continuing Appropriations and Extensions Act of 2025, the PRV program will officially begin to phase out after December 20, 2024. This program has been vital for encouraging the development of treatments for rare pediatric diseases by offering priority review vouchers to qualifying sponsors, which can be redeemed or sold.
Key Points from the 2024 Update:
- Program Sunset: As of December 20, 2024, the FDA will no longer award PRVs unless the product has been designated as a rare pediatric disease drug by that date. The drug also must receive FDA approval no later than September 30, 2026, for the sponsor to qualify for a voucher.
- Urgency for Submission: Under this newly condensed timeline, sponsors must act quickly to submit rare pediatric disease designation requests before December 20, 2024. Any delays or a surge in requests close to the deadline could make it difficult for the FDA to meet review timelines, potentially disqualifying some applications from their voucher eligibility.
Important Reminders from the 2019 FDA Draft Guidance:
- Early Submission is Critical: The 2019 Draft Guidance strongly encourages early submission of rare pediatric disease designation requests. To avoid delays, sponsors should submit these requests alongside orphan drug designation or fast-track designation. With the sunset approaching, early action is even more critical to ensure timely review.
- Eligibility Requirements: To qualify for a PRV, a drug must target a serious or life-threatening condition that primarily affects pediatric populations. It must also meet the FDA’s criteria for rare disease designation under the Federal Food, Drug, and Cosmetic (FD&C) Act. Submissions are required to include robust, tangible clinical data to demonstrate these conditions.
- Conditional Designations: The FDA may issue conditional designations for rare pediatric disease products—this means that the final voucher eligibility depends on approval, emphasizing urgency for sponsors to submit their applications before the December 20, 2024 deadline for designation securement.
For sponsors interested in submitting designation requests, detailed instructions can be found in the 2019 Draft Guidance on Rare Pediatric Disease Priority Review Vouchers.
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