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  1. Medical products for rare diseases and conditions

Rare Pediatric Disease (RPD) Designation and Voucher Programs

Under Section 529 to the Federal Food, Drug, and Cosmetic Act (FD&C Act), FDA will award priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. Under this program, a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

RPD Designation Program Sunset Provisions

On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended. Under the current statutory sunset provisions, after September 30, 2024, FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug, and that designation was granted by September 30, 2024. After September 30, 2026, FDA may not award any rare pediatric disease priority review vouchers.

For any questions, please contact the Office of Orphan Products Development at orphan@fda.hhs.gov.

On July 29, 2019, FDA issued a revised draft guidance, Rare Pediatric Disease Priority Review Vouchers - Guidance for Industry

The revised draft guidance incorporates public comments received on the initial draft and provides FDA’s thinking regarding the provisions of the Advancing Hope Act of 2016, which updated the definition of a rare pediatric disease as one that is a rare disease and one where the disease is serious or life-threatening with the serious or life-threatening manifestations primarily affecting individuals from age zero to 18. Among the key changes from the initial draft are an explanation of the rare pediatric disease priority review voucher eligibility requirements, the rare pediatric disease designation process and examples to illustrate the agency’s current thinking on these review determinations.

Contact Information

Questions about the RPD designation program, contact the Office of Orphan Products and Development (OOPD):

orphan@fda.hhs.gov or 301-796-8660

Questions about drug products, RPD PRV eligibility, contact the Center for Drug Evaluation and Research (CDER):

PriorityReviewVouchers@fda.hhs.gov or 301-796-1394 

Questions about biologic products, RPD PRV eligibility, contact the Center for Biologics Evaluation and Research (CBER):

industry.biologics@fda.hhs.gov or 240-402-8020

When sponsors use a priority review voucher to obtain a priority review of a drug application, they are required to pay a fee in order to do so.  The Federal Register notice concerning the fee for redeeming a Rare Disease Priority Review Voucher in fiscal year 2017 program is available here: https://www.gpo.gov/fdsys/pkg/FR-2016-09-30/pdf/2016-23624.pdf

Section 529 of the FD&C Act is codified at 21 U.S.C. 360ff.  the full text is available at: https://www.gpo.gov/fdsys/pkg/USCODE-2012-title21/pdf/USCODE-2012-title21-chap9-subchapV-partB-sec360ff.pdf (please note that this version does not contain the advancing Hope Act of 2016 amendments).

Request for Rare Pediatric Disease designation should be sent to orphan@fda.hhs.gov or by mail to:

Office of Orphan Products Development 
Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD  20993-0002

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