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  1. Developing Products for Rare Diseases & Conditions

Rare Pediatric Disease (RPD) Designation Program

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.

On July 29, 2019, FDA issued a revised draft guidance, Rare Pediatric Disease Priority Review Vouchers - Guidance for Industry.  The revised draft guidance incorporates public comments received on the initial draft and provides FDA’s thinking regarding the provisions of the Advancing Hope Act of 2016, which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.

Questions regarding designations under this program should be directed to OOPD at orphan@fda.hhs.gov or 301-796-8660

Product-specific questions about rare pediatric disease priority review voucher eligibility should be directed to the appropriate review division within the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research (CBER). 

General questions related to the rare pediatric disease priority review voucher program can be directed to OOPD at orphan@fda.hhs.gov or 301-796-8660 or to CDER's Rare Diseases Program at CDERONDRareDiseaseProgram@fda.hhs.gov or either 301-796-4842 or 301-796-4061.

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:

The Office of Orphan Products Development (OOPD)
Food and Drug Administration
WO32-5295
10903 New Hampshire Avenue
Silver Spring, MD  20993-0002

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