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  1. Developing Products for Rare Diseases & 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.

During this public health emergency associated with the COVID-19 pandemic, the Office of Orphan Products Development (OOPD) is providing sponsors with increased flexibility for submission of orphan drug designation requests and related submissions (amendments, annual reports, etc.). During this public health emergency, orphan drug designation, humanitarian use device designation, and rare pediatric disease designation requests and submissions may be submitted electronically by email to the OOPD.

When transmitting information to the Orphan Drug Designation Program via email, please utilize the mailbox orphan@fda.hhs.gov. The use of automated read receipt is recommended to avoid the need to call to verify receipt of the email. Sponsors and others who plan to email information to FDA that is considered to be private, sensitive, proprietary, or commercial confidential are strongly encouraged to send it from an FDA secured email address so the transmission is encrypted. The OOPD will assume that the addresses of emails received or email addresses provided as a point of contact are FDA secure when responding to those email addresses.

Sponsors and others can establish a secure email address link to FDA by sending a request to SecureEmail@fda.hhs.gov. There may be a fee to a commercial enterprise for establishing a digital certificate as part of the set-up process before emails can be sent to FDA encrypted.

RPD Designation Program Sunset Provisions

According to the current statutory sunset provisions for the Rare Pediatric Disease Priority Review Voucher Program, after September 30, 2020, 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, 2020. After September 30, 2022, FDA may not award any rare pediatric disease priority review vouchers.

Requests for rare pediatric disease designation submitted within two weeks of a request for fast track designation or orphan drug designation are entitled to a 60-day review. Requests for rare pediatric disease designation not submitted with a request for fast track designation or orphan drug designation are reviewed in a timely manner, however, the 60-day response time does not apply. Friday, July 31, 2020, is the last business day that is not less than 60 days prior to September 30, 2020. The Offices of Orphan Products Development and Pediatric Therapeutics will continue to review all rare pediatric disease designation requests, but we cannot commit to providing a response to requests received after July 31, 2020.

If you submit an orphan-drug designation and/or fast track request at the same time that you submit a rare pediatric disease designation request, note this in the cover letter for the rare pediatric disease designation request or directly notify the Office of Orphan Products Development at orphan@fda.hhs.gov. See FDA’s Draft Guidance on Rare Pediatric Disease Priority Review Vouchers for additional details.

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):

CDERONDRareDiseaseProgram@fda.hhs.gov or either 301-796-4842 or 301-796-4061

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

Julienne.Vaillancourt@fda.hhs.gov or 301-796-1827

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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