Rare Pediatric Disease Designation and Priority Review Voucher Programs

On this page:

• About rare pediatric disease designation and priority review vouchers (PRVs) 
• 2024 update: Rare Pediatric Disease PRV program sunset
• Important information for sponsors on submitting designation requests in 2024
• Requesting rare pediatric disease designation
• Contact information
• Related links

About rare pediatric disease designation and priority review vouchers (PRVs) 

The rare pediatric disease PRV program aims to incentivize drug development for rare pediatric diseases. 

Under this voucher program, a sponsor who receives an approval for a drug or biological product for a rare pediatric disease may qualify for a voucher that can be redeemed to receive priority review for a different product. The sponsor may also transfer or sell the voucher to another sponsor. 

FDA awards rare pediatric disease PRVs to sponsors of rare pediatric disease products that are approved and meet certain criteria. Prior to submitting a marketing application for its drug, a sponsor that plans to request a rare pediatric disease PRV may request rare pediatric disease designation.  

See FDA’s Draft Guidance on Rare Pediatric Disease Priority Review Vouchers.

2024 update: Rare Pediatric Disease PRV program sunset

Updated September 27, 2024: Under the current provisions in the law, as amended by the Continuing Appropriations and Extensions Act, 2025, the rare pediatric disease PRV program will begin to sunset after December 20, 2024. Section 529(b)(5) of the Federal Food, Drug, and Cosmetic Act states:

• (5) TERMINATION OF AUTHORITY.—The Secretary may not award any priority review vouchers [. . .] after December 20, 2024, unless the rare pediatric disease product application— 
  • (A) is for a drug that, not later than December 20, 2024, is designated [. . .] as a drug for a rare pediatric disease; and
  • (B) is, not later than September 30, 2026, approved under section 505(b)(1) of this Act or section 351(a) of the Public Health Service Act.

Important information for sponsors on submitting designation requests in 2024

We will strive to respond to each rare pediatric disease designation request within the applicable review timeline. See FDA’s Draft Guidance on Rare Pediatric Disease Priority Review Vouchers for details regarding review timelines. However, given the resources available to review such requests, if we receive a surge in requests in the few months prior to the deadline, meeting all review timelines may not be feasible. If FDA is unable to grant a request for rare pediatric disease designation by December 20, 2024, a marketing application for that drug cannot be eligible for a voucher under the current provisions of the law. 

Requesting rare pediatric disease designation

See FDA’s Draft Guidance on Rare Pediatric Disease Priority Review Vouchers for details about what to include in your request.

The sponsor should indicate in the rare pediatric disease designation request whether or not it is requesting orphan-drug designation or fast track designation at the same time.

Send requests for rare pediatric disease designation to orphan@fda.hhs.gov or by mail to: 

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

FDA-secured email

Sponsors and others who plan to email information to FDA that is private, sensitive, proprietary or commercial confidential are strongly encouraged to send it from an FDA-secured email address so the transmission is encrypted. The agency will assume the addresses of emails received or email addresses provided as a point of contact are 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 before encrypted emails can be sent to FDA.

Contact information

• Contact orphan@fda.hhs.gov or 301-796-8660 with questions about rare pediatric disease designation.
• Contact PriorityReviewVouchers@fda.hhs.gov or 301-796-1394 with questions about drug products and rare pediatric disease PRV eligibility.
• Contact industry.biologics@fda.hhs.gov or 240-402-8020 with questions about biologic products and rare pediatric disease PRV eligibility.

Related links

• Fast Track Designation Requests
• Designating an Orphan Product: Drugs and Biological Products
• Fee Rate for Using a Priority Review Voucher in Fiscal Year 2024 (Federal Register notice)