Rare Pediatric Disease Priority Review Voucher (PRV) program

Expedite Development of New Pediatric Drugs 

The legislation for priority review of rare pediatric diseases is intended to expedite the development and approval of drugs for serious or life-threatening conditions affecting children. A summary of program and its key provisions follow. 

Definitions 

• Priority Review: Ensures that the FDA reviews and acts upon applications for drugs treating rare pediatric diseases within six months of receipt. 
• Priority Review Voucher: Issued to the sponsor of a rare pediatric disease product application, allowing priority review of a subsequent drug application. 
• Rare Pediatric Disease: Refers to serious or life-threatening diseases primarily affecting individuals from birth to 18 years old, with limited prevalence. 
• Rare Pediatric Disease Product Application: Human drug application for a drug intended to prevent or treat a rare pediatric disease, meeting specific criteria. 

Award of Priority Review Voucher 

Upon approval of a rare pediatric disease product application, the FDA awards a priority review voucher to the sponsor. The voucher can be transferred, allowing other sponsors to benefit from priority review for their drug applications. 

Limitations and Requirements 

Sponsors cannot receive a voucher if their application was submitted before a specified date. Sponsors must notify the FDA of their intent to request a priority review voucher. The rare pediatric disease product application must rely on pediatric clinical data and cannot seek approval for an adult indication initially. 

Priority Review User Fee 

Sponsors of rare pediatric disease product applications must pay a user fee, determined annually by the FDA. The fee is in addition to other user fees associated with the drug application. 

Designation Process 

Manufacturers or sponsors may request designation of a new drug for a rare pediatric disease. The FDA determines whether the disease and the drug application meet the criteria for rare pediatric disease designation. 

Marketing Requirements 

The FDA may revoke a priority review voucher if the rare pediatric disease product is not marketed in the U.S. within a specified period. Sponsors must submit post-approval production reports to provide information on disease prevalence, demand, and distribution of the rare pediatric disease product. 

September 2024 Sunset of the PRV Program 

The Rare Pediatric Disease Priority Review Voucher (PRV) program is set to sunset after September 30, 2024, according to Section 529(b)(5) of the Federal Food, Drug, and Cosmetic Act. This means that if Congress does not reauthorize the Act, FDA will not award any priority review vouchers beyond this date unless certain conditions are met: 

1. The drug must be designated as a rare pediatric disease product by September 30, 2024. 
2. The drug must be approved under section 505(b)(1) of the Act or section 351(a) of the Public Health Service Act by September 30, 2026. 

For sponsors intending to submit designation requests in 2024, it is crucial to note that the FDA aims to respond to each rare pediatric disease designation request within the applicable review timeline. However, if there is a surge in requests close to the deadline, meeting all review timelines may become challenging due to resource constraints. If the FDA cannot grant a request for rare pediatric disease designation by September 30, 2024, the marketing application for that drug will not be eligible for a voucher under the current provisions of the law. 

Summary 

This legislation incentivizes the development of treatments for rare pediatric diseases by expediting the review process and providing financial benefits to sponsors. It aims to address the unmet medical needs of children with serious or life-threatening conditions, ensuring timely access to potentially life-saving therapies. 

However, the program will be ended in September 2024 unless Congress reauthorizes it. 

Links 

Read more about the program on the FDA website. 

Read the USC legislation for the program.