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Focus on Innovation: FDA’s Rare Pediatric Disease Priority Review Voucher Program

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On June 30, 2015, Gayatri R. Rao, M.D., J.D., of the FDA's Office of Orphan Products Development (OOPD) presented an overview on the Rare Pediatric Disease Priority Review Voucher Program. Dr. Rao discussed the background and purpose of the program, application and review process, sunset provision, and what's next for the program followed by a short question and answer session.

Given the need for new approved therapies in a vulnerable population, the Rare Pediatric Disease Priority Review Voucher Program was enacted in the Food and Drug Administration (FDA) Safety and Innovation Act of 2012 to incentivize product development for rare pediatric diseases. For an eligible rare pediatric disease (RPD) marketing application, such as New Drug Application or Biologic License Application, the FDA will grant a priority review voucher (PRV) to sponsors upon its approval. The sponsor can redeem the voucher toward the FDA review of a future drug in its pipeline or sell the voucher to another company. The use of a priority review allows for a shortened FDA review timeline for a marketing application that would not otherwise receive priority review-6 months for a priority review versus 10 months for a standard review.

Since the program’s establishment, the FDA has granted three RPD priority review vouchers. The applications covered the treatment of mucopolysaccharidosis type IVA, neuroblastoma, and a single enzyme defect leading to bile acid synthesis and peroxisomal disorders. Two of the PRVs were sold for $67.5 million and $245 million, while one remains unsold. When the third voucher was awarded on March 17, 2015, it triggered the sunset clause, with the program now set to expire on March 16, 2016. After the last day of the 1-year period of the sunset provision, no further vouchers can be awarded unless Congress extends the program.

A couple of highlights from the presentation and interview include

  • An overview of the number of RPD requests and determinations since the inception of the program in 2012
  • Dr. Rao’s recommendation that from an efficiency standpoint, it may be in the sponsor’s best interest to submit the RPD request prior to submitting the marketing application to the Center for Drug Evaluation and Research or the Center for Biologics Evaluation and Research, as in either process, her office will be consulted for the determination

For additional information, read the draft guidance and browse the FDA’s website.

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