Snapshot

The Rare Pediatric Disease Designation and Priority Review Voucher Programs pathway has been developed by the of United States.

This pathway can be used where Drug or biological product for a rare pediatric disease..

This pathway facilitates activities during development.

It is an abridged review (a reliance pathway).

Target time assessment is If a sponsor chooses to request a rare pediatric disease designation, section 529(d)(2) provides that it shall do so “at the same time” that they submit a request for orphan-drug designation under section 526 or a request for fast-track designation under section 506. FDA will recognize a request for rare pediatric disease designation to be submitted “at the same time” as a request for orphan-drug designation or fast track designation if the requests are received by FDA within two weeks of each other. If sponsors submit a timely request for rare pediatric disease designation, section 529(d)(3) directs FDA to make a decision on the request no later than 60 days after submission. The statute directs FDA to decide whether to designate the drug as a drug for a “rare pediatric disease” and whether to designate the application for the drug as “a rare pediatric disease product application”. FDA is willing to accept designation requests submitted at a different time than that provided by statute as long as FDA receives the designation request before FDA has filed the NDA/BLA for the drug for the relevant indication. Although the FDA will aim to respond to such requests in a timely manner, the 60-day response deadline does not apply. FDA will not accept requests for rare pediatric disease designation received after FDA has already filed the NDA/BLA for the drug for the relevant indication. Whether or not a sponsor receives rare pediatric disease designation for its drug, the sponsor must include a request for a rare pediatric disease priority review voucher in its original NDA/BLA submission (either in the initial package or up until the point of NDA/BLA filing) in order to be eligible to receive a voucher. Although FDA’s goal is to take action on the application within 6 months after the 60-day filing period for an application involving a new molecular entity or within 6 months after the date of receipt of an application not involving a new molecular entity, this timeframe is not guaranteed. Note that “take action” in this context means that FDA aims to complete its review of the filed application and issue an approval or complete response letter within this timeframe; it does not mean that the application will be approved within this timeframe. As per section 529(f)(1), FDA will publish a notice in the Federal Register and on its website within 30 days after issuing a priority review voucher under section 529 and within 30 days after approving a drug application for which the sponsor used such a voucher..