21 U.S.C. § 360ff — Priority review to encourage treatments for rare pediatric diseases

(a) Definitions. This section uses these terms. "Priority review" means the FDA reviews and acts on a drug application within 6 months of receiving it, based on FDA policy manuals and goals set in 2012 user-fee letters. A "priority review voucher" is a certificate the FDA gives to a rare pediatric disease drug's sponsor. It lets the holder get priority review of one other drug application later. A "rare pediatric disease" is a serious or life-threatening disease that mainly affects people from birth to age 18, and that also counts as a rare disease under section 360bb. A "rare pediatric disease product application" is an application for a drug or biologic that treats a rare pediatric disease, where: the drug's active ingredient has never been approved before in another application; the FDA finds it eligible for priority review; it relies on clinical data from studies of children and child-sized doses; it does not seek approval for use in adults; and it is approved after September 30, 2016. (b) Priority review voucher. The FDA must award a voucher when it approves a rare pediatric disease product application. The sponsor can sell or otherwise transfer the voucher, and there is no limit on how many times it can be transferred before it's used. Whoever receives a transferred voucher must tell the FDA within 30 days. A sponsor cannot get a voucher if its application was filed before October 7, 2012 (90 days after July 9, 2012). Starting December 29, 2016 (90 days after September 30, 2016), a sponsor that wants a voucher must tell the FDA when it submits its application. Sponsors whose applications were filed but not yet approved by September 30, 2016 can still qualify if they filed on or after October 7, 2012 and on or before September 30, 2016, and otherwise meet the requirements. Before using a voucher, a sponsor must notify the FDA at least 90 days before submitting the application that will use it; that notice is a binding promise to pay the required fee. After giving that notice, the sponsor can still transfer the voucher, as long as it hasn't yet submitted the application. The FDA cannot award any new vouchers under this section after September 30, 2029. (c) Priority review user fee. Sponsors using a voucher must pay a special fee, on top of any other required fees. Each year, the FDA sets the fee amount based on the gap between the average cost of reviewing a priority-review application and the average cost of reviewing a regular one, in the prior fiscal year. The FDA must set each year's fee before that fiscal year starts (for years after September 30, 2012). The fee is due when the application is submitted. If a sponsor asks to use a voucher but doesn't pay this fee and all other required fees, the FDA treats the application as incomplete. The FDA cannot waive, exempt, reduce, or refund this fee. Fees collected go into the FDA's own funding account, and the FDA can only collect them in years when Congress's appropriations law allows it. (d) Designation process. A drug's manufacturer or sponsor can ask the FDA to officially designate the drug as treating a rare pediatric disease, and its application as a rare pediatric disease product application. This request must be made at the same time as a request for orphan-disease status or fast-track designation, but getting this designation is not required to receive a voucher. Within 60 days of the request, the FDA must decide whether the disease qualifies as a rare pediatric disease and whether the application qualifies. (e) Marketing of rare pediatric disease products. The FDA can take back a voucher if the drug it was awarded for is not being sold in the U.S. within 365 days of approval. The sponsor of an approved drug must send the FDA a report within 5 years of approval, covering each of the first 4 years after approval: the estimated number of U.S. patients with the disease, the estimated U.S. demand for the drug, and how much of the drug was actually distributed in the U.S. (f) Notice and report. Within 30 days of awarding a voucher, or of approving a drug for which a voucher was used, the FDA must publish notice in the Federal Register and on its website. If, more than a year after the third voucher is awarded, a sponsor uses a voucher for an application, the FDA must tell two Congressional committees which drug used the voucher. (g) Eligibility for other programs. Using this voucher program doesn't stop a sponsor from also using other incentive programs. But no sponsor can get more than one priority review voucher, under any section of this chapter, for the same drug's application. (h) Relation to other provisions. This section adds to, but doesn't replace, other laws that encourage developing drugs for tropical and rare pediatric diseases. (i) GAO study and report. Once the third voucher is awarded, the Comptroller General must study how well this voucher program works. The study must look at: what each rare-disease drug was approved for; whether the program actually met an unmet medical need; how much a transferred voucher was worth; which drugs used vouchers; and how much time passed between a voucher being awarded and being used. Within 1 year of the study starting, the Comptroller General must report the results to the same two Congressional committees.