§360ff Priority Review to Encourage Treatments for Rare Pediatric Diseases

Summary

The FDA must give a priority review voucher to the company that gets approval for a qualifying drug for a rare pediatric disease. Priority review means the FDA will act on a drug application within 6 months. A priority review voucher is a transferable ticket that lets its holder get priority review for one later drug application after the rare pediatric drug is approved. A rare pediatric disease is a serious or life‑threatening condition that mainly affects people from birth through 18 and is rare. A rare pediatric disease product application is a new drug or biologic application for that disease that relies on pediatric clinical data, does not ask for an adult use in the original filing, contains no previously approved active ingredient, and is approved after September 30, 2016. Vouchers can be sold or transferred any number of times. Each new owner must tell the FDA within 30 days. A sponsor won’t get a voucher for an application filed before the date 90 days after July 9, 2012. Starting 90 days after September 30, 2016, sponsors who want a voucher must tell the FDA when they submit the rare pediatric application. Sponsors whose rare pediatric applications were filed between 90 days after July 9, 2012 and September 30, 2016 and were not yet approved by September 30, 2016 can still be eligible. A voucher can’t be awarded after September 30, 2029. To use a voucher, the sponsor must notify the FDA at least 90 days before filing the later drug application and must pay a special user fee when they submit that application. The FDA will set that fee each year based on the extra cost of priority reviews. The fee is due at submission, can’t be waived, and fees go to the FDA account as provided by appropriations. The FDA may take back a voucher if the approved rare pediatric product is not marketed within 365 days of approval. Five years after approval, the sponsor must report on estimated patients, estimated demand, and actual product distributed for each of the first four years. The FDA must publish notices within 30 days when it awards a voucher and when it approves a drug that used a voucher. After the third voucher is awarded, the Comptroller General must study the program and report to Congress within one year, and the FDA must notify Congress when certain vouchers are later used. This program adds to other incentives and does not replace them, and a sponsor cannot get more than one voucher for the same drug under the chapter.