Expediting drug development: Breakthrough therapy designation

Abstract

In 2012, Congress approved the Food and Drug Administration Safety Innovation Act (FDASIA), and as part of that act, a new designation was recognized-breakthrough therapy designation. Breakthrough drugs are drugs that are intended alone, or in combination with one or more other drugs, to treat a serious or life-threatening disease and for which preliminary clinical evidence suggests that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Drugs assigned breakthrough status are granted fast-track status, more extensive FDA guidance on development of the drug, and eligibility for priority and rolling review by the FDA. As of 2015, a total of 384 requests for breakthrough therapy designation status were received by the FDA, of which 118 (31 %) were granted. This chapter summarizes breakthrough therapy designations and reviews specific examples related to oncology drug development.