title: Drug development in serious diseases: the new "breakthrough therapy" designation.
abstract: The US Food and Drug Administration Safety and Innovation Act (FDASIA) was signed on 9 July 2012. This law created a new "breakthrough therapy" (BT) designation program for investigational drugs and biologics intended to treat a serious disease and having "preliminary clinical evidence [that] indicates that the drug may demonstrate substantial clinical improvement over existing therapies on one or more clinically significant endpoints."(1) Many questions have been posed about the impact of this new program on drug development.
authors.affiliation: Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
authors.forename: J
authors.initials: J
authors.lastname: Woodcock
mesh_terms: D017277:Drug Approval / Q000331:legislation & jurisprudence*; D015195:Drug Design; D015507:Drugs, Investigational; D023381:Endpoint Determination; D006801:Humans; D014481:United States; D014486:United States Food and Drug Administration / Q000331:legislation & jurisprudence*
chemical_list: D015507:Drugs, Investigational