The FDA Breakthrough-Drug Designation — Four Years of Experience image

The New England Journal of Medicine, April 12, 2018
Jonathan J. Darrow (Student Fellow Alumnus), Jerry Avorn, and Aaron S. Kesselheim,


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In 2012, Congress created the “breakthrough therapy” designation to expedite Food and Drug Administration (FDA) testing and approval of medications that were intended to treat serious or life-threatening conditions and that preliminary evidence suggested may provide a substantial improvement over existing treatments with regard to one or more clinically significant end points. The creation of this designation was motivated by the concept that advances in precision medicine would enable the development of therapies with large treatment effects that were seen early, such that random assignment to receive placebo might be unethical and phase 2 trials could provide sufficient evidence for approval. One of the sponsors of the law, Senator Michael Bennet (R-CO), explained that the pathway was intended to speed the approval of drugs that showed “exceptional results for patients.”

Manufacturers reacted vigorously. According to Janet Woodcock, the head of the FDA Center for Drug Evaluation and Research, the agency was “inundated” with hundreds of requests, despite expectations that only about 2 drugs per year would receive the designation. From 2014 to 2016, a total of 26 (24%) of the 108 new molecular agents and original biologic agents that were approved by the FDA received the breakthrough designation. 

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