Research Letter
November 2015

A Randomized Trial Testing US Food and Drug Administration “Breakthrough” Language

Author Affiliations
  • 1Department of Engineering and Public Policy, Carnegie Mellon University, Pittsburgh, Pennsylvania
  • 2The Dartmouth Institute for Health Policy and Clinical Practice, Geisel School of Medicine at Dartmouth, Lebanon, New Hampshire
  • 3Department of Social and Decision Sciences, Carnegie Mellon University, Pittsburgh, Pennsylvania

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JAMA Intern Med. 2015;175(11):1856-1858. doi:10.1001/jamainternmed.2015.5355

In colloquial terms, “breakthrough” connotes an important, definitive advance. Since the 2012 US Food and Drug Administration (FDA) Safety and Innovation Act became law, however, the FDA can assign the breakthrough designation to a drug that “treats a serious or life-threatening condition” and “may demonstrate a substantial improvement…over available therapies” based only on preliminary evidence (eg, uncontrolled studies, surrogate outcomes).1 Such drugs often receive “accelerated approval.”24

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