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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.”2- 4
Krishnamurti T, Woloshin S, Schwartz LM, Fischhoff B. A Randomized Trial Testing US Food and Drug Administration “Breakthrough” Language. JAMA Intern Med. 2015;175(11):1856-1858. doi:10.1001/jamainternmed.2015.5355