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Comment & Response
April 11, 2017

FDA Approval of Eteplirsen for Muscular Dystrophy—Reply

Author Affiliations
  • 1Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women’s Hospital, Boston, Massachusetts
JAMA. 2017;317(14):1481-1482. doi:10.1001/jama.2017.2605

In Reply The accelerated approval pathway was developed in the early 1990s by the FDA, not in 2012 by Congress,1 but it did not change the fundamental legal requirement that manufacturers must present “substantial evidence” of a drug’s efficacy derived from “adequate and well-controlled clinical trials” to the FDA prior to approval.2 Drs Nelson and Miceli, by contrast, would accept new drugs based on small changes in laboratory tests, along with the “collective clinical experience” of clinicians and historical controls. The nation abandoned this low evidentiary standard half a century ago in favor of clinical trials, precisely because the conventional professional wisdom criterion often led to the use of ineffective or unsafe products. Recently, many drugs appeared to have promising biomarker results, only to fail in tests that measured patient benefit.3,4 In the eteplirsen pivotal trial, the observed induced dystrophin production was of trivial magnitude, far below anything likely to help patients.