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Research Letter
September 2014

Publication of Pivotal Efficacy Trials for Novel Therapeutic Agents Approved Between 2005 and 2011: A Cross-sectional Study

Author Affiliations
  • 1Yale University School of Medicine, New Haven, Connecticut
  • 2Department of Internal Medicine, Yale University School of Medicine, New Haven, Connecticut
JAMA Intern Med. 2014;174(9):1518-1520. doi:10.1001/jamainternmed.2014.3438

Pivotal efficacy trials, which are trials that form the basis of the US Food and Drug Administration’s (FDA) decision to approve a novel therapeutic agent,1 have great relevance to clinical practice because when these therapies are first approved for use, few clinical trials have been conducted. However, studies focused on pharmacotherapies approved prior to 2005 found that more than one-quarter of these trials were not published,2,3 although efforts to promote clinical trial transparency and dissemination have since intensified.4 We determined publication status and timeliness among pivotal trials supporting the approval of novel therapeutics by FDA from 2005 through 2011.