Clinical Trial Evidence Supporting FDA Approval of Drugs Granted Breakthrough Therapy Designation

JAMA. 2018 Jul 17;320(3):301-303. doi: 10.1001/jama.2018.7619.


This study used the Drugs@FDA database to review FDA-approved “breakthrough” therapies from 2012 to 2017 (characterizing the pivotal clinical trials that serve as the basis of FDA approval), and premarket development and review times to assess the strength of evidence supporting breakthrough approvals.

MeSH terms

  • Clinical Trials as Topic*
  • Drug Approval*
  • Drug Discovery
  • Humans
  • Statistics, Nonparametric
  • United States
  • United States Food and Drug Administration