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Research Letter
July 17, 2018

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

Author Affiliations
  • 1Yale School of Medicine, New Haven, Connecticut
  • 2Collaboration for Research Integrity and Transparency, Yale University, New Haven, Connecticut
  • 3Department of Internal Medicine, Yale School of Medicine, New Haven, Connecticut
JAMA. 2018;320(3):301-303. doi:10.1001/jama.2018.7619

The US Food and Drug Administration (FDA) Safety and Innovation Act of 2012 created the Breakthrough Therapy designation to expedite development and review of drugs and biologics intended to treat serious or life-threatening conditions for which preliminary clinical evidence may demonstrate substantial improvement over existing therapies, allowing the FDA to provide “intensive guidance on efficient drug development” and “rolling review.”1 Although physicians and patients often perceive that breakthrough approvals are based on rigorous clinical evidence,2,3 no systematic evaluation of the evidence supporting breakthrough approvals has occurred. We reviewed all new FDA approvals granted Breakthrough Therapy designation, characterizing the pivotal clinical trials that serve as the basis of FDA approval,4 and premarket development and review times.