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July 13, 2021

Fulfilling the Mandate of the US Food and Drug Administration’s Accelerated Approval Pathway: The Need for Reforms

Author Affiliations
  • 1Departments of Oncology and Public Health Sciences, Queen’s University, Kingston, Ontario, Canada
  • 2Section of General Medicine and the National Clinician Scholars Program, Department of Internal Medicine, Yale School of Medicine, New Haven, Connecticut
  • 3Program On Regulation, Therapeutics and Law (PORTAL), Department of Medicine, Brigham and Women’s Hospital, Harvard Medical School, Boston, Massachusetts
JAMA Intern Med. Published online July 13, 2021. doi:10.1001/jamainternmed.2021.4604

The US Food and Drug Administration (FDA) initiated the accelerated approval pathway in 1992 in response to the HIV epidemic as a way to expedite clinical trial evaluations of promising drugs in the treatment of serious or life-threatening disease. The accelerated approval pathway explicitly allows biomarkers to be used as primary efficacy end points and permits those biomarkers to be only “reasonably likely” to predict clinical benefit. When a drug receives accelerated approval, there is usually lesser certainty of clinical benefit than if approval were based on clinical outcomes, such as improvements in how a patient feels or functions or prolonged survival. In contrast, the FDA’s regular approval pathway is typically based on clinical outcomes, although in recent years the agency has permitted the use of surrogate markers for clinical benefit with increasing frequency.1

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