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November 17, 2016

Overall Survival in Cancer Drug Trials as a New Surrogate End Point for Overall Survival in the Real World

Author Affiliations
  • 1Myeloma Service, Division of Hematologic Oncology, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York
  • 2Division of Hematology Oncology, Knight Cancer Institute, Portland, Oregon
  • 3Department of Public Health and Preventive Medicine, Oregon Health & Science University, Portland
JAMA Oncol. Published online November 17, 2016. doi:10.1001/jamaoncol.2016.5296

Cancer regulatory approvals aim to permit drugs to be marketed that can be used to improve survival or quality of life for patients in the United States. Approximately one-third of cancer drugs approved come to market on the basis of demonstrated improvement in overall survival in randomized clinical trials, while two-thirds are approved on the basis of a surrogate end point.1 Although overall survival is an ideal end point, the US Food and Drug Administration (FDA) has demonstrated flexibility in accepting surrogate end points for life-threatening conditions with few alternative treatment options.1 In recent years, however, 3 related findings—survival gains in trials of cancer drugs are marginal, trials of cancer drugs are conducted in unrepresentative populations, and real-world outcomes data find no benefits or diminished benefits of cancer drugs—have coalesced to yield a surprising conclusion: even overall survival in clinical trials of cancer drugs may be a surrogate end point.

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