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November 5, 2020

Drug Approvals in Hepatocellular Carcinoma—Filling the Nonexistent Gap?

Author Affiliations
  • 1Division of Medical Oncology, Department of Medicine, Mayo Clinic, Rochester, Minnesota
  • 2Division of Hematology/Oncology, Department of Medicine, University of California, San Francisco
  • 3Department of Epidemiology and Biostatistics, University of California, San Francisco
JAMA Oncol. 2021;7(2):173-174. doi:10.1001/jamaoncol.2020.4811

Hepatocellular carcinoma (HCC) is an aggressive cancer that mostly affects patients with chronic liver disease and cirrhosis. Hepatocellular carcinoma is usually diagnosed late in its course; hence, the disease has a median survival of only 6 to 20 months.1 In addition to numerous surgical and interventional therapies, systemic molecularly targeted therapies and immune checkpoint inhibition form an important backbone in the treatment of HCC. Recently, the US Food and Drug Administration (FDA) approved the combination of nivolumab and ipilimumab for use in patients with HCC previously treated with sorafenib tosylate.2 In this Viewpoint, we critically appraise the evidence behind FDA drug approvals for HCC.

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