Use of cancer therapies for indications not approved by the US Food and Drug Administration (FDA), also known as “off-label” use, is widely practiced in oncology care.1 Examples include bevacizumab in metastatic esophageal cancer or cetuximab in metastatic prostate cancer. In the United States, off-label use of 10 common cancer drugs accounted for nearly $5 billion in costs in 2010,1 an amount that likely substantially underrepresents current off-label spending given the escalation in oncology drug pricing and number of available products, but recent comprehensive estimates have not been published.
Green AK, Wood WA, Basch EM. Time to Reassess the Cancer Compendia for Off-label Drug Coverage in Oncology. JAMA. 2016;316(15):1541-1542. doi:10.1001/jama.2016.12770