Reimbursement for cancer treatment: coverage of off-label drug indications.

Approximately half of the uses of anticancer chemotherapy drugs are for indications other than those referenced in the United States Food and Drug Administration approved label. Some managed care organizations and private health insurance plans have declined to reimburse the cost of drugs used off-label to treat cancer on the ground that these uses are "experimental" or "investigational." Cancer patients and their providers have experienced similar problems in the Medicare and Medicaid program. To a large extent, these issues have been addressed through legislation enacted in 1993 that requires coverage of medically appropriate cancer therapies including off-label uses recognized by established drug compendia and peer-reviewed literature. Congress has fashioned a system that has worked well, as reflected in improvements in cancer morbidity and mortality. Now, however, after more than a decade of success, the system requires attention. This statement of policy from the American Society of Clinical Oncology encourages the Secretary of the United States Department of Health and Human Services to address these unmet needs in order to ensure that patients with cancer have access to clinically appropriate treatment, as reflected in timely compendia listings and reports of studies in the medical literature.