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Evaluating Off-Label Uses of Anticancer Drugs: Time for a Change
- Harold C. Sox, MD, Editor
In the United States, the term off-label use means using approved drugs for clinical indications that the U.S. Food and Drug Administration (FDA) has not approved. Once the FDA has approved a drug, physicians have few constraints on how they use it. In 1996 and 1997, the FDA issued guidance documents that forbade drug companies from advertising off-label uses of drugs. Shortly thereafter, the Food and Drug Administration Modernization Act loosened the strict prohibition, and case law has further freed drug companies to promote off-label indications (1). Now, the major restraint is payers' willingness to pay for the drug. In 1993, the U.S. Congress gave Medicare legal guidance for approving off-label uses of 1 class of drugs—cancer chemotherapy—within the Omnibus Budget Reconciliation Act. That system is now under intense scrutiny, which continues with a key report in this issue. This editorial will contrast the present system for off-label indications with existing alternatives and end with several proposals.
In the United States, policy constrains medical prescribing practices. States can suspend physicians' licenses when they indiscriminately prescribe addictive drugs. The FDA controls access to the market by insisting on evidence from randomized trials that a drug is more effective than placebo for at least 1 indication; it does not regulate other uses for an approved drug. Practice guidelines, which represent a professional consensus about the best drugs in specific clinical situations, often shape policies with real clout: medical insurance coverage and measures of performance and quality. Insurance coverage decisions determine what insurance will pay for. Performance and quality measures have become the standard for evaluating physician and health system performance, which can influence payment. Given their importance, do policies based on practice guidelines use an unbiased selection of the evidence? With guideline programs that use …
