Off-Label Drugs

Pursuant to a congressional request, GAO examined the prevalence of the off-label use of anticancer drugs, focusing on: (1) the extent to which approved anticancer drugs are being prescribed for off-label uses, and how patient characteristics, therapeutic intent, and type of cancer influence their use; (2) third party reimbursements to physicians that prescribe anticancer drugs for off-label uses; and (3) ways in which physicians alter the way they treat cancer patients because of difficulties in obtaining reimbursement for off-label drug use.

GAO surveys found: (1) prescribing of off-label cancer drugs to treat other types of cancer is widespread, and off-label use has generally increased in treating the more difficult cases including cases in which the cancer has advanced to the point where it is no longer curable or chemotherapy is relatively ineffective; (2) more than half of the surveyed reported reimbursement problems for the use of drugs off-label as well as for the setting where they were administered; (3) reimbursement problems varied significantly across the 11 states reviewed; (4) there was no relationship between drug regimen costs and the likelihood that payment would be denied; and (5) oncologists reported that reimbursement policies and cost of certain drugs have altered their preferred treatment and forced them to change the setting in which they treat patients, by admitting patients to the hospital solely to circumvent restrictions imposed by reimbursement policies.

Recommendations

Our recommendations from this work are listed below with a Contact for more information. Status will change from "In process" to "Open," "Closed - implemented," or "Closed - not implemented" based on our follow up work.

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