Reimbursement policies constrain the practice of oncology

To determine the prevalence of off-label anticancer drug use (ie, using drugs to treat conditions other than those listed on the Food and Drug Administration's approved drug label), the extent of reimbursement denials for these uses, and the effect of denials on the treatment of cancer patients.

A three-part questionnaire was sent to a randomized sample of oncologists taken from the 1990 membership of the American Society of Clinical Oncology in March 1990. A response rate of 56% yielded information from 681 oncologists on the drugs they prescribed to 2018 new cancer patients and on those they frequently use in treating 11 specific types of cancer.

The labeled uses of the drugs prescribed and frequently used in chemotherapies were checked against the indications treated to determine whether they were prescribed for off-label uses. Three drug compendia were used as cited sources of off-label drug uses. The extent of reimbursement denials over the last 12 months and their effects on treatments were based on respondent opinions.

Off-label drug use is prevalent and dominates the chemotherapy regimens of cancers that are more difficult to treat and for which there are no cures or agreements on standardized treatment. Most oncologists reported frustration with shifting reimbursement policies in general and with an increasing rate of denials for some off-label drug uses. Indeed, a surprising number of oncologists claimed that these policies caused them to alter preferred treatments and site of care.

These findings suggest the need for public discussion of off-label drug use issues so that appropriate policies can be developed.