The Association of Community Cancer Centers (ACCC) reports that the growth of off-label prescribing has slowed since 2005.

The term "off-label" refers to pharmaceuticals prescribed for purposes other than for which the drug is formally approved by the Food and Drug Administration. Doctors most commonly prescribe drugs off-label when the scientific and clinical evidence supports its use, there are few or no approved treatments for the condition, or other therapies were not effective. Off-label use of drugs is fairly common and accounts for up to one-fifth of all drugs prescribed.

Once an FDA-approved drug undergoes two successful clinical trials for another condition, it can be listed for off-label use in drug compendia. These compendia are used by many physicians when deciding whether to prescribe off-label treatments, and compendia are used by the Centers for Medicare and Medicaid Services (CMS) as legally approved guidelines for the reimbursement of off-label drug use. However, Medicare still provides limited coverage of off-label treatments, excluding some as too experimental.

According to doctors like Kenneth Hoffman, an oncologist in Teaneck, New Jersey, the most important off-label prescribing currently occurring in multiple myeloma is the use of Revlimid ^[1] (lenalidomide) as front-line therapy. Although there are studies supporting the use of Revlimid as a front-line therapy, it is only approved by the FDA for use in combination with dexamethasone ^[2] for myeloma patients who have had at least one prior treatment.

In the ACCC report, 50 percent of doctors surveyed believe that off-label use of cancer therapies is extremely important. But according to the respondents, reimbursement and coverage issues have caused them to cut back. Specifically, 26 percent of doctors surveyed said that off-label use has increased in the past five years, compared with 42 percent in a 2005 survey.

Among the doctors surveyed, 40 percent to 45 percent frequently changed their usage of off-label treatments in response to private payer and Medicare policies. Restrictions on off-label use can slow down the process of obtaining approval for reimbursement and can delay treatment. The survey reports that 71 percent of doctors have seen treatment delays of more than a week.

Recently, the CMS expanded the number of compendia used to approve off-label prescribing This policy change may reduce the number of coverage and reimbursement issues and lead to increased off-label use of cancer drugs.

For more information, read about the survey on OncologyStat ^[3] and access the original report by the Association of Community Cancer Centers ^[4]. More information about the recent Medicare policy change can be found in a related Myeloma Beacon ^[5] article.