A recent policy change has broadened Medicare’s coverage of off-label cancer drugs. The new policy took effect in November 2008.

The Food and Drug Administration (FDA) approves drugs for the treatment of specific conditions or diseases based on the results of clinical trials. But many physicians prescribe these drugs to patients for conditions other than those for which they have been formally approved, which is known as off-label use. Government spending on off-label prescriptions is an issue that has long been debated.

To determine which off-label uses of cancer drugs to cover, Medicare relies on so-called compendiums, or reference guides about drugs. According to the new Medicare rules, if at least one Medicare-approved compendium supports the off-label use of a drug, Medicare is obliged to pay for its use unless another compendium specifically advises against it. The new Medicare policy change increases the current number of compendiums from one to four, thereby increasing the opportunities for approval of off-label uses of cancer drugs.

Prescribing drugs off-label can give patients access to treatments that may potentially benefit them, as was once the case with thalidomide (Thalomid). For many years, physicians prescribed thalidomide off-label for the treatment of multiple myeloma. Only years later was the drug formally approved by the FDA for this purpose.

However, evidence for the effectiveness of off-label prescriptions is often scarce or lacking entirely. When little is known about a drug’s usefulness against a specific cancer, patients are put at risk of unknown and potentially dangerous side effects. And although they are published by non-profit organizations, compendiums can have ties to the pharmaceutical drug industry, potentially biasing their conclusions.

Nonetheless, the expansion of Medicare-approved off-label prescribing grants physicians the freedom to decide on a case-by-case basis which treatments may be appropriate for their patients. If physicians feel the data suggesting efficacy and safety of a drug is sufficient, they may prescribe it to patients who do not respond to available drugs approved for the disease.

Multiple myeloma patients should consult their physicians for information about how the Medicare policy changes might affect their treatment options. The Medicare policy changes may also affect private health care insurers, which often follow the agency’s lead on such issues.

Further details can be found in a New York Times article and a related Myeloma Beacon article about the policy change.