Medicare provides guidance on the coverage of drugs in the Medicare Benefit Policy Manual, Chapter 15, Section 50.
Specifically, Section 50.4.1 discusses that drugs approved by the Food and Drug Administration (FDA) and used for indications specified on the labeling (package insert) are generally covered by Medicare if they are medically necessary and all other applicable coverage requirements are met.
Off-label uses of an FDA approved drugs, that is when the drug is used for indications other than what is indicated on the official label, may be covered by Medicare if the Medicare Administrative Contractor (MAC) determines the use to be medically accepted. The MAC will consider information from the major drug compendia, authoritative medical literature and/or accepted standards of medical practice. This is discussed in Section 50.4.2.
Section 50.4.5 specifically addresses the off-label use of drugs and biologicals in an anti-cancer chemotherapeutic regimen. “Off-label, medically accepted indications are supported in either one or more of the compendia or in peer-reviewed medical literature.” This section includes a list of the approved compendia and acceptable category/wording to support off-label use.
Section 50.4.5 also gives the following guidance on acceptable literature and a list of acceptable publications. “Peer-reviewed medical literature may appear in scientific, medical, and pharmaceutical publications in which original manuscripts are published, only after having been critically reviewed for scientific accuracy, validity, and reliability by unbiased, independent experts prior to publication.”