Off-label pharmaceutical use

An Advanced Beneficiary Notice should be obtained, as well as special informed consent.

Your patient recently read about new treatment options for age-related macular degeneration in the AARP magazine. It references the colon cancer drug Avastin (bevacizumab, Genentech) as a possible treatment for the wet (exudative) form of AMD. She is interested in learning more about this therapy and is also concerned about Medicare coverage for it.

Will Medicare cover the off-label use of a medication?

The answer is, “Sometimes.” The Medicare Benefit Policy Manual, Chapter 15, §50.4.2 states, “An unlabeled use of a drug is a use that is not included as an indication on the drug’s label as approved by the [Food and Drug Administration]. FDA-approved drugs used for indications other than what is indicated on the official label may be covered under Medicare if the carrier determines the use to be medically accepted, taking into consideration the major drug compendia, authoritative medical literature and/or accepted standards of medical practice.”

There are several criteria that must be met for coverage to be considered. They include the following:

Were standard, accepted treatments, including approved drugs, tried or contraindicated before considering a drug for an off-label use?

Do any of the major drug compendia and related peer-reviewed scientific articles support the off-label use (MCM 2049.1)?

Do any specialty society publications recommend this off-label use?

Is this off-label use an accepted standard of medical practice?

Is authoritative medical literature available to support this use? Are the level of evidence, endpoint measured, and number of patients adequate (MCM 2049.C.3)?

The “acceptable drug compendia” include the U.S. Pharmacopoeia Drug Information (USPDI), the American Hospital Formulary Service Drug Information and American Medical Association Drug Evaluations. Peer-reviewed medical literature would include publications in the American Journal of Medicine, the Journal of the American Medical Association, the New England Journal of Medicine and similar journals.

It is important to note that if the drug is considered noncovered, the method of administration is also noncovered. MBPM, Chapter 15, 50.4.2, states, “If a medication is determined not to be reasonable and necessary for diagnosis or treatment of an illness or injury according to these guidelines, the carrier excludes the entire charge (ie, for both the drug and its administration). Also, carriers exclude from payment any charges for other services (such as office visits) which were primarily for the purpose of administering a noncovered injection (ie, an injection that is not reasonable and necessary for the diagnosis or treatment of an illness or injury).”

If coverage of the procedure and drug is uncertain, the patient must be made aware of his or her potential financial liability. Get a signed Advance Beneficiary Notice (ABN) whenever you believe that Medicare is likely to deny your claim for an item or service you are planning to provide. Always get it signed in advance, before providing the item or service. Submit your claim with modifier GA appended to the appropriate CPT or HCPCS code.

By signing an ABN, the Medicare beneficiary acknowledges that he or she has been advised that Medicare will probably not pay for the item or service and agrees to be responsible for payment, either personally or through other insurance. Medicaid qualifies as “other insurance” so get an ABN even for Medicare-Medicaid patients. When providing noncovered services, physicians are permitted to collect payment from the patient at the time of service.

Beside the financial discussion that accompanies the use of an off-label drug, the consent form for these procedures is unique. In addition to the conventional language in an informed consent form addressing risks, benefits and alternatives to the procedure, the consent form should clearly state that the drug is being used in an off-label fashion and that there is little evidence available describing the efficacy of the treatment or possible side effects. It also should explain in easy-to-understand terms the meaning of an off-label use. Care must be taken to address the lack of data in a balanced manner so as not to lead the patient to believe that there are no side effects. The informed consent also might mention that collection of this type of data is under way.

In the case of Avastin, there are few Medicare policies published to date, but they are unanimous in the determination to deem Avastin to treat wet AMD as investigational and noncovered. That decision pertains to both the injectable medication and intravitreal injection.