Medicare recently increased the number of drug compendia that it relies on when deciding whether to cover the off-label use of a drug. This policy change will likely result in expanded coverage of off-label uses of cancer drugs. While a previous Myeloma Beacon article reported on the expanded coverage, this article takes a closer look at the compendia and at what this policy change might mean for multiple myeloma patients.

Off-label prescribing is when a drug is prescribed for purposes other than for which the drug is formally approved by the Food and Drug Administration (FDA). Compendia are authoritative drug reference books that present findings from clinical research, including indications that support specific off-label uses of drugs. The compendia are especially important for the treatment of cancer, where up to 75 percent of treatments are used off-label. Often, there are few FDA-approved treatment options for rare cancers, so physicians need the ability to try other treatments.

Four compendia were approved by Medicare in 2008: the American Hospital Formulary Service - Drug Information (AHFS-DI) published by the American Society of Health-System Pharmacists, the National Comprehensive Cancer Networks' (NCCN) Drug Information & Biologics Compendium, Thomson Micromedex's DrugDex, and Elsevier Gold Standard’s Clinical Pharmacology. Medicare is expected to review its list of compendia each year.

At a forum held by the Foundation for Evidence-Based Medicine in September 2008, representatives from each of the publishers presented overviews of their publications, details about their review processes, and their clearly defined standards for entering new listings.

Each of this year's four compendia accepts applications for new entries from payers, industry, and providers. Information used to evaluate these applications comes directly from the scientific literature. None of the publishers cull information from other compendia. Medical experts do not make recommendations with reimbursement in mind, but some publishers may incorporate this element in the future. Currently, the AHFS-DI indicates certain uses as a "reasonable choice," which may be relevant to reimbursement costs. Only the AHFS-DI specially marks new or updated information.

Furthermore, each publisher explained that their policies are designed to mitigate conflicts of interest. The NCCN, for example, passes information through a board of 30 doctors, which they say is a large enough number to override the bias of any one person.

The NCCN compendium is the only compendium that can be accessed for free by registering an email address. It lists appropriate uses of drugs for each specific cancer. Potential off-label uses are categorized based on the level of support for the use. Off-label uses listed under Category 1 or 2A are "medically accepted indications," with high levels of evidence uniformly supporting their use. Uses listed under Category 3 are not medically recommended. If not mentioned (i.e., Category 2B), then the evidence is neither supportive nor non-supportive of the use.

According to the NCCN, the following drugs will be covered for "smoldering myeloma (asymptomatic or stage I) that has progressed beyond stage II or active myeloma (symptomatic, all stages)," the majority of which are conclusively approved for non-transplant candidates only: dexamethasone (Decadron), Doxil (doxorubicin), melphalan (Alkeran), prednisone, Revlimid (lenalidomide), thalidomide (Thalomid), vincristine (Oncovin), Velcade (bortezomib).

Ultimately, patients will see the effects of this policy change in the form of reimbursements for drugs previously not covered by Medicare. Ask your doctor to examine the newly approved compendia to discover how the additional compendia may benefit you.

For more information on the policy change, please refer to the summary published by Louisiana Medicare Services. To read more on the September forum, please access the article at the Foundation of Evidence-Based Medicine. The New York Times has also reported on the issues related to the policy change.