Drug compendia DRUGDEX and DrugPoints, used by the Centers for Medicaid and Medicare Services (CMS) as legally approved guidance documents regarding reimbursement for off-label uses of drugs (that is, for uses other than that defined by the FDA in their approval of the drug), have listed Revlimid in combination with dexamethasone as an off-label frontline therapy for myeloma. Currently, Revlimid has been FDA approved only for myeloma patients who have had at least one prior therapy. Drug compendia can list an off-label use for a drug if at least two clinical studies that demonstrate the efficacy of the drug in the as yet unapproved setting have been published. Such a listing not only sets the groundwork for Medicare reimbursement of frontline Revlimid, but may ease the process of reimbursement from other insurers as well.
Following is the listing in the DRUGDEX and Drug Point compendia for Revlimid in the frontline setting:
Multiple myeloma, In combination with dexamethasone, first-line therapy
FDA Approval: Adult, no; Pediatric, no Efficacy: Adult, Evidence favors efficacy Recommendation: Adult, Class IIa Strength of Evidence: Adult, Category B
Summary: Lenalidomide, in combination with dexamethasone, demonstrated a favorable overall response rate in a single-arm, phase II trial (n=34) when used as first-line therapy in patients with newly diagnosed multiple myeloma (Rajkumar et al, 2005).
In an unpublished, randomized, phase III trial (n=445), all patients receiving lenalidomide and dexamethasone as first-line therapy for multiple myeloma had high 1-year and overall survival rates; however, patients treated with lenalidomide plus low-dose dexamethasone had significantly higher 1-year and overall survival rates and a lower incidence of nonhematologic toxicity than patients treated with lenalidomide plus high-dose dexamethasone (Rajkumar, 2007; Rajkumar et al, 2007).