Appeal type: Prescription Drug
Decision: Unfavorable

You asked the Plan to cover Venclexta. The Plan did not grant this request. We received this case toconduct an independent review.We asked our Physician Reviewer to determine whether the Part D Plan was correct in denying the requestfor Venclexta 10-50-100 mg starter pack. The drug is requested to treat Waldenstrom's macroglobulinemia(WM), which is an off-label (non-FDA approved) use. The is a 71 year old patient with a longhistory of WM. The patient has been treated with Cytoxan, Prednisone, Rituximab, 2CDA, Velcade,Dexamethasone, Bendamustine, and Ibrutinib. She now has relapse of WM with rising IgM levels and lowplatelet counts. The prescriber requests Venclexta for treatment. Citations including from theJournal of Clinical Oncology that reports a phase I trial that included 4 patients with WM out of 106patients on the trial were submitted and reviewed. The conclusion states additional trials are ongoing. Thisreviewer did not find any additional citations in a PubMed search. In the opinion of this reviewer thesubmitted article does not meet the Medicare criteria for use to determine a medically accepted indication.The other articles cited by the prescriber do not reflect the enrollee's clinical situation.The Medicare-approved compendia do not contain any citations to support the use of the requested drug, asprescribed, for treatment of the noted condition. There was no peer-reviewed, clinically relevant, medicalliterature from an approved journal submitted to support this appeal. Therefore, the requested drug is notbeing prescribed for a medically accepted indication as defined by Medicare law. As such, an exceptionsrequest based on medical necessity cannot be considered because the requested drug, as prescribed, doesnot meet the definition of a Medicare Part D drug. Therefore, the requested drug is not eligible for coverageunder Medicare Part D for the noted condition.For coverage under Part D, Medicare requires that the drug must be prescribed for a medically acceptedindication. For Part D drugs used in an anticancer chemotherapeutic regimen, a medically acceptedindication means a use that is approved by the FDA or a use that is supported by one or more citations inMedicare approved compendia or peer-reviewed medical literature in CMS-approved journals orpublications.Our Physician Reviewer reviewed this file and determined that there are no citations in the Medicareapproved compendia and there was no peer-reviewed literature from an approved journal submitted tosupport the use of Venclexta for the diagnosed condition. As a result, the drug is not being prescribed for amedically accepted indication as defined by Medicare law. As such, it cannot be covered under theMedicare Part D benefit for this condition. An exceptions request based on medical necessity cannot beconsidered because this drug, as prescribed, does not meet the definition of a Medicare Part D drug.Therefore, our decision is that the Plan is not required to provide coverage for Venclexta.This decision does not concern the medical necessity of the requested drug. It only establishes if paymentcan be made under the Medicare Part D benefit.