Appeal type: Prescription Drug
Decision: Unfavorable

You asked the Plan to cover Venclexta. The Plan did not grant this request. We received this case to conduct an independent review.We asked our Physician Reviewer to determine whether the Part D Plan was correct in denying the request for Venetoclax. The drug is requested to treat newly diagnosed acute myelogenous leukemia (AML), which is an off-label (non-FDA approved) use. The enrollee is a 59 year old man with comorbidities and a new diagnosis of AML with panymelosis and high risk myelodysplastic syndrome. He had pancytopenia and fever. He was started on Decitabine. You request Venetoclax to be given with Decitabine to treat him. He has received transfusions. The initial request was for 50 mg pills; 30 per 30 days. And the diagnosis was not clearly AML in the submitted documents. In response to inquiry, you corrected the dose requested and submitted the bone marrow report from December 23, 2019. The report indicates the biopsy shows a diffusely fibrotic marrow. Megakaryocytes are markedly increased in proportion and exhibit significant cytologic atypia. Islands of left shifted myeloid cells and erythroid elements are noted. Mature neutrophils are diminished. The aspirate smears are quite hemodilute secondary to the fibrosis. Increase myeloblasts as well as dysplastic myeloid and erythroid elements are noted on the smear, but an accurate differential count cannot be performed. The estimated proportion of blasts in the dilute smear was 12%. As a formal blast count could not be performed there is uncertainty in the diagnosis. I note that the patient has a complex karyotype. The most likely differential diagnosis includes a myelodysplastic syndrome with excess blasts and fibrosis and acute myeloid leukemia. You stated the diagnosis is AML and cited an article from the New England Journal of Medicine, meeting abstracts and a press release in support of the request. The article does not reflect the enrollee's clinical situation as patients in the trial were all over 60 years of age. The Medicare-approved compendia do not contain any citations to support the use of the requested drug, as prescribed, for treatment of the noted condition. There was no peer-reviewed, clinically relevant, medical literature from an approved journal submitted to support this appeal. Therefore, the requested drug is not being prescribed for a medically accepted indication as defined by Medicare law. As such, an exceptions request based on medical necessity cannot be considered because the requested drug, as prescribed, does not meet the definition of a Medicare Part D drug. Therefore, the requested drug is not eligible for coverage under Medicare Part D for the noted condition.For coverage under Part D, Medicare requires that the drug must be prescribed for a medically accepted indication. For Part D drugs used in an anticancer chemotherapeutic regimen, a medically accepted indication means a use that is approved by the FDA or a use that is supported by one or more citations in Medicare approved compendia or peer-reviewed medical literature in CMS-approved journals or publications. Our Physician Reviewer reviewed this file and determined that there are no citations in the Medicare approved compendia and there was no peer-reviewed literature from an approved journal submitted to support the use of Venclexta for the diagnosed condition. As a result, the drug is not being prescribed for a medically accepted indication as defined by Medicare law. As such, it cannot be covered under the Medicare Part D benefit for this condition. An exceptions request based on medical necessity cannot be considered 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 payment can be made under the Medicare Part D benefit.