Appeal type: Prescription Drug
Decision: Unfavorable

You asked the Plan to cover Xospata, 40 mg tablet. 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 Xospata, 40 mg tablet; 28/14.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 71 year old man with newly diagnosed AML. His leukemia has an FLT3 mutation by a non-FDA approved companion test. He has been treated with Hydrea, intrathecal Cytarabine, Decitabine, Venetoclax and Gilteritinib (Xospata). He developed renal failure requiring hemodialysis and chemotherapy was held. Repeat bone marrow was hypocellular but flow cytometry was positive for his FLT3 positive disease. The prescriber states they are unsure of the next course of treatment. The prescriber requests Xospata to treat him.Xospata is approved and supported for relapsed or refractory AML. The submitted documentation does not support that the enrollee has either relapsed or refractory AML. Therefore the requested use is not a medically accepted indication. 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. This reviewer did not find any supporting citations in a PubMed search. 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 Xospata, 40 mg tablet 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 Xospata, 40 mg tablet. 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.