Appeal type: Prescription Drug
Decision: Unfavorable

You asked the Plan to cover Jakafi. 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 Jakafi (Ruxolitinib). The drug is requested to treat acute myelogenous leukemia (AML), which is an off-label (non-FDA approved) use.The enrollee is a 66-year-old man with multiple comorbidities and newly diagnosed AML. There is a JAK2 mutation present in the AML cells. The bone marrow reported on 4/17/20 states the morphologic findings and ancillary studies are most consistent with a myeloproliferative neoplasm (favor primary myelofibrosis) now with disease progression, either accelerated phase or blast transformation. He has massive splenomegaly. The prescriber requests Jakafi to be given with Decitabine to treat him.The prescriber states the enrollee is receiving the drug in the hospital and requires outpatient treatment. The prescriber submitted a 2018 article reporting a phase I study from a non-CMS approved journal, Blood Advances, in support of the request. The article summarizes that required patients to have a preexisting diagnosis of essential thrombocythemia, polycythemia vera or primary myelofibrosis (MF). The enrollee does not have a preexisting diagnosis of those disorders.A page from the NCCN Guidelines Version 3.2019 Myelofibrosis was also submitted. The page indicates treatment with hypomethylating therapy or low intensity induction chemotherapy. A footnote indicates Ruxolitinib (Jakafi) may be continued near to the start of therapy for the improvement of splenomegaly and other disease related symptoms. This reviewer interprets this to mean Ruxolitinib is not a part of the treatment of the advanced phase or AML phase of the disorder but may be continued if being given for MF.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 Jakafi 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 Jakafi. 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.