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 anindependent review.We asked our Physician Reviewer to determine whether the Part D Plan was correct in denying the requestfor Jakafi. The drug is requested to treat chronic myeloproliferative disease which is an off-label (non-FDAapproved) use.The enrollee is a 59 year old man with comorbidities and with chronic myeloproliferative disorder withleukocytosis and essential thrombocytosis. He is JAK2 positive. Erythropoietin is low at 5. He has not hada bone marrow exam. He has had phlebotomy for elevated hematocrit. He was treated with Hydrea butstopped it due to fatigue. He takes aspirin. Blood counts on January 28, 2020: white blood count 12,000,hemoglobin 16.7, hematocrit 46.8 and platelets 521,000. You request Jakafi to treat him.In response to inquiry you confirmed the diagnosis as chronic myeloproliferative disease. You state therequested use is not off-label and is supported as second line therapy by NCCN. Without treatment theenrollee is at risk of thrombosis and stroke.The drug is FDA approved for intermediate or high risk myelofibrosis, polycythemia vera without adequateresponse to Hydrea and for a type of graft versus host disease. The enrollee has none of these conditions.The NCCN compendia also supports the use for myelofibrosis and polycythemia vera. It does not listchronic myeloproliferative disorder or essential thrombocytosis which was noted in the progress note ofDecember 27, 2019. DrugDex notes Jakafi has an orphan drug status for essential thrombocytosis. TheNCCN Guidelines Version 3.2019 Essential thrombocyhemia page ET-3 lists Hydrea, Interferon andAnagrelide as treatment options. You did not address options other than Hydrea.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 Jakafi 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 Jakafi.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.