Appeal type: Prescription Drug
Decision: Unfavorable

You asked the Plan to cover Rydapt. 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 Rydapt 25 mg. The drug is requested to treat systemic mastocytosis of unstated type, which is an off-label (non-FDA approved) use. The enrollee is a 67 year old woman with systemic mastocytosis (SM). It is c-Kit negative. She has been treated with Gleevec, Hydrea, Cromolyn and has been on Midostaurin (Rydapt) since June, 2017. The prescriber requests Rydapt to continue to treat her. In response to inquiry the prescriber did not adequately address the subtype of systemic mastocytosis. A bone marrow report was submitted indicating that at the time of the marrow in 2015, her white blood count was 4,690, hemoglobin 11.9 and platelets 346,000. The marrow had multifocal dense infiltrates consisting of >15 mast cells in aggregates (major criterion.) Occasional mast cells show spindle-shape morphology. Flow cytometric analysis performed revealed aberrant co-expression of CD2 and CD25 by these atypical mast cells (minor criterion.). A page from the NCCN Guidelines Version 2.2019 Systemic Mastocytosis was submitted. The page notes the drug is indicated for aggressive SM. This reviewer found that on pages SM-C and D of the same guideline the criteria for the diagnosis of aggressive SM and mast cell leukemia are given. The documentation submitted does not include information to determine whether the enrollee meets the criteria for aggressive SM, SM associated with hematologic neoplasm or mast cell leukemia.The FDA approval and the other CMS approved compendia support the use of Rydapt for aggressive SM, SM with associated hematological neoplasm, or mast cell leukemia. The submitted documents do not support these diagnoses. Therefore the requested use does not meet medically accepted use.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 Rydapt 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 Rydapt. 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.Please note: the request for a tiering exception (lower copayment) for Rydapt was not reviewed. We cannot consider a tiering exception for a drug for which the Part D Plan is unable to provide coverage.