Appeal type: Prescription Drug
Decision: Unfavorable

You asked the Plan to cover Tafinlar. 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 Tafinlar. The drug is requested to treat Erdheim-Chester disease, which is an off-label (non-FDA approved) use.The enrollee is a 76-year-old man who has comorbidities and Erdheim-Chester disease affecting his lungs, bones and lymph nodes. He has shortness of breath requiring oxygen. Next generation sequencing identified a BRAF V660E mutation. He is being treated with pulse steroids pending review of the appeal for Tafinlar. The prescriber submitted medical citations in support of the request. These included a non-CMS approved journal and a retrospective study and an expert review from Blood. There were no articles meeting the CMS rules for use to determine medically accepted use. This is a rare disease. In response to inquiry, the prescriber stated options include Cladribine, Interferon and Vemurafenib. The prescriber states Caldribine and Interferon will be too toxic and myelosuppressive for the enrollee. He states clinical trials have shown Dabrafenib (Tafinlar) is better tolerated than Vemurafenib. He cites an expert in the disease who is the author of the Blood review. He states if unable to start Tafinlar, he will start Cladribine.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 Tafinlar 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 Tafinlar.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.