Appeal type: Prescription Drug
Decision: Unfavorable

You asked the Plan to cover Afinitor. 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 Afinitor (Everolimus) 10 mg, 30 per 30 days. The drug is requested to treat extrahepatic cholangiocarcinoma, which is an off-label (non-FDA approved) use.The enrollee is a 73 year old woman with extrahepatic cholangiocarcinoma involving soft tissue in the porta hepatis, peritoneum and celiac trunk region. She has been treated with surgery, radiation, Xeloda, Cisplatin, Gemcitabine, 5FU, Leucovorin, and Oxaliplatin. CT scanning on February 27, 2020 notes recurrent disease is of concern due to new ascites and complete thrombosis of the portal vein. FoundationOne CDx identified microsatellite stable and PIK3CA mutation. The latter suggests possible response to Everolimus. The molecular tumor board recommended off-label Everolimus or Alpelisib. You request Everolimus to treat her. A journal article from a CMS approved journal was submitted and reviewed. (Buzzoni, et al, Activity and Safety of RAD001 (everolimus) in patients affected by biliary tract cancer progressing after prior chemotherapy: a phase II ITMO study, Annals of Oncology 2014; 25: 1597-1603.) The authors conclude that larger trials are needed to better establish the role of Everolimus in the treatment of BTC and identify prognostic biomarkers predictive of response to targeted therapies. Therefore the requested use is investigational.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. 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.Our Physician Reviewer reviewed this file and determined that there are no citations in the Medicare approved compendia that support the use of Afinitor 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 Afinitor. 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.