Appeal type: Prescription Drug
Decision: Unfavorable

You asked the Plan to cover Brukinsa. 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 Brukinsa 80 mg; 120/30. The drug is requested to treat Waldenstrom's macroglobulinemia, which is an off-label (non-FDA approved) use. The enrollee is an 88 year old man with Waldenstrom's macroglobulinemia (WM). He has been treated with Imbruvica and Rituxan. He stopped Imbruvica due to diarrhea. The prescriber requests Brukinsa to treat him. Journal references were submitted in support of the request. This reviewer reviewed them. One was an abstract which cannot be used to determine medically accepted use per Medicare rules. The other was published in a CMS approved journal: Blood (2020) 136 (18): 2038-2050. The reported study found a not statistically significant higher rate of complete and very good partial response. The phase 2 study subjects had MYD88-L265P disease. Patients with wild type or unknown status were not treated on the randomized arm. Another study: Trotman, et al, Blood (2020) 136 (18): 2027-2037 published the results of the non-randomized cohort. In their conclusion they state a phase 3 study is underway. In the opinion of this reviewer, these studies do not adequately support that the drug meets the Medicare criteria for determining medically accepted use. The requested use remains 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. 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 Brukinsa 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 Brukinsa.  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.