Transplant recipients are vulnerable to coverage denial under Medicare Part D

Transplant immunosuppressants are often used off‐label because of insufficient randomized prospective trial data to achieve organ‐specific US Food and Drug Administration (FDA) approval. Transplant recipients who rely on Medicare Part D for immunosuppressant drug coverage are vulnerable to coverage denial for off‐label prescriptions, unless use is supported by Centers for Medicare & Medicaid Services (CMS)–approved compendia. An integrated dataset including national transplant registry data and 3 years of dispensed pharmacy records was used to identify the prevalence of immunosuppression use that is both off‐label and not supported by CMS‐approved compendia. Numbers of potentially vulnerable transplant recipients were identified. Off‐label and off‐compendia immunosuppression regimens are frequently prescribed (3‐year mean: lung 66.5%, intestine 34.2%, pancreas 33.4%, heart 21.8%, liver 16.5%, kidney 0%). The annual retail cost of these at‐risk medications exceeds $30 million. This population‐based study of transplant immunosuppressants vulnerable to claim denials under Medicare Part D coverage demonstrates a substantial gap between clinical practice, current FDA approval processes, and policy mandates for pharmaceutical coverage. This coverage barrier reduces access to life‐saving medications for patients without alternative resources and may increase the risk of graft loss and death from medication nonadherence. The Medicare Prescription Drug Benefit Manual obligates Part D plan sponsors to cover medications for Food and Drug Administration–approved indications or select off‐label indications only, leaving many nonkidney transplant recipients vulnerable to coverage denials for lifesaving immunosuppressant medications.