Solving the problem of financing one-time treatments with evidence uncertainty: which types of outcomes-based payment models could work best for novel CAR-T therapies in multiple myeloma? A systematic review of the published literature

Managed entry agreements (MEAs) are arrangements between a manufacturer and payer/ health care provider that grant access (coverage/reimbursement) to a health technology, subject to fulfillment of specific conditions. An increasing number of payers in different countries are implementing MEAs to enable timelier access to cancer therapies. Novel chimeric antigen receptor (CAR) T-cell therapies are one such example where MEAs can play a prominent role in accelerating patient access. CAR-T therapies are administered to patients as a single, one-time treatment. ‘Spread payment’ models can help to reduce the (front-loaded) cost impact on payers’ annual health care budgets. Furthermore, CAR-T therapies are associated with uncertainty around their clinical and economic value proposition, whilst data on their long-term safety and clinical effectiveness are pending. Such uncertainty can be ameliorated, in the most part, through outcomes based MEAs, where payments can be made in installments, according to patient outcomes that are observed in routine clinical practice. Well-validated and objective measures are however prerequisite to successful outcomes-based schemes, as is high quality data together with the ease of implementation. A systematic review of the published literature over a 5-year period (2015–2020) was performed to better understand the nature of outcomes-based MEAs employed to date, as a conditional requirement for the reimbursement of CAR-T therapies. Their applicability to, and suitability for, a future generation of CAR-T therapies in development for multiple myeloma (MM) are considered, drawing on the recent experience from regulatory approved CAR-Ts in acute lymphocytic leukemia (ALL), diffuse large B-cell lymphoma (DLBCL) and mantle cell lymphoma (MCL).

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