OBJECTIVES: This study aims to identify and characterize the cost-effectiveness evaluations of CAR-T cell therapies for reimbursement within various health systems globally.

METHODS: We obtained cost-effectiveness assessments for the incorporation of CAR-T therapies from documents of agencies in Australia (MSAC), Canada (CADTH), France (HAS), Scotland (SMC), and the United Kingdom (NICE), with searches conducted up to July 2023.

RESULTS: The study reviewed twenty-nine evaluations for five CAR-T therapies: axicabtagene ciloleucel (MSAC, CADTH, HAS, SMC, NICE), brexucabtagene autoleucel (MSAC, HAS, CADTH, SMC, NICE), ciltacabtagene autoleucel (MSAC), idecabtagene vicleucel (CADTH), and tisagenlecleucel (MSAC, CADTH, HAS, SMC, NICE). These therapies were evaluated for eight types of lymphomas and leukemias, predominantly diffuse large B-cell lymphoma and acute lymphoblastic leukemia. Comparators included standard care, rescue chemotherapy, allogeneic hematopoietic stem cell transplantation, and other treatments, such as tyrosine kinase inhibitors. A favorable decision for reimbursement was reached in 79% of the evaluations. The incremental cost-effectiveness ratio (ICER) results displayed significant variation among the agencies. CADTH reported CAR-T therapy ICERs ranging from CAD 53,269/QALY to CAD 679,053/QALY. HAS’s evaluations showed a range between EUR 111,649/QALY and EUR 295,406/QALY. SMC’s findings varied from GBP 44,151/QALY to GBP 49,711/QALY, whereas NICE indicated a most likely ICER range from below GBP 30,000/QALY to above GBP 50,000/QALY, with some specific evaluations demonstrating a wider range. MSAC maintained confidentiality regarding their ICER results.

CONCLUSIONS: This study highlights the increasing global attention on CAR-T cell therapies. Despite the high ICER associated with these therapies, the majority of the evaluations led to favorable reimbursement decisions. Nonetheless, the substantial costs linked to CAR-T therapies call for discussions on innovative financing methods for their incorporation into health systems, emphasizing the need to balance efficacy, cost, and long-term benefits in healthcare decision-making.