OBJECTIVES: Healthcare costs analyses from CAR-T cell therapy are highly relevant to determine whether and how to offer patients these highly personalized immunotherapy. This study describes CAR-T cell therapy effectiveness, safety and costs in the real-life setting of a Portuguese Comprehensive Cancer Center.

METHODS: Retrospective descriptive study of patients with refractory or relapsed aggressive diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL) referred to CAR-T cell therapy (May/19-February/21). Data was obtained from clinical/administrative records. Rates of response to treatment, toxicity and survival were analyzed by the intention-to-treat. Demographic and clinical characteristics were evaluated using descriptive statistics. Survival was calculated using Kaplan-Meier. Direct medical costs, overall and stratified by inpatient-care, outpatient-care, and diagnostic-therapeutic procedures (DTP), were derived based on resources used by application of unit costs (2017 Price Tables of the Portuguese National Health Service, hospital costing system and Portuguese medicines agency).

RESULTS: Twenty patients were included (median age 49.5y; 55%male; 70%DLBCL; 50% with primary refractory disease history). Best overall and complete response rates were 81.3% and 56.3%, respectively. Median overall (OS) and progression-free survivals were 9.2 and 7.3 months; 12-month OS rate was 42.6% (95%CI:23.2-78.3). In 18 patients infused with CAR-T cells (65%Axicabtagene-ciloleucel, 35%Tisagenlecleucel; 2 patients not infused due to disease progression), 12-month OS rate was 49.0% (95%CI:27.7-86.6). Grade≥3cytokine release syndrome and neurotoxicity occurred in 5.6% and 11.1% of patients, respectively. Median total cost was 355.165€/patient; CAR-T cell drug expenses accounted for 97% of the overall cost. Excluding CAR T-cell acquisition cost (344.498€), inpatient-care, DTP and outpatient-care contributed to 57%, 38% and 5% of the overall cost/patient, respectively. CAR-T cells total cost, including adverse events management expenses, was 7.176.196€, or 286.238€ when excluding its acquisition cost.

CONCLUSIONS: Our findings highlight the heavy economic burden of CAR-T cell therapy driven by drug acquisition costs.