OBJECTIVES: Axicabtagene ciloleucel (axi-cel) demonstrated superior overall survival versus standard of care (SOC; salvage chemoimmunotherapy followed by high-dose chemotherapy with autologous stem-cell transplantation for responders) in the treatment of relapsed or refractory large B-cell lymphoma (2L LBCL) in adults in the ZUMA-7 trial (NCT03391466). This study aimed to estimate the cost-effectiveness of axi-cel versus SOC in 2L LBCL from the German statutory health insurance perspective.

METHODS: Using a partitioned survival model, we estimated costs, life-years (LYs), and quality-adjusted life-years (QALYs) over a lifetime time horizon. The model was populated with efficacy, safety, and utility inputs based on patient level data from ZUMA-7. Time-to-event data were extrapolated beyond the trial period using mixture cure models validated by clinical experts. Medical resource use data and 2023-year unit costs in German Euros (€) were obtained from the literature and German price databases. Outcomes and costs were discounted at 3% per annum. Deterministic and probabilistic sensitivity analyses were conducted to assess robustness of the results.

RESULTS: 2L axi-cel treatment resulted in LY and QALY gains of 1.74 and 1.64, respectively, and incremental costs of €85,255 compared to SOC, resulting in an incremental cost-effectiveness ratio (ICER) of €51,830 per QALY. Results were driven by improved survival and quality of life associated with axi-cel, despite substantial utilization of chimeric antigen receptor-T cell therapy in third line in the SOC arm. Model results were robust in sensitivity analyses. Key drivers included the standardized mortality ratio applied to the general population mortality for long-term axi-cel survivors and the mean age of the patients considered in the model.

CONCLUSIONS: Axi-cel treatment results in meaningful increases in LYs and QALYs compared to SOC in adult patients with 2L LBCL. Based on its ICER and commonly cited willingness-to-pay thresholds in Germany, axi-cel can be considered a cost-effective treatment for Germany.