Cost-utility analysis of second-line axicabtagene ciloleucel versus standard of care in Japan based on the ZUMA-7 trial

<b>Aim:</b> To perform a cost–effectiveness analysis comparing axicabtagene ciloleucel (axi-cel) with standard of care (SoC; salvage chemoimmunotherapy, followed by high-dose therapy with autologous stem cell rescue for responders) for second-line (2L) treatment of adults with relapsed or refractory large B-cell lymphoma (r/r LBCL) in the pivotal ZUMA-7 trial data from a Japanese payer perspective. <b>Materials &amp; methods:</b> A three-state partitioned survival model was utilized using population and clinical inputs from the ZUMA-7 trial data over a lifetime horizon. <b>Results:</b> Axi-cel was associated with greater incremental quality-adjusted life-years (2.06) and higher incremental total costs ($48,685.59/¥6.9 million) leading to an incremental cost–effectiveness ratio of $23,590.34/¥3.3 million per quality-adjusted life-years compared with SoC. <b>Conclusion:</b> Axi-cel is a cost-effective treatment alternative to SoC for 2L treatment of adults with r/r LBCL. The current standard of care (SoC) for the treatment of relapsed or refractory large B-cell lymphoma (r/r LBCL) is associated with substantial economic burden due to associated poor outcomes and relatively longer hospital stays in the pre-CAR T era. Current SoC therapies alone provide limited coverage to the rapidly aging Japanese population as advanced age precludes eligibility for transplant. We conducted a cost–effectiveness analysis of second-line (2L) axicabtagene ciloleucel (axi-cel) versus current SoC for the treatment of adult patients with r/r LBCL from a Japanese payer perspective. Our base case analysis showed that axi-cel treatment resulted in higher incremental effectiveness (2.06 quality-adjusted life-years [QALYs]) compared with SoC. Substantial cost offsets (-$152,700.14/-¥1083 million)were observed with 2L axi-cel treatment for subsequent therapy and disease management leading to a modestly higher total cost ($50,252.99/¥6.9 million) and thus resulting in an incremental cost–effectiveness ratio (ICER) of $23,590.34 (¥3.3 million) per QALY gained, within the accepted willingness-to-pay (WTP) threshold ($53,191.49 [¥7.5 million] per QALY) from the Japanese payer perspective. Therefore, treatment with axi-cel may be considered cost-effective compared with SoC. The base case ICER was most sensitive to the overall survival extrapolations used for SoC and axi-cel, acquisition cost of axi-cel and tisagenlecleucel, and proportion of patients in the SoC arm receiving third-line axi-cel. Axi-cel remained cost-effective compared with SoC at a drug acquisition cost lower than $283,687.94 (¥40.0 million) considering the WTP threshold of $53,191.49 (¥7.5 million) per QALY.