Loupas MA, Theodoratou D, Kourlaba G, Value in Health, 2022. Volume 25, Issue 12, S112 https://doi.org/10.1016/j.jval.2022.09.545

Objectives: Patients with relapsed/refractory mantle cell lymphoma (r/r MCL) post-bruton tyrosine kinase inhibitors (BTKi) failure currently have no recognized clinical standard of care (SoC). Brexucabtagene autoleucel (brexu-cel) is a novel cell therapy (CAR-T) with promising outcomes. We aimed to evaluate its cost-effectiveness versus best alternative treatment (BAT) currently available for patients in Greece.

Methods: A three-state partitioned survival model was used for projecting lifetime costs and outcomes for patients with r/r MCL post-BTKi, from a public payer perspective. Patients entered the model in the pre-progression health state and could transition to progression and/or death. Clinical data were derived from ZUMA-2 (Wang et al, NEJM 2020). Survival data for BAT were sourced from a literature-based meta-analysis. National data on general population mortality were applied. Costs (€, 2022) were derived from publicly available national sources and published literature. Analyses were based on list prices (ex-factory) and annual discount rate of 3.5% was applied for both cost and health outcomes.

Results: Βrexu-cel was associated with both significantly higher median survival (7.48 vs 1.61 years with BAT) and quality-adjusted life years (QALYs) gained (5.42 vs. 1.19 on BAT) at an additional cost of €368,851. The incremental cost-effectiveness ratio (ICER) was estimated at €62,763 and €87,281 per LY and QALY gained respectively. When applying the respective pricing provisions for supply of medicinal products in public hospitals, this is further reduced to €54.291 and €75.499 per LY and QALY gained respectively. At a willingness to pay threshold of €100,000/QALY gained, as indicated for life-threatening diseases, the probability for brexu-cel to be cost-effective was 99%.

Conclusions: Considering the lack of established clinical SoC and the recognized insufficient outcomes for patients with r/r MCL post-BTKi failure, brexu-cel provides a valuable and potentially cost-effective alternative to current BAT, deriving its value from incremental survival and health-related quality-of-life benefits.