Abstract
Simple SummaryCAR T therapies axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) have been approved in Spain for patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL), given their favourable outcomes for overall survival and progression-free survival. However, the cost of these treatments must be weighed within the context of the Spanish health system. In this study, we assessed the cost-effectiveness and cost-utility of axi-cel vs. tisa-cel from the Spanish National Health System perspective. Using commonly applied willingness-to-pay thresholds in Spain, our analysis shows that axi-cel is highly cost-effective when compared to tisa-cel in R/R DLBCL. These results could be used to support decision-making criteria for axi-cel financing.The study aimed to assess the cost-effectiveness of axicabtagene ciloleucel (axi-cel) vs. tisagenlecleucel (tisa-cel) for the treatment of relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) after ≥2 lines of systemic therapy in Spain. A lifetime partitioned survival mixture cure model, which comprises pre-progression, post-progression, and death health states, was used to estimate the accumulated costs and outcomes in terms of life years gained (LYG) and quality-adjusted life years (QALY). A matching-adjusted indirect comparison was used to reweight patient-level data from ZUMA-1, the pivotal clinical trial for axi-cel, to aggregate-level data from the pivotal tisa-cel trial, JULIET. The analysis was performed from the National Health System perspective, thus only direct costs were included. Sensitivity analyses (SA) were performed. Axi-cel yielded 2.74 incremental LYG and 2.31 additional QALY gained per patient compared to tisa-cel. Total incremental lifetime costs for axi-cel versus tisa-cel were €30,135/patient. The incremental cost-effectiveness ratio of axi-cel versus tisa-cel resulted in €10,999/LYG and the incremental cost-utility ratio in €13,049/QALY gained. SA proved robustness of the results. Considering the frequently assumed willingness-to-pay thresholds in Spain (€22,000/QALY and €60,000/QALY), axi-cel is a cost-effective treatment vs. tisa-cel for adult patients with R/R DLBCL in Spain.
Highlights
Non-Hodgkin lymphoma (NHL) comprises a heterogenous group of lymphoproliferative malignancies originating primarily in B lymphocytes [1]
Costs were somewhat higher for axi-cel compared with tisa-cel, the pre-progression disease management costs, due to a longer progression-free survival (PFS) with axi-cel, as well as the drug acquisition costs and stem cell transplant (SCT) costs
When comparing axi-cel versus tisa-cel, the analysis showed an increment of 2.31 quality-adjusted life years (QALY) per patient and incremental costs of €30,135 per patient, which leads to an incremental cost-utility ratio (ICUR) of €13,049/QALY gained
Summary
Non-Hodgkin lymphoma (NHL) comprises a heterogenous group of lymphoproliferative malignancies originating primarily in B lymphocytes [1]. Aggressive subtypes of B-cell NHL include diffuse large B-cell lymphoma (DLBCL) and primary mediastinal. Despite the improvements in overall survival (OS) with the addition of rituximab, approximately 30% of patients with DLBCL will develop relapsed/refractory (R/R) disease after first line treatment, which remains a major cause of morbidity and mortality [2,4]. Patients with R/R DLBCL are frequently ineligible for stem cell transplant (SCT) and have poor OS, with an expected median OS of approximately 6 months with a 2-year OS of 20% [5]. The treatment landscape has experienced a milestone with the development of chimeric antigen receptor (CAR) T-cell therapies, which have demonstrated remarkable improvements in OS, and the possibility of achieving above 50%
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