The Health Impacts of Better Access to Axicabtagene Ciloleucel: The Case of Spain.

Simple Summary: Axicabtagene ciloleucel (axi-cel) has been shown to improve the health outcomes of patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL); however, the actual number of patients treated in Spain is lower than the epidemiology estimations. The aim of our study was to assess the value of axi-cel versus chemotherapy in patients with R/R DLBCL after ≥2 lines of therapy based on the number of patients treated. Considering that the entire cohort was eligible for treatment with axi-cel (n = 490) compared to the currently treated population (n = 187), the use of axi-cel rather than chemotherapy in all eligible patients could lead to 2173 life years gained and 1706 quality-adjusted life years. Furthermore, if all eligible patients were treated with CAR T-cell therapy, an additional 85 patients would be alive, and 78 patients would be alive without disease progression at 5 years. In this study, the health impacts of improving access to treatment with axicabtagene ciloleucel (axi-cel) was assessed in patients with relapsed/refractory diffuse large B-cell lymphoma after ≥2 lines of therapy in Spain. A partitioned survival mixture cure model was used to estimate the lifetime accumulated life years gained (LYG) and quality-adjusted life years (QALYs) per patient treated with axi-cel versus chemotherapy. Efficacy data were extracted from the ZUMA-1 trial for axi-cel and from the SCHOLAR-1 study for chemotherapy. In the base case, the incremental outcomes of axi-cel versus chemotherapy were evaluated in a cohort of 187 patients treated with CAR T-cell therapies, as reported by the "Spanish National Health System Plan for Advanced Therapies", and in the alternative scenario in the full eligible population based on epidemiological estimates (n = 490). Taking those currently treated with axi-cel, compared with chemotherapy, axi-cel provided an additional 1341 LYGs and 1053 QALYs. However, when all eligible patients (n = 490) were treated, axi-cel provided an additional 3515 LYs and 2759 QALYs. Therefore, if all eligible patients were treated with axi-cel rather than those currently treated as per the registry (n = 187), there would have been an additional 303 patients treated, resulting in an additional 2173 LYGs and 1706 QALYs in total. The lack of access in Spain has led to a loss of a substantial number of LYGs and QALYs, and efforts should be made to improve access for all eligible patients. [ABSTRACT FROM AUTHOR]