Caillon Megane, Brethon Benoit, van Beurden-Tan Chrissy, Supiot Romain, Le Mezo Antoine, Chauny Jean-Vannak, Majer Istvan, Petit Arnaud
Amgen (France) SAS, Arcs de Seine, 18-20 Quai du Point du Jour, 92100, Boulogne-Billancourt, France.
Pediatric Hematology and Immunology Department, Robert-Debré Hospital, AP-HP, Paris, France.
Pharmacoecon Open. 2023 Jul;7(4):639-653. doi: 10.1007/s41669-023-00411-4. Epub 2023 Apr 18.
Based on the results of the phase III randomized 20120215 trial, the European Medicines Agency granted the approval of blinatumomab for the treatment of pediatric patients with high-risk first-relapsed Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL). In France, blinatumomab received reimbursement for this indication in May 2022. This analysis assessed the cost effectiveness of blinatumomab compared with high-risk consolidation chemotherapy (HC3) in this indication from a French healthcare and societal perspective.
A partitioned survival model with three health states (event-free, post-event and death) was developed to estimate life-years (LYs), quality-adjusted life-years (QALYs) and costs over a lifetime horizon. Patients who were alive after 5 years were considered to be cured. An excess mortality rate was applied to capture the late effects of cancer therapy. Utility values were based on the TOWER trial using French tariffs, and cost input data were identified from French national public health sources. The model was validated by clinical experts.
Treatment with blinatumomab over HC3 was estimated to provide gains of 8.39 LYs and 7.16 QALYs. Total healthcare costs for blinatumomab and HC3 were estimated to be €154,326 and €102,028, respectively, resulting in an increment of €52,298. The incremental cost-effectiveness ratio was estimated to be €7308 per QALY gained from a healthcare perspective. Results were robust to sensitivity analyses, including analysis from the societal perspective.
Blinatumomab administered as part of consolidation therapy in pediatric patients with high-risk first-relapsed ALL is cost effective compared with HC3 from the French healthcare and societal perspective.
基于III期随机20120215试验的结果，欧洲药品管理局批准了博纳吐单抗用于治疗高危首次复发的费城染色体阴性B细胞前体急性淋巴细胞白血病（ALL）的儿科患者。在法国，博纳吐单抗于2022年5月获得该适应症的报销批准。本分析从法国医疗保健和社会角度评估了博纳吐单抗与高危巩固化疗（HC3）在该适应症中的成本效益。
开发了一个具有三种健康状态（无事件、事件后和死亡）的分区生存模型，以估计终身的生命年（LYs）、质量调整生命年（QALYs）和成本。5年后仍存活的患者被视为治愈。应用超额死亡率来捕捉癌症治疗的晚期影响。效用值基于使用法国关税的TOWER试验，成本输入数据来自法国国家公共卫生来源。该模型由临床专家进行了验证。
估计与HC3相比，使用博纳吐单抗治疗可带来8.39个生命年和7.16个质量调整生命年的收益。博纳吐单抗和HC3的总医疗成本估计分别为154,326欧元和102,028欧元，增量为52,298欧元。从医疗保健角度估计，每获得一个质量调整生命年的增量成本效益比为7308欧元。结果对敏感性分析具有稳健性，包括从社会角度的分析。
从法国医疗保健和社会角度来看，在高危首次复发的ALL儿科患者中，作为巩固治疗一部分给予的博纳吐单抗与HC3相比具有成本效益。
