Analysis Group, Inc., Boston, MA, USA.
Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
J Med Econ. 2020 Sep;23(9):1016-1024. doi: 10.1080/13696998.2020.1769109. Epub 2020 Jun 2.
This study estimated the total costs associated with tisagenlecleucel treatment in adult patients with relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL) based on the JULIET trial from a United States hospital's perspective. An economic model was developed to assess the total costs associated with tisagenlecleucel treatment (from leukapheresis to two months post-infusion) in adults (aged ≥18 years) with r/r DLBCL using a fee-for-service approach. Costs were considered during the pre-treatment, tisagenlecleucel infusion, and follow-up periods, and were estimated based on the health resource utilization and safety data from the JULIET trial. Cost components included leukapheresis, lymphodepleting chemotherapy, tisagenlecleucel infusion/administration, inpatient and intensive care unit (ICU) admission, medical professional visits, lab tests/procedures, and management of adverse events (AEs). The base-case model estimated the total costs using observed hospitalization, ICU, and AE data from JULIET, while scenario analyses varied key assumptions related to AEs and hospitalization. The estimated overall cost associated with tisagenlecleucel treatment from leukapheresis to two months post-infusion was $437,927/patient, of which $64,784 (14.8%) was additional to tisagenlecleucel's list price ($373,000) and the associated administration cost ($143). The top three key drivers of the additional cost were AE management ($30,594; 47.2%), inpatient/ICU not attributed to AEs ($24,285; 37.5%), and lab tests/procedures ($5,443; 8.4%). In the scenario analyses, total costs ranged from $382,702 (no AEs, no hospitalization) to $469,006 (cytokine release syndrome and B-cell aplasia, hospitalization).: This analysis was limited to two months of follow-up after tisagenlecleucel infusion, which cannot capture long-term safety outcomes associated with the treatment and may underestimate AE costs. The total cost of tisagenlecleucel administration from leukapheresis to two months was estimated at $437,927. In addition to tisagenlecleucel's price, the main drivers were AE management costs and inpatient/ICU costs. Future studies based on real-world, long-term use of tisagenlecleucel are warranted.
本研究从美国医院的角度基于 JULIET 试验估算了靶向 CD19 的嵌合抗原受体 T 细胞（tisagenlecleucel）治疗成人复发/难治性弥漫性大 B 细胞淋巴瘤（r/r DLBCL）的总费用。采用按服务项目付费的方法，建立了一个经济模型以评估 r/r DLBCL 成人患者（≥18 岁）接受 tisagenlecleucel 治疗（自白细胞分离术至输注后两个月）的总费用。在治疗前、tisagenlecleucel 输注期间和随访期间考虑了成本，并根据 JULIET 试验的卫生资源利用和安全性数据进行了估算。成本构成包括白细胞分离术、淋巴清除化疗、tisagenlecleucel 输注/管理、住院和重症监护病房（ICU）入院、医疗专业人员就诊、实验室检查/程序和不良事件（AE）管理。基础模型使用 JULIET 试验中观察到的住院、ICU 和 AE 数据估算了总费用，而情景分析则对与 AE 和住院相关的关键假设进行了调整。从白细胞分离术至输注后两个月，tisagenlecleucel 治疗的总费用估计为每位患者 437927 美元，其中 64784 美元（14.8%）是除 tisagenlecleucel 标价（373000 美元）和相关管理费用（143 美元）之外的额外费用。额外费用的三个主要驱动因素是 AE 管理（30594 美元；47.2%）、与 AE 无关的住院/ICU（24285 美元；37.5%）和实验室检查/程序（5443 美元；8.4%）。在情景分析中，总费用范围为 382702 美元（无 AE，无住院）至 469006 美元（细胞因子释放综合征和 B 细胞再生障碍，住院）。本分析仅限于 tisagenlecleucel 输注后两个月的随访，无法捕获与治疗相关的长期安全性结果，可能会低估 AE 成本。从白细胞分离术到输注后两个月，tisagenlecleucel 的总管理费用估计为 437927 美元。除了 tisagenlecleucel 的价格，主要驱动因素是 AE 管理成本和住院/ICU 成本。需要开展基于 tisagenlecleucel 的真实世界、长期使用的进一步研究。
