加拿大治疗弥漫性大 B 细胞淋巴瘤的 CAR T 细胞疗法:成本效用分析》。

IF 3.1 3区 医学 Q2 HEALTH CARE SCIENCES & SERVICES
Medical Decision Making Pub Date : 2024-04-01 Epub Date: 2024-03-14 DOI:10.1177/0272989X241234070
Lisa Masucci, Feng Tian, Stephen Tully, Zeny Feng, Tom McFarlane, Kelvin K W Chan, William W L Wong
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引用次数: 0

摘要

背景:嵌合抗原受体(CAR)T 细胞疗法是一种治疗非霍奇金淋巴瘤的新型细胞疗法。CAR T 细胞疗法的发展改变了肿瘤治疗,为治愈提供了可能。然而,由于这些疗法成本高昂,且符合条件的患者人数众多,决策者面临着艰难的筹资决策。我们的目标是利用最近 JULIET 试验的最新生存数据,评估 tisagenlecleucel 治疗加拿大复发/难治性弥漫大 B 细胞淋巴瘤成人患者的成本效益:我们建立了一个个体模拟离散事件仿真模型,以评估与挽救性化疗相比,替沙根来曲塞的成本和质量调整生命年(QALY)。生存期估计值来自已发表的临床试验和回顾性分析。如果患者持续 5 年无进展,则假定其处于长期缓解期。成本计算和效用数据来自报告和公开发表的资料。采用了加拿大医疗支付方的观点,并对终生结果进行了建模。成本和疗效的贴现率为每年 1.5%,成本以 2021 年的加元计算。我们采用了概率分析,并在单向敏感性分析和情景分析中改变了模型参数:在纳入最新的临床证据后,与挽救性化疗相比,替沙格列脲的额外成本为503417加元,额外疗效为2.48 QALYs,增量成本效益比为202991加元。在100,000美元/QALY的支付意愿阈值下,tisagenlecleucel具有成本效益的可能性为0%:结论:以目前的药物价格计算,替沙根来曲塞并不具有成本效益。这些结果在很大程度上取决于对长期生存率和CAR T价格的假设,需要真实世界的证据来减少不确定性:对于两线或更多线系统治疗失败的弥漫大 B 细胞淋巴瘤患者,在 100,000 美元的支付意愿阈值下,CAR T 并非一种具有成本效益的治疗方案。这些结果在很大程度上取决于预期的长期生存期,未来可能会利用真实世界的证据来改善模型的不确定性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
CAR T-cell Therapy for Diffuse Large B-cell Lymphoma in Canada: A Cost-Utility Analysis.

Background: Chimeric antigen receptor (CAR) T-cell therapy is a novel cell therapy for treating non-Hodgkin lymphoma. The development of CAR T-cell therapy has transformed oncology treatment by offering a potential cure. However, due to the high cost of these therapies, and the large number of eligible patients, decision makers are faced with difficult funding decisions. Our objective was to assess the cost-effectiveness of tisagenlecleucel for adults with relapsed/refractory diffuse large B-cell lymphoma in Canada using updated survival data from the recent JULIET trial.

Methods: We developed an individual-simulated discrete event simulation model to assess the costs and quality-adjusted life-years (QALY) of tisagenlecleucel compared with salvage chemotherapy. Survival estimates were obtained from a published clinical trial and retrospective analysis. If patients remained progression free for 5 y, they were assumed to be in long-term remission. Costing and utility data were obtained from reports and published sources. A Canadian health care payer perspective was used, and outcomes were modeled over a lifetime horizon. Costs and outcomes were discounted at 1.5% annually, with costs reported in 2021 Canadian dollars. A probabilistic analysis was used, and model parameters were varied in 1-way sensitivity analyses and scenario analyses.

Results: After we incorporated the latest clinical evidence, tisagenlecleucel led to an additional cost of $503,417 and additional effectiveness of 2.48 QALYs, with an incremental cost-effectiveness ratio of $202,991 compared with salvage chemotherapy. At a willingness-to-pay threshold of $100,000/QALY, tisagenlecleucel had a 0% likelihood of being cost-effective.

Conclusions: At the current drug price, tisagenlecleucel was not found to be a cost-effective option. These results heavily depend on assumptions regarding long-term survival and the price of CAR T. Real-world evidence is needed to reduce uncertainty.

Highlights: For patients with diffuse large B-cell lymphoma who failed 2 or more lines of systemic therapy, CAR T was not found to be a cost-effective treatment option at a willingness-to-pay threshold of $100,000.These results heavily depend on the expected long-term survival. The uncertainty in the model may be improved using real-world evidence reported in the future.

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来源期刊
Medical Decision Making
Medical Decision Making 医学-卫生保健
CiteScore
6.50
自引率
5.60%
发文量
146
审稿时长
6-12 weeks
期刊介绍: Medical Decision Making offers rigorous and systematic approaches to decision making that are designed to improve the health and clinical care of individuals and to assist with health care policy development. Using the fundamentals of decision analysis and theory, economic evaluation, and evidence based quality assessment, Medical Decision Making presents both theoretical and practical statistical and modeling techniques and methods from a variety of disciplines.
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