基于 I/II 期临床试验的阿卡替尼治疗复发慢性淋巴细胞白血病的早期经济性评价。
Phase I/II Clinical Trial-Based Early Economic Evaluation of Acalabrutinib for Relapsed Chronic Lymphocytic Leukaemia.
机构信息
Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Universiteitsweg 99, 3584 CG, Utrecht, The Netherlands.
The National Health Care Institute (ZIN), Diemen, The Netherlands.
出版信息
Appl Health Econ Health Policy. 2019 Dec;17(6):883-893. doi: 10.1007/s40258-019-00496-1.
OBJECTIVES
The objective of this study was to construct an early economic evaluation for acalabrutinib for relapsed chronic lymphocytic leukaemia (CLL) to assist early reimbursement decision making. Scenarios were assessed to find the relative impact of critical parameters on incremental costs and quality-adjusted life-years (QALYs).
METHODS
A partitioned survival model was constructed comparing acalabrutinib and ibrutinib from a UK national health service perspective. This model included states for progression-free survival (PFS), post-progression survival (PPS) and death. PFS and overall survival (OS) were parametrically extrapolated from ibrutinib publications and a preliminary hazard ratio based on phase I/II data was applied for acalabrutinib. Deterministic and probabilistic sensitivity analyses were performed, and 1296 scenarios were assessed.
RESULTS
The base-case incremental cost-effectiveness ratio (ICER) was £61,941/QALY, with 3.44 incremental QALYs and incremental costs of £213,339. Deterministic sensitivity analysis indicated that survival estimates, utilities and treatment costs of ibrutinib and acalabrutinib and resource use during PFS have the greatest influence on the ICER. Probabilistic results under different development scenarios indicated that greater efficacy of acalabrutinib would decrease the likelihood of cost effectiveness (from 63% at no effect to 2% at maximum efficacy). Scenario analyses showed that a reduction in PFS did not lead to great QALY differences (- 8 to - 14% incremental QALYs) although it did greatly affect costs (- 47 to - 122% incremental pounds). For OS, the opposite was true (- 89 to - 93% QALYs and - 7 to - 39% pounds).
CONCLUSIONS
Acalabrutinib is not likely to be cost effective compared with ibrutinib under current development scenarios. The conflicting effects of OS, PFS, drug costs and utility during PFS show that determining the cost effectiveness of acalabrutinib without insight into all parameters complicates health technology assessment decision making. Early assessment of the cost effectiveness of new products can support development choices and reimbursement processes through effective early dialogues between stakeholders.
目的
本研究旨在构建阿卡鲁替尼治疗复发慢性淋巴细胞白血病（CLL）的早期经济评价，以协助早期报销决策。评估了各种方案，以确定关键参数对增量成本和质量调整生命年（QALY）的相对影响。
方法
从英国国家医疗服务体系的角度，构建了阿卡鲁替尼与伊布替尼的分割生存模型。该模型包括无进展生存期（PFS）、进展后生存期（PPS）和死亡状态。基于伊布替尼的出版物，对 PFS 和总生存期（OS）进行了参数外推，同时应用了基于 I/II 期数据的初步风险比来预测阿卡鲁替尼。进行了确定性和概率敏感性分析，并评估了 1296 种方案。
结果
基础病例增量成本效益比（ICER）为 61941 英镑/QALY，增量 QALY 为 3.44，增量成本为 213339 英镑。确定性敏感性分析表明，伊布替尼和阿卡鲁替尼的生存估计、效用和治疗成本以及 PFS 期间的资源利用对 ICER 影响最大。不同开发方案下的概率结果表明，阿卡鲁替尼疗效的提高将降低成本效益的可能性（从无效果的 63%到最大效果的 2%）。方案分析表明，PFS 的缩短不会导致巨大的 QALY 差异（增量 QALY 减少 8%至 14%），尽管它确实会极大地影响成本（增量英镑减少 47%至 122%）。对于 OS，则相反（增量 QALY 减少 89%至 93%，增量英镑减少 7%至 39%）。
结论
在当前的开发方案下，阿卡鲁替尼与伊布替尼相比不太可能具有成本效益。OS、PFS、药物成本和 PFS 期间的效用的相互冲突的影响表明，如果没有对所有参数的深入了解，确定阿卡鲁替尼的成本效益会使卫生技术评估决策复杂化。对新产品的成本效益进行早期评估，可以通过利益相关者之间的有效早期对话，支持新产品的开发选择和报销流程。
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