Herring William, Pearson Isobel, Purser Molly, Nakhaipour Hamid Reza, Haiderali Amin, Wolowacz Sorrel, Jayasundara Kavisha
RTI Health Solutions, 200 Park Offices Drive, Research Triangle Park, Durham, NC, 27709, USA.
RTI Health Solutions, Didsbury, Manchester, UK.
Pharmacoeconomics. 2016 Jan;34(1):77-90. doi: 10.1007/s40273-015-0332-5.
Our objective was to estimate the cost effectiveness of ofatumumab plus chlorambucil (OChl) versus chlorambucil in patients with chronic lymphocytic leukaemia for whom fludarabine-based therapies are considered inappropriate from the perspective of the publicly funded healthcare system in Canada.
A semi-Markov model (3-month cycle length) used survival curves to govern progression-free survival (PFS) and overall survival (OS). Efficacy and safety data and health-state utility values were estimated from the COMPLEMENT-1 trial. Post-progression treatment patterns were based on clinical guidelines, Canadian treatment practices and published literature. Total and incremental expected lifetime costs (in Canadian dollars [$Can], year 2013 values), life-years and quality-adjusted life-years (QALYs) were computed. Uncertainty was assessed via deterministic and probabilistic sensitivity analyses.
The discounted lifetime health and economic outcomes estimated by the model showed that, compared with chlorambucil, first-line treatment with OChl led to an increase in QALYs (0.41) and total costs ($Can27,866) and to an incremental cost-effectiveness ratio (ICER) of $Can68,647 per QALY gained. In deterministic sensitivity analyses, the ICER was most sensitive to the modelling time horizon and to the extrapolation of OS treatment effects beyond the trial duration. In probabilistic sensitivity analysis, the probability of cost effectiveness at a willingness-to-pay threshold of $Can100,000 per QALY gained was 59 %.
Base-case results indicated that improved overall response and PFS for OChl compared with chlorambucil translated to improved quality-adjusted life expectancy. Sensitivity analysis suggested that OChl is likely to be cost effective subject to uncertainty associated with the presence of any long-term OS benefit and the model time horizon.
我们的目的是从加拿大公共资助医疗体系的角度，评估奥法木单抗联合苯丁酸氮芥（OChl）与苯丁酸氮芥相比，在被认为不适合接受基于氟达拉滨治疗的慢性淋巴细胞白血病患者中的成本效益。
采用半马尔可夫模型（周期长度为3个月），利用生存曲线来控制无进展生存期（PFS）和总生存期（OS）。疗效和安全性数据以及健康状态效用值来自COMPLEMENT-1试验。进展后治疗模式基于临床指南、加拿大治疗实践和已发表文献。计算了总预期寿命成本和增量预期寿命成本（以加元计，2013年价值）、生命年和质量调整生命年（QALYs）。通过确定性和概率性敏感性分析评估不确定性。
模型估计的贴现终身健康和经济结果表明，与苯丁酸氮芥相比，OChl一线治疗导致QALYs增加（0.41）和总成本增加（27,866加元），每获得一个QALY的增量成本效益比（ICER）为68,647加元。在确定性敏感性分析中，ICER对建模时间范围和试验持续时间之外的OS治疗效果外推最为敏感。在概率性敏感性分析中，每获得一个QALY支付意愿阈值为100,000加元时成本效益的概率为59%。
基础病例结果表明，与苯丁酸氮芥相比，OChl改善的总体缓解率和PFS转化为质量调整预期寿命的改善。敏感性分析表明，考虑到与任何长期OS益处的存在和模型时间范围相关的不确定性，OChl可能具有成本效益。
