Mandrik Olena, Corro Ramos Isaac, Knies Saskia, Al Maiwenn, Severens Johan L
Institute of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, the Netherlands.
Institute of Medical Technology Assessment (iMTA), Erasmus University Rotterdam, Rotterdam, the Netherlands.
Cancer Manag Res. 2015 Aug 25;7:279-89. doi: 10.2147/CMAR.S79258. eCollection 2015.
The aim of this study was to assess the cost-effectiveness, from a health care perspective, of adding rituximab to fludarabine and cyclophosphamide scheme (FCR versus FC) for treatment-naïve and refractory/relapsed Ukrainian patients with chronic lymphocytic leukemia. A decision-analytic Markov cohort model with three health states and 1-month cycle time was developed and run within a life time horizon. Data from two multinational, prospective, open-label Phase 3 studies were used to assess patients' survival. While utilities were generalized from UK data, local resource utilization and disease-associated treatment, hospitalization, and side effect costs were applied. The alternative scenario was performed to assess the impact of lower life expectancy of the general population in Ukraine on the incremental cost-effectiveness ratio (ICER) for treatment-naïve patients. One-way, two-way, and probabilistic sensitivity analyses were conducted to assess the robustness of the results. The ICER (in US dollars) of treating chronic lymphocytic leukemia patients with FCR versus FC is US$8,704 per quality-adjusted life year gained for treatment-naïve patients and US$11,056 for refractory/relapsed patients. When survival data were modified to the lower life expectancy of the general population in Ukraine, the ICER for treatment-naïve patients was higher than US$13,000. This value is higher than three times the current gross domestic product per capita in Ukraine. Sensitivity analyses have shown a high impact of rituximab costs and a moderate impact of differences in utilities on the ICER. Furthermore, probabilistic sensitivity analyses have shown that for refractory/relapsed patients the probability of FCR being cost-effective is higher than for treatment-naïve patients and is close to one if the threshold is higher than US$15,000. State coverage of rituximab treatment may be considered a cost-effective treatment for the Ukrainian population under conditions of economic stability, cost-effectiveness threshold growth, or rituximab price negotiations.
本研究的目的是从医疗保健角度评估，对于初治及难治性/复发性乌克兰慢性淋巴细胞白血病患者，在氟达拉滨和环磷酰胺方案（FCR对比FC）中添加利妥昔单抗的成本效益。构建了一个具有三种健康状态、周期为1个月的决策分析马尔可夫队列模型，并在终身范围内运行。使用两项跨国、前瞻性、开放标签3期研究的数据来评估患者的生存率。效用值从英国数据中进行了归纳，同时应用了当地的资源利用以及与疾病相关的治疗、住院和副作用成本。进行了替代情景分析，以评估乌克兰普通人群较低的预期寿命对初治患者增量成本效益比（ICER）的影响。进行了单向、双向和概率敏感性分析，以评估结果的稳健性。对于初治慢性淋巴细胞白血病患者，FCR对比FC治疗的ICER（以美元计）为每获得一个质量调整生命年8704美元，对于难治性/复发性患者为11056美元。当将生存数据调整为乌克兰普通人群较低的预期寿命时，初治患者的ICER高于13000美元。该值高于乌克兰当前人均国内生产总值的三倍。敏感性分析表明，利妥昔单抗成本对ICER影响较大，效用差异对ICER影响中等。此外，概率敏感性分析表明，对于难治性/复发性患者，FCR具有成本效益的概率高于初治患者，如果阈值高于15000美元，则该概率接近1。在经济稳定、成本效益阈值增长或利妥昔单抗价格谈判的情况下，利妥昔单抗治疗的国家覆盖范围可被视为乌克兰人群具有成本效益的治疗方法。
