Tisagenlecleucel for relapsed/refractory acute lymphoblastic leukemia in the Irish healthcare setting: cost-effectiveness and value of information analysis
Tisagenlecleucel for relapsed/refractory acute lymphoblastic leukemia in the Irish healthcare setting: cost-effectiveness and value of information analysis
复制标题
DOI：
10.1017/s0266462322000356
复制
发表时间：
2022-07-11
影响因子：
3.2
通讯作者：
Barry, Michael
中科院分区：
文献类型：
--
作者：
Carey, Niamh;Leahy, Joy;Barry, Michael
目的：本研究评估了 tisagenlecleucel（一种 CAR T 细胞疗法）与 blinatumomab 相比，在爱尔兰医疗机构中治疗复发/难治性急性淋巴细胞白血病 (R/R ALL) 儿童和青年患者的成本效益。通过完美信息期望值 (EVPI) 和部分 EVPI (EVPPI) 分析来评估进行进一步研究的价值，以调查与决策问题相关的不确定性的价值。方法：开发了一个三状态分区生存模型。短期决策树根据输注状态将患者分为 tisagenlecleucel 组。生存期推断为 60 个月；然后应用具有标准化死亡率的总人口死亡率。根据决策发生率将估计的 EVPI 和 EVPPI 扩大到人群。 结果：按标价计算，每个质量调整生命年 (QALY) 增量成本效益比为 73,086 欧元（增量成本 156,928 欧元；增量 QALY 2.15）。在每 QALY 45,000 欧元的支付意愿门槛下，成本效益的概率为 16%。在此阈值下，人口 EVPI 为 314,455 欧元；每个参数类别的人群 EVPPI 均低于 100,000 欧元。结论：与 blinatumomab 相比，Tisagenlecleucel 在爱尔兰治疗儿童和年轻成人 R/R ALL 患者时不具有成本效益（按标价）。在规定的支付意愿阈值下，进一步降低决策（参数）不确定性的研究可能没有价值。然而，该分析存在高度的不确定性，EVPI 分析可能无法捕获这些不确定性。
Objectives: This study evaluates the cost-effectiveness of tisagenlecleucel (a CAR T-cell therapy), versus blinatumomab, for the treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (R/R ALL) in the Irish healthcare setting. The value of conducting further research, to investigate the value of uncertainty associated with the decision problem, is assessed by means of expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses.Methods: A three-state partitioned survival model was developed. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 60 months; general population mortality with a standardized mortality ratio was then applied. Estimated EVPI and EVPPI were scaled up to population according to the incidence of the decision.Results: At list prices, the incremental cost-effectiveness ratio was EUR 73,086 per quality-adjusted life year (QALY) (incremental costs EUR 156,928; incremental QALYs 2.15). The probability of cost-effectiveness, at the willingness-to-pay threshold of EUR 45,000 per QALY, was 16 percent. At this threshold, population EVPI was EUR 314,455; population EVPPI was below EUR 100,000 for each parameter category.Conclusions: Tisagenlecleucel is not cost effective, versus blinatumomab, for the treatment of pediatric and young adult patients with R/R ALL in Ireland (at list prices). Further research to decrease decision (parameter) uncertainty, at the defined willingness-to-pay threshold, may not be of value. However, there is a high degree of uncertainty underpinning the analysis, which may not be captured by EVPI analysis.