National Centre for Pharmacoeconomics, Old Stone Building, St James's Hospital, Dublin, Ireland.
Department of Pharmacology and Therapeutics, Trinity Centre for Health Sciences, St James's Hospital, Dublin, Ireland.
Int J Technol Assess Health Care. 2022 Jul 11;38(1):e56. doi: 10.1017/S0266462322000356.
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.
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.
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.
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.
本研究评估了 tisagenlecleucel（一种 CAR-T 细胞疗法）与blinatumomab 相比，用于治疗爱尔兰医疗保健环境中患有复发/难治性急性淋巴细胞白血病（R/R ALL）的儿科和年轻成年患者的成本效益。通过预期完美信息价值（EVPI）和部分 EVPI（EVPPI）分析来评估进一步研究以调查与决策问题相关的不确定性价值的重要性。
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 tisagenlecleucel 臂中的患者分为两组。生存情况外推至 60 个月；然后应用标准化死亡率的一般人群死亡率。根据决策的发生率对估计的 EVPI 和 EVPPI 进行规模调整。
以标价计算，每增加一个质量调整生命年（QALY）的增量成本效益比为 73086 欧元（增量成本为 156928 欧元；增量 QALYs 为 2.15）。在 45000 欧元/QALY 的支付意愿阈值下，成本效益的可能性为 16%。在此阈值下，人群 EVPI 为 314455 欧元；对于每个参数类别，人群 EVPPI 均低于 100000 欧元。
在爱尔兰（以标价计算），tisagenlecleucel 与blinatumomab 相比，治疗 R/R ALL 的儿科和年轻成年患者并不具有成本效益。在既定的支付意愿阈值下，进一步研究以降低决策（参数）不确定性可能没有价值。然而，分析的基础存在高度的不确定性，这可能无法通过 EVPI 分析来捕捉。
