Do Ngoc, Thielen Frederick W
Erasmus School of Health Policy & Management, Erasmus University of Rotterdam, Rotterdam, The Netherlands; School of Speech, Language, and Hearing Sciences, San Diego State University, CA, USA.
Erasmus School of Health Policy & Management, Erasmus University of Rotterdam, Rotterdam, The Netherlands; Erasmus Centre for Health Economics Rotterdam (EsCHER), Erasmus University Rotterdam, Rotterdam, The Netherlands.
Value Health. 2023 Apr;26(4):477-486. doi: 10.1016/j.jval.2022.11.002. Epub 2022 Nov 11.
Efficacy of venetoclax plus obinutuzumab (VenO) compared with chlorambucil plus obinutuzumab (ClbO) for treatment-naïve adult patients with chronic lymphocytic leukemia (CLL) with coexisting medical conditions was investigated in CLL14 (NCT02242942). Our aim was to evaluate the cost-effectiveness of VenO versus ClbO for these patients from a Dutch societal perspective.
A 3-state partitioned survival model was constructed to evaluate the cost-effectiveness of VenO. The outcome of the analysis was the incremental cost-effectiveness ratio (ICER) with effectiveness measured in quality-adjusted life-years (QALYs) gained. Uncertainty was explored through deterministic and probabilistic sensitivity analyses, scenario analyses, and value of information analysis (VOI).
The base case resulted in a discounted ICER -49 928 EUR/QALY gained (with incremental negative costs and positive effects). None of the ICERs resulted from deterministic sensitivity and scenario analyses exceeded the chosen willingness-to-pay threshold of 20 000 EUR/QALY, and > 99% of the iterations in the probabilistic sensitivity analysis were cost-effective. VOI analyses showed a maximum expected value of eliminating all model parameter uncertainty of 183 591 EUR.
Our study demonstrated VenO being dominant over ClbO in treatment-naïve adult patients with CLL assuming a Dutch societal perspective. We concluded that our results are robust as tested through sensitivity and scenario analyses. Additionally, the VOI analyses confirmed that our current evidence base is strong enough to generate reliable results for our study. Nevertheless, further research based on real-world data or longer follow-up period could further contribute to the robustness of the current study's conclusions.
在CLL14（NCT02242942）研究中，调查了维奈克拉联合奥妥珠单抗（VenO）与苯丁酸氮芥联合奥妥珠单抗（ClbO）相比，用于初治且伴有合并症的成年慢性淋巴细胞白血病（CLL）患者的疗效。我们的目的是从荷兰社会角度评估VenO与ClbO对这些患者的成本效益。
构建一个三状态分区生存模型来评估VenO的成本效益。分析结果是增量成本效益比（ICER），效益以获得的质量调整生命年（QALY）衡量。通过确定性和概率敏感性分析、情景分析以及信息价值分析（VOI）来探索不确定性。
基础病例得出贴现后的ICER为-49 928欧元/QALY（增量成本为负，效果为正）。确定性敏感性分析和情景分析得出的ICER均未超过选定的支付意愿阈值20 000欧元/QALY，概率敏感性分析中>99%的迭代具有成本效益。VOI分析表明，消除所有模型参数不确定性的最大预期价值为183 591欧元。
我们的研究表明，从荷兰社会角度来看，在初治成年CLL患者中，VenO优于ClbO。我们得出结论，通过敏感性和情景分析测试，我们的结果是稳健的。此外，VOI分析证实，我们目前的证据基础足够强大，能够为我们的研究产生可靠的结果。然而，基于真实世界数据或更长随访期的进一步研究可能会进一步增强当前研究结论的稳健性。
