Department of Pharmacoeconomics, School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, China.
Center for Pharmacoeconomics and Outcomes Research, China Pharmaceutical University, Nanjing, China.
Front Public Health. 2022 Apr 15;10:869960. doi: 10.3389/fpubh.2022.869960. eCollection 2022.
Unresectable hepatocellular carcinoma (uHCC) is the main histological subtype of liver cancer and causes a great disease burden in China. We aimed to evaluate the cost-effectiveness of five first-line systemic treatments newly approved in the Chinese market for the treatment of uHCC, namely, sorafenib, lenvatinib, donafenib, sintilimab plus bevacizumab (D + A), and atezolizumab plus bevacizumab (T + A) from the perspective of China's healthcare system, to provide a basis for decision-making.
We constructed a network meta-analysis of 4 clinical trials and used fractional polynomial models to indirectly compare the effectiveness of treatments. The partitioned survival model was used for cost-effectiveness analysis. Primary model outcomes included the costs in US dollars and health outcomes in quality-adjusted life-years (QALYs) and the incremental cost-effectiveness ratio (ICER) under a willingness-to-pay threshold of $33,521 (3 times the per capita gross domestic product in China) per QALY. We performed deterministic and probabilistic sensitivity analyses to investigate the robustness. To test the effect of active treatment duration on the conclusions, we performed a scenario analysis.
Compared with sorafenib, lenvatinib, donafenib, D + A, and T + A regimens, it yielded an increase of 0.25, 0.30, 0.95, and 1.46 life-years, respectively. Correspondingly, these four therapies yielded an additional 0.16, 0.19, 0.51, and 0.86 QALYs and all four ICERs, $40,667.92/QALY gained, $27,630.63/QALY gained, $51,877.36/QALY gained, and $130,508.44/QALY gained, were higher than $33,521 except for donafenib. T + A was the most effective treatment and donafenib was the most economical option. Sensitivity and scenario analysis results showed that the base-case analysis was highly reliable.
Although combination therapy could greatly improve patients with uHCC survival benefits, under the current WTP, donafenib is still the most economical option.
不可切除肝细胞癌(uHCC)是肝癌的主要组织学亚型,在中国造成了巨大的疾病负担。我们旨在从中国医疗保健系统的角度评估五种新批准的用于治疗 uHCC 的一线系统治疗药物(索拉非尼、仑伐替尼、多纳非尼、信迪利单抗联合贝伐珠单抗(D+A)和阿替利珠单抗联合贝伐珠单抗(T+A))的成本效益,为决策提供依据。
我们构建了一个包含 4 项临床试验的网络荟萃分析,并使用分数多项式模型间接比较治疗效果。分割生存模型用于成本效益分析。主要模型结果包括以美元计的成本和以质量调整生命年(QALY)计的健康结果,以及意愿支付阈值为 33521 美元/QALY(中国人均国内生产总值的 3 倍)的增量成本效益比(ICER)。我们进行了确定性和概率敏感性分析以检验稳健性。为了检验治疗持续时间对结论的影响,我们进行了情景分析。
与索拉非尼、仑伐替尼、多纳非尼、D+A 和 T+A 方案相比,仑伐替尼、多纳非尼、D+A 和 T+A 方案分别使生命年增加 0.25、0.30、0.95 和 1.46 年。相应地,这四种疗法使 QALY 分别额外增加 0.16、0.19、0.51 和 0.86,且所有四种 ICER 均高于 33521 美元,分别为 40667.92 美元/QALY、27630.63 美元/QALY、51877.36 美元/QALY 和 130508.44 美元/QALY,除了多纳非尼。T+A 是最有效的治疗方法,而多纳非尼是最经济的选择。敏感性和情景分析结果表明,基础分析结果非常可靠。
尽管联合治疗可以极大地提高 uHCC 患者的生存获益,但在目前的 WTP 下,多纳非尼仍然是最经济的选择。