背景:初始干预和后续治疗的治疗持续时间(DOT)是确定抗癌药物预算影响分析(BIA)计算准确性的关键。然而,现有的研究只使用简单的假设作为点的代理,导致高度的偏差。
目的:为了提高抗癌药物BIA的准确性和可靠性,解决有关DOT的问题,我们提出了一种基于个体患者数据(IPD)的替代方法,该方法从已发表的KaplanMeier生存曲线重建IPD,以估计DOT.
方法:我们为这种新方法开发了一个四步方法框架,以帕博利珠单抗治疗微卫星不稳定性高(MSI-H)晚期结直肠癌为例:(1)重建IPD;(2)计算每位患者初始干预和后续治疗的总DOT;(3)分配随机时间和DOT;(4)多重置换采样和平均值的计算.
结果:使用这种方法,可以计算BIA时间范围内每年初始干预和后续治疗的平均DOT,并将其用于计算每年消耗的资源和成本.在我们的例子中,从第一年到第四年,pembrolizumab初始干预的平均DOT为4.90、6.60、5.24和5.06个月,分别,而后续治疗的平均DOT为0.75、2.84、2.99和2.50个月,分别。
结论:与常规方法相比,基于IPD的重建方法可以提高抗癌药物BIA的准确性和可靠性,并且可以广泛使用,尤其是对抗癌药物具有优异的疗效。
BACKGROUND: The duration of treatment (DOT) of the initial intervention and subsequent treatment is the key to determining the accuracy of anticancer-drug budget impact analysis (BIA) calculations. However, existing studies only use simple assumptions as a proxy for DOT, resulting in a high degree of bias.
OBJECTIVE: To enhance the accuracy and reliability of anticancer-drug BIA and solve the problem regarding DOT, we propose an alternative individual patient data (IPD)-based approach that reconstructs IPD from the published Kaplan Meier survival curves to estimate DOT.
METHODS: We developed a four-step methodological framework for this new approach, taking the use of pembrolizumab in treating microsatellite-instability-high (MSI-H) advanced colorectal cancer as an example: (1) reconstructing the IPD; (2) calculating the total DOT of the initial intervention and subsequent treatment for each patient; (3) assigning a randomized time and DOT; and (4) multiple replacement sampling and calculation of the mean value.
RESULTS: Using this approach, the average DOT for the initial intervention and subsequent treatment in each year of the BIA time horizon can be calculated and used to calculate the resources consumed and costs in each year. In our example, the average DOT for the initial intervention with pembrolizumab from the first to the fourth year was 4.90, 6.60, 5.24, and 5.06 months, respectively, while the average DOT for subsequent treatment was 0.75, 2.84, 2.99, and 2.50 months, respectively.
CONCLUSIONS: The reconstructed IPD-based approach can improve the accuracy and reliability of anticancer-drug BIA compared with conventional methods, and can be widely used, especially for anticancer drugs with excellent efficacy.