Division of Biostatistics, School of Public Health, University of Minnesota, Minneapolis, MN, USA.
Division of Environmental Health Sciences, School of Public Health, University of Minnesota, Minneapolis, MN, USA.
J Biopharm Stat. 2023 Sep 3;33(5):653-676. doi: 10.1080/10543406.2023.2170404. Epub 2023 Mar 6.
Individuals can vary drastically in their response to the same treatment, and this heterogeneity has driven the push for more personalized medicine. Accurate and interpretable methods to identify subgroups that respond to the treatment differently from the population average are necessary to achieving this goal. The Virtual Twins (VT) method is a highly cited and implemented method for subgroup identification because of its intuitive framework. However, since its initial publication, many researchers still rely heavily on the authors' initial modeling suggestions without examining newer and more powerful alternatives. This leaves much of the potential of the method untapped. We comprehensively evaluate the performance of VT with different combinations of methods in each of its component steps, under a collection of linear and nonlinear problem settings. Our simulations show that the method choice for Step 1 of VT, in which dense models with high predictive performance are fit for the potential outcomes, is highly influential in the overall accuracy of the method, and Superlearner is a promising choice. We illustrate our findings by using VT to identify subgroups with heterogeneous treatment effects in a randomized, double-blind trial of very low nicotine content cigarettes.
个体对相同治疗的反应可能有很大差异,这种异质性推动了更个性化医疗的发展。为了实现这一目标,有必要采用准确且可解释的方法来识别对治疗的反应与人群平均水平不同的亚组。由于其直观的框架,虚拟双胞胎 (VT) 方法是一种被高度引用和实施的亚组识别方法。然而,自最初发表以来,许多研究人员仍然严重依赖作者最初的建模建议,而没有检查更新和更强大的替代方法。这使得该方法的大部分潜力未被挖掘。我们在一系列线性和非线性问题设置下,全面评估了 VT 在其每个组成步骤中与不同方法组合的性能。我们的模拟结果表明,VT 第一步中方法的选择,即对潜在结果拟合具有高预测性能的密集模型,对该方法的整体准确性有很大影响,而 Superlearner 是一个很有前途的选择。我们通过使用 VT 来识别随机、双盲试验中极低尼古丁含量香烟的治疗效果异质性亚组来说明我们的发现。