论文标题

使用观察性研究改善试验的推广性

Improving trial generalizability using observational studies

论文作者

Lee, Dasom, Yang, Shu, Dong, Lin, Wang, Xiaofei, Zeng, Donglin, Cai, Jianwen

论文摘要

随机对照试验(RCT)和观察性研究(OSS)的互补特征可共同估计目标人群的平均治疗效应。我们提出了一个校准加权估计器,该估计值可以实现RCT和OS之间的协变量平衡,从而改善了基于试验的估计器的概括性。利用半参数效率理论,我们提出了一个双重强大的增强校准加权估计器,该估计值可实现在识别假设下得出的效率。提供了一种非参数筛分方法作为参数方法的替代方法,该方法可以稳健地近似促进功能和数据适应性的结果预测因子进行校准。我们建立渐近结果,并通过模拟实验确认所提出的估计量的有限样本性能,并应用辅助化学疗法在手术后早期非小细胞肺患者中的治疗效果的估计。

Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces the covariate balance between the RCT and OS, therefore improving the trial-based estimator's generalizability. Exploiting semiparametric efficiency theory, we propose a doubly robust augmented calibration weighting estimator that achieves the efficiency bound derived under the identification assumptions. A nonparametric sieve method is provided as an alternative to the parametric approach, which enables the robust approximation of the nuisance functions and data-adaptive selection of outcome predictors for calibration. We establish asymptotic results and confirm the finite sample performances of the proposed estimators by simulation experiments and an application on the estimation of the treatment effect of adjuvant chemotherapy for early-stage non-small cell lung patients after surgery.

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