An important consideration in clinical research studies is proper evaluation of internal and external validity. While randomized clinical trials can overcome several threats to internal validity, they may be prone to poor external validity. Conversely, large prospective observational studies sampled from a broadly generalizable population may be externally valid, yet susceptible to threats to internal validity, particularly confounding. Thus, methods that address confounding and enhance transportability of study results across populations are essential for internally and externally valid causal inference, respectively. We develop a weighting method which estimates the effect of an intervention on an outcome in an observational study which can then be transported to a second, possibly unrelated target population. The proposed methodology employs calibration estimators to generate complementary balancing and sampling weights to address confounding and transportability, respectively, enabling valid estimation of the target population average treatment effect. A simulation study is conducted to demonstrate the advantages and similarities of the calibration approach against alternative techniques. We also test the performance of the calibration estimator-based inference in a motivating real data example comparing whether the effect of biguanides versus sulfonylureas - the two most common oral diabetes medication classes for initial treatment - on all-cause mortality described in a historical cohort applies to a contemporary cohort of US Veterans with diabetes.
翻译:临床研究的一个重要考虑是对内部和外部有效性的适当评估。尽管随机临床试验可以克服对内和外部有效性的几种威胁,但可能缺乏外部有效性。相反,从广泛普遍人口抽样的大规模未来观察研究可能具有外部有效性,但有可能对内部有效性造成威胁,特别是混乱。因此,解决不同人口之间研究结果的混乱和可转移性的方法对内部和外部有效的因果关系推断分别至关重要。我们开发了一种加权方法,用以估计干预对观察研究结果的影响,然后可以传送到第二个可能不相干的目标人口。拟议方法采用校准估计器,产生互补的平衡和抽样权重,分别处理混杂和可移动性,从而能够有效估计目标人口平均治疗效果。进行模拟研究,以表明校准方法相对于其他技术的利弊端和相似性。我们还测试了校准估计基于推断的性表现,以真实数据为示例,比较大量子体和磺基质(SOSYureas)的效应。拟议方法采用两种最常见的口服糖尿病药物类,用于首次治疗。