论文标题

随机试验及其观察仿真:基准测试和联合分析的框架

Randomized trials and their observational emulations: a framework for benchmarking and joint analysis

论文作者

Dahabreh, Issa J., Steingrimsson, Jon A., Robins, James M., Hernán, Miguel A.

论文摘要

一项随机试验和旨在分别模拟试验样本观察结果的观测数据的分析,但具有相同的资格标准,收集有关某些共享基线协变量的信息,并比较相同处理对相同结果的影响。可以将试验及其仿真的治疗效果估计与基准观测分析方法进行比较。在简化的环境中,完全遵守指定的治疗策略并且没有损失到遵循的损失,我们表明,基准测试依赖于试验及其仿真人群之间的交换性条件,以说明它们之间协变量分布的差异。当这种交换性条件成立,并且还具有因果解释所需的估计所需的常规条件时,我们就会得出对观察到的数据定律的限制。当数据与限制兼容时,可以进行联合分析及其仿真。当数据与限制不兼容时,基于​​(1)估计的估计差异是基于从试验到仿真基础的人群的扩展推断,并且(2)仿真本身可能反映出无法进行基准测试(例如,由于选择性参与该试验)或由于仿真失败(例如,由于案例的失败),但由于案例的效果不佳,但我们无法使用该数据。我们的分析揭示了基准测试尝试如何结合因果假设,数据分析方法和实质性知识来检查观察性分析方法的有效性。

A randomized trial and an analysis of observational data designed to emulate the trial sample observations separately, but have the same eligibility criteria, collect information on some shared baseline covariates, and compare the effects of the same treatments on the same outcomes. Treatment effect estimates from the trial and its emulation can be compared to benchmark observational analysis methods. In a simplified setting with complete adherence to the assigned treatment strategy and no loss-to-follow-up, we show that benchmarking relies on an exchangeability condition between the populations underlying the trial and its emulation, to account for differences in the distribution of covariates between them. When this exchangeability condition holds, and the usual conditions needed for the estimates from the trial and its emulation to have a causal interpretation also hold, we derive restrictions on the law of the observed data. When the data are compatible with the restrictions, joint analysis of the trial and its emulation is possible. When the data are incompatible with the restrictions, a discrepancy between (1) estimates based on extending inferences from the trial to the population underlying the emulation and (2) the emulation itself may reflect either inability to benchmark (e.g., due to selective participation into the trial) or a failure of the emulation (e.g., due to unmeasured confounding), but we cannot use the data to determine which is the case. Our analysis reveals how benchmarking attempts combine causal assumptions, data analysis methods, and substantive knowledge to examine the validity of observational analysis methods.

扫码加入交流群

加入微信交流群

微信交流群二维码

扫码加入学术交流群,获取更多资源