Xuan Jingyi, Mt-Isa Shahrul, Latimer Nicholas, Bell Gorrod Helen, Malbecq William, Vandormael Kristel, Yorke-Edwards Victoria, White Ian R
MRC Clinical Trials Unit at UCL, University College London, London, UK.
Biostatistics and Research Decision Sciences (BARDS) Health Technology Assessment (HTA) Statistics, MSD, Zurich, Switzerland.
Stat Methods Med Res. 2025 Feb;34(2):286-306. doi: 10.1177/09622802241289559. Epub 2024 Dec 12.
Deviation from the treatment strategy under investigation occurs in many clinical trials. We term this intervention deviation. Per-protocol analyses are widely adopted to estimate a hypothetical estimand without the occurrence of intervention deviation. Per-protocol by censoring is prone to selection bias when intervention deviation is associated with time-varying confounders that also influence counterfactual outcomes. This can be corrected by inverse probability of censoring weighting, which gives extra weight to uncensored individuals who had similar prognostic characteristics to censored individuals. Such weights are computed by modelling selected covariates. Inverse probability of censoring weighting relies on the no unmeasured confounding assumption whose plausibility is not statistically testable. Suboptimal implementation of inverse probability of censoring weighting which violates the assumption will lead to bias. In a simulation study, we evaluated the performance of per-protocol and inverse probability of censoring weighting with different implementations to explore whether inverse probability of censoring weighting is a safe alternative to per-protocol. Scenarios were designed to vary intervention deviation in one or both arms with different prevalences, correlation between two confounders, effect of each confounder, and sample size. Results show that inverse probability of censoring weighting with different combinations of covariates outperforms per-protocol in most scenarios, except for an unusual case where selection bias caused by two confounders is in two directions, and 'cancels' out.
在许多临床试验中都会出现与正在研究的治疗策略的偏差。我们将这种偏差称为干预偏差。采用符合方案分析来估计一个假设的估计量,前提是不存在干预偏差。当干预偏差与也会影响反事实结果的随时间变化的混杂因素相关时,通过截尾进行的符合方案分析容易产生选择偏倚。这可以通过截尾加权的逆概率来校正,该方法会给与截尾个体具有相似预后特征的未截尾个体额外的权重。此类权重通过对选定的协变量进行建模来计算。截尾加权的逆概率依赖于无未测量混杂因素的假设,而该假设的合理性无法通过统计检验。违反该假设的截尾加权逆概率的次优实施会导致偏差。在一项模拟研究中,我们评估了不同实施方式下符合方案分析和截尾加权逆概率的性能,以探讨截尾加权逆概率是否是符合方案分析的安全替代方法。设计了不同的场景,使一个或两个组中的干预偏差在不同的患病率、两个混杂因素之间的相关性、每个混杂因素的效应以及样本量方面有所不同。结果表明,在大多数情况下,不同协变量组合的截尾加权逆概率优于符合方案分析,但存在一种不寻常的情况,即由两个混杂因素导致的选择偏倚方向相反且相互“抵消”。