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Chengxin Yang

Publications and source records attributed to Chengxin Yang.

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Sample size and power calculations for causal inference with time-to-event outcomes

This paper develops power and sample size formulas for causal inference with time-to-event outcomes. The target estimand is the marginal hazard ratio: the coefficient of a marginal structural Cox proportional hazard model with treatment as the only predictor. We extend the robust sandwich variance theory and derive the analytical form of the asymptotic variance for the inverse probability weighted partial likelihood estimator. Building on this, we derive a new analytical sample size formula valid at any prespecified effect size, applicable to both randomized trials and observational studies. For randomized trials, the formula requires only the canonical inputs of treatment proportion, effect size, and event rate. The new formula corrects the mischaracterization of classic log-rank-based formulas. For observational studies, one additional input suffices: an overlap coefficient summarizing covariate similarity between comparison groups. We further develop a variance inflation approach applicable to any propensity score balancing weights, anchored to the corrected baseline variance. We provide an online calculator and an R package 'PSpower' to implement the method.

stat.ME

Demystify Doubly-Robust Estimation: The Role of Overlap

The doubly-robust (DR) estimator is popular for evaluating causal effects in observational studies and is often perceived as more desirable than inverse probability weighting (IPW) or outcome modeling alone because it provides extra protection against model misspecification. However, double robustness is an asymptotic property that may not hold in finite samples. We investigate how the finite sample performance of the DR estimator depends on the degree of covariate overlap between comparison groups. Using analytical illustrations and extensive simulations under various scenarios with different degrees of covariate overlap and model specifications, we examine the bias and variance of the DR estimator relative to IPW and outcome modeling estimators. We find that: (i) specification of the outcome model has a stronger influence on the DR estimates than specification of the propensity score model, and this dominance increases as overlap decreases; (ii) with poor overlap, the DR estimator generally amplifies the adverse consequences of extreme weights (large bias and/or variance) regardless of model specifications, and is often inferior to both the IPW and outcome modeling estimators. As a practical guide, we recommend always first checking the degree of overlap in applications. In the case of poor overlap, analysts should consider shifting the target population to a subpopulation with adequate overlap via methods such as trimming or overlap weighting.

stat.ME

Propensity score weighted Cox regression for survival outcomes in observational studies with multiple or factorial treatments

In observational studies with survival or time-to-event outcomes, a propensity score weighted marginal Cox proportional hazard model with the treatment variable as the only predictor is commonly used to estimate the causal marginal hazard ratio between two treatments. Observational studies often have more than two treatments, but corresponding analysis methods are limited. In this paper, we combine the propensity score weighting method for multiple treatments and a marginal Cox model with indicators for each treatment to estimate the causal hazard ratios between multiple treatments and a common reference treatment. We illustrate two weighting schemes: inverse probability of treatment weighting and overlap weighting. We prove the consistency of the maximum weighted partial likelihood estimator of the causal marginal hazard ratio and derive a robust sandwich variance estimator. As an important special case of multiple treatments, we elaborate the Cox model for two-way factorial treatments. We apply the method to evaluate the real-world comparative effectiveness of three types of anti-obesity medications on heart failure. We develop an associated R package 'PSsurvival'.

stat.ME

Sample size and power calculations for causal inference of observational studies

This paper investigates the theoretical foundation and develops analytical formulas for sample size and power calculations for causal inference with observational data. By analyzing the variance of an inverse probability weighting estimator of the average treatment effect, we decompose the power calculation into three components: propensity score distribution, potential outcome distribution, and their correlation. We show that to determine the minimal sample size of an observational study, in addition to the standard inputs in the power calculation of randomized trials, it is sufficient to have two parameters, which quantify the strength of the confounder-treatment and the confounder-outcome association, respectively. For the former, we propose using the Bhattacharyya coefficient, which measures the covariate overlap and, together with the treatment proportion, leads to a uniquely identifiable and easily computable propensity score distribution. For the latter, we propose a sensitivity parameter bounded by the R-squared statistic of the regression of the outcome on covariates. Our procedure relies on a parametric propensity score model and a semiparametric restricted mean outcome model, but does not require distributional assumptions on the multivariate covariates. We develop an associated R package PSpower and an online calculator.

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Differentially Private Methods for Releasing Results of Stability Analyses

Data stewards and analysts can promote transparent and trustworthy science and policy-making by facilitating assessments of the sensitivity of published results to alternate analysis choices. For example, researchers may want to assess whether the results change substantially when different subsets of data points (e.g., sets formed by demographic characteristics) are used in the analysis, or when different models (e.g., with or without log transformations) are estimated on the data. Releasing the results of such stability analyses leaks information about the data subjects. When the underlying data are confidential, the data stewards and analysts may seek to bound this information leakage. We present methods for stability analyses that can satisfy differential privacy, a definition of data confidentiality providing such bounds. We use regression modeling as the motivating example. The basic idea is to split the data into disjoint subsets, compute a measure summarizing the difference between the published and alternative analysis on each subset, aggregate these subset estimates, and add noise to the aggregated value to satisfy differential privacy. We illustrate the methods using regressions in which an analyst compares coefficient estimates for different groups in the data, and in which analysts fit two different models on the data.

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