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1. Analysis of the time-varying Cox model for the cause-specific hazard functions with missing causes

   https://doi.org/10.1007/s10985-020-09497-y  

   Heng, Fei; Sun, Yanqing; Hyun, Seunggeun; Gilbert, Peter B. (January 2020, Lifetime Data Analysis)

   This paper studies theCox model with time-varying coefficients for cause-specific hazard functions when the causes of failure are subject to missingness. Inverse probability weighted and augmented inverse probability weighted estimators are investigated. The latter is considered as a two-stage estimator by directly utilizing the inverse probability weighted estimator and through modeling available auxiliary variables to improve efficiency. The asymptotic properties of the two estimators are investigated. Hypothesis testing procedures are developed to test the null hypotheses that the covariate effects are zero and that the covariate effects are constant. We conduct simulation studies to examine the finite sample properties of the proposed estimation and hypothesis testing procedures under various settings of the auxiliary variables and the percentages of the failure causes that are missing. These simulation results demonstrate that the augmented inverse probability weighted estimators are more efficient than the inverse probability weighted estimators and that the proposed testing procedures have the expected satisfactory results in sizes and powers. The proposed methods are illustrated using the Mashi clinical trial data for investigating the effect of randomization to formula-feeding versus breastfeeding plus extended infant zidovudine prophylaxis on death due to mother-to-child HIV transmission in Botswana. 

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2. Nonstationary A/B Tests: Optimal Variance Reduction, Bias Correction, and Valid Inference

   https://doi.org/10.1287/mnsc.2022.01205  

   Wu, Yuhang; Zheng, Zeyu; Zhang, Guangyu; Zhang, Zuohua; Wang, Chu (June 2025, Management Science)

   We develop an analytical framework to appropriately model and adequately analyze A/B tests in presence of nonparametric nonstationarities in the targeted business metrics. A/B tests, also known as online randomized controlled experiments, have been used at scale by data-driven enterprises to guide decisions and test innovative ideas to improve core business metrics. Meanwhile, nonstationarities, such as the time-of-day effect and the day-of-week effect, can often arise nonparametrically in key business metrics involving purchases, revenue, conversions, customer experiences, and so on. First, we develop a generic nonparametric stochastic model to capture nonstationarities in A/B test experiments, where each sample represents a visit or action associated with a time label. We build a practically relevant limiting regime to facilitate analyzing large-sample estimator performances under nonparametric nonstationarities. Second, we show that ignoring or inadequately addressing nonstationarities can cause standard A/B test estimators to have suboptimal variance and nonvanishing bias, therefore leading to loss of statistical efficiency and accuracy. We provide a new estimator that views time as a continuous strata and performs poststratification with a data-dependent number of stratification levels. Without making parametric assumptions, we prove a central limit theorem for the proposed estimator and show that the estimator attains the best achievable asymptotic variance and is asymptotically unbiased. Third, we propose a time-grouped randomization that is designed to balance treatment and control assignments at granular time scales. We show that when the time-grouped randomization is integrated to standard experimental designs to generate experiment data, simple A/B test estimators can achieve asymptotically optimal variance. A brief account of numerical experiments are conducted to illustrate the analysis. This paper was accepted by Baris Ata, stochastic models and simulation. Supplemental Material: The online appendices and data files are available at https://doi.org/10.1287/mnsc.2022.01205 . 

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3. Statistical and Causal Robustness for Causal Null Hypothesis Tests

   Yang, Junhui; Bhattacharya, Rohit; Lee, Youjin; Westling, Ted (April 2024, arXiv)

   Prior work applying semiparametric theory to causal inference has primarily focused on deriving estimators that exhibit statistical robustness under a prespecified causal model that permits identification of a desired causal parameter. However, a fundamental challenge is correct specification of such a model, which usually involves making untestable assumptions. Evidence factors is an approach to combining hypothesis tests of a common causal null hypothesis under two or more candidate causal models. Under certain conditions, this yields a test that is valid if at least one of the underlying models is correct, which is a form of causal robustness. We propose a method of combining semiparametric theory with evidence factors. We develop a causal null hypothesis test based on joint asymptotic normality of asymptotically linear semiparametric estimators, where each estimator is based on a distinct identifying functional derived from each of candidate causal models. We show that this test provides both statistical and causal robustness in the sense that it is valid if at least one of the proposed causal models is correct, while also allowing for slower than parametric rates of convergence in estimating nuisance functions. We demonstrate the effectiveness of our method via simulations and applications to the Framingham Heart Study and Wisconsin Longitudinal Study. 

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4. Statistical and Causal Robustness for Causal Null Hypothesis Tests

   Yang, Junhui; Bhattacharya, Rohit; Lee, Youjin; Westling, Ted (July 2024, Proceedings of Machine Learning Research)

   Prior work applying semiparametric theory to causal inference has primarily focused on deriving estimators that exhibit statistical robustness under a prespecified causal model that permits identification of a desired causal parameter. However, a fundamental challenge is correct specification of such a model, which usually involves making untestable assumptions. Evidence factors is an approach to combining hypothesis tests of a common causal null hypothesis under two or more candidate causal models. Under certain conditions, this yields a test that is valid if at least one of the underlying models is correct, which is a form of causal robustness. We propose a method of combining semiparametric theory with evidence factors. We develop a causal null hypothesis test based on joint asymptotic normality of K asymptotically linear semiparametric estimators, where each estimator is based on a distinct identifying functional derived from each of K candidate causal models. We show that this test provides both statistical and causal robustness in the sense that it is valid if at least one of the K proposed causal models is correct, while also allowing for slower than parametric rates of convergence in estimating nuisance functions. We demonstrate the effectiveness of our method via simulations and applications to the Framingham Heart Study and Wisconsin Longitudinal Study. 

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5. Covariate-adjusted log-rank test: guaranteed efficiency gain and universal applicability

   https://doi.org/10.1093/biomet/asad045  

   Ye, Ting; Shao, Jun; Yi, Yanyao (July 2023, Biometrika)

   Summary Nonparametric covariate adjustment is considered for log-rank-type tests of the treatment effect with right-censored time-to-event data from clinical trials applying covariate-adaptive randomization. Our proposed covariate-adjusted log-rank test has a simple explicit formula and a guaranteed efficiency gain over the unadjusted test. We also show that our proposed test achieves universal applicability in the sense that the same formula of test can be universally applied to simple randomization and all commonly used covariate-adaptive randomization schemes such as the stratified permuted block and the Pocock–Simon minimization, which is not a property enjoyed by the unadjusted log-rank test. Our method is supported by novel asymptotic theory and empirical results for Type-I error and power of tests. 

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