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Data Fusion Using Weakly Aligned Sources

利用弱对齐数据源进行数据融合

We introduce a new data fusion method that utilizes multiple data sources to estimate a smooth, finite-dimensional parameter. Most existing methods only make use of fully aligned data sources that share common conditional distributions of one or more variables of interest. However, in many settings, the scarcity of fully aligned sources can make existing methods require unduly large sample sizes to be useful. Our approach enables the incorporation of weakly aligned data…

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Inference for Treatment-Specific Survival Curves Using Machine Learning

利用机器学习推断处理特定生存曲线

In the absence of data from a randomized trial, researchers may aim to use observational data to draw causal inference about the effect of a treatment on a time-to-event outcome. In this context, interest often focuses on the treatment-specific survival curves, that is, the survival curves were the population under study to be assigned to receive the treatment or not. Under certain conditions, including that all confounders of the treatment-outcome relationship are observed,…

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A General Framework for Inference on Algorithm-Agnostic Variable Importance

与算法无关的变量重要性推断的一般框架

In many applications, it is of interest to assess the relative contribution of features (or subsets of features) toward the goal of predicting a response - in other words, to gauge the variable importance of features. Most recent work on variable importance assessment has focused on describing the importance of features within the confines of a given prediction algorithm. However, such assessment does not necessarily characterize the prediction potential of features, and may…

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Estimating and Testing Vaccine Sieve Effects Using Machine Learning

利用机器学习估计和检验疫苗筛效应

When available, vaccines are an effective means of disease prevention. Unfortunately, efficacious vaccines have not yet been developed for several major infectious diseases, including HIV and malaria. Vaccine sieve analysis studies whether and how the efficacy of a vaccine varies with the genetics of the pathogen of interest, which can guide subsequent vaccine development and deployment. In sieve analyses, the effect of the vaccine on the cumulative incidence corresponding…

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Sensitivity Analysis of Per-Protocol Time-to-Event Treatment Efficacy in Randomized Clinical Trials

随机临床试验中依方案生存终点疗效的敏感性分析

Assessing per-protocol treatment effcacy on a time-to-event endpoint is a common objective of randomized clinical trials. The typical analysis uses the same method employed for the intention-to-treat analysis (e.g., standard survival analysis) applied to the subgroup meeting protocol adherence criteria. However, due to potential post-randomization selection bias, this analysis may mislead about treatment efficacy. Moreover, while there is extensive literature on methods for…

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Partially Hidden Markov Model for Time-Varying Principal Stratification in HIV Prevention Trials

HIV预防试验中时变主分层的部分隐马尔可夫模型

It is frequently of interest to estimate the intervention effect that adjusts for post-randomization variables in clinical trials. In the recently completed HPTN 035 trial, there is differential condom use between the three microbicide gel arms and the No Gel control arm, so that intention to treat (ITT) analyses only assess the net treatment effect that includes the indirect treatment effect mediated through differential condom use. Various statistical methods in causal…

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Proportional hazards models with continuous marks

带连续标记的比例风险模型

For time-to-event data with finitely many competing risks, the proportional hazards model has been a popular tool for relating the cause-specific outcomes to covariates [Prentice et al. Biometrics34 (1978) 541-554]. This article studies an extension of this approach to allow a continuum of competing risks, in which the cause of failure is replaced by a continuous mark only observed at the failure time. We develop inference for the proportional hazards model in which the…

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Semiparametric Estimation of Treatment Effects Given Base-Line Covariates on an Outcome Measured After a Post-Randomization Event Occurs

随机化后事件发生才观测结局时,给定基线协变量的治疗效应半参数估计

Summary We consider estimation, from a double-blind randomized trial, of treatment effect within levels of base-line covariates on an outcome that is measured after a post-treatment event E has occurred in the subpopulation 𝒫E,E that would experience event E regardless of treatment. Specifically, we consider estimation of the parameters γ indexing models for the outcome mean conditional on treatment and base-line covariates in the subpopulation 𝒫E,E. Such parameters are…

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Large sample theory of maximum likelihood estimates in semiparametric biased sampling models

半参数偏倚抽样模型中最大似然估计的大样本理论

Vardi [Ann.Statist.13 178-203 (1985)] introduced an $s$-sample biased sampling model with known selection weight functions, gave a condition under which the common underlying probability distribution $G$ is uniquely estimable and developed simple procedure for computing the nonparametric maximum likelihood estimator (NPMLE) $\mathbb{G}_n$ of $G$. Gill, Vardi and Wellner thoroughly described the large sample properties of Vardi’s NPMLE, giving results on uniform consistency,…

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