Variance Estimation under Two‐Phase Sampling

We consider the variance estimation of the weighted likelihood estimator (WLE) under two‐phase stratified sampling without replacement. Asymptotic variance of the WLE in many semiparametric models contains unknown functions or does not have a closed form. The standard method of the inverse probability weighted (IPW) sample variances of an estimated influence function is then not available in these models. To address this issue, we develop the variance estimation procedure for the WLE in a general semiparametric model. The phase I variance is estimated by taking a numerical derivative of the IPW log likelihood. The phase II variance is estimated based on the bootstrap for a stratified sample in a finite population. Despite a theoretical difficulty of dependent observations due to sampling without replacement, we establish the (bootstrap) consistency of our estimators. Finite sample properties of our method are illustrated in a simulation study.     

An inverse-probability-weighted approach to the estimation of distribution function with middle-censored data

In this article, we propose an inverse-probability-weighted (IPW) estimator of distribution function for middle-censored data. By Jammalamadaka and Mangalam (2003), the IPW estimator is the nonparametric maximum likelihood estimator (NPMLE) when all censored intervals contain at least one uncensored observation. The asymptotic properties of the IPW estimator are derived. A simulation study is conducted to compare the performance between the IPW estimator and the self-consistent estimator (SCE). Simulation results indicate that the performance of the IPW estimator is close to that of the SCE.     

Estimation of Stratified Mark‐Specific Proportional Hazards Models with Missing Marks

Abstract. An objective of randomized placebo‐controlled preventive HIV vaccine efficacy trials is to assess the relationship between the vaccine effect to prevent infection and the genetic distance of the exposing HIV to the HIV strain represented in the vaccine construct. Motivated by this objective, recently a mark‐specific proportional hazards (PH) model with a continuum of competing risks has been studied, where the genetic distance of the transmitting strain is the continuous ‘mark’ defined and observable only in failures. A high percentage of genetic marks of interest may be missing for a variety of reasons, predominantly because rapid evolution of HIV sequences after transmission before a blood sample is drawn from which HIV sequences are measured. This research investigates the stratified mark‐specific PH model with missing marks where the baseline functions may vary with strata. We develop two consistent estimation approaches, the first based on the inverse probability weighted complete‐case (IPW) technique, and the second based on augmenting the IPW estimator by incorporating auxiliary information predictive of the mark. We investigate the asymptotic properties and finite‐sample performance of the two estimators, and show that the augmented IPW estimator, which satisfies a double robustness property, is more efficient.     

Empirical likelihood for nonlinear regression models with nonignorable missing responses

This article develops three empirical likelihood (EL) approaches to estimate parameters in nonlinear regression models in the presence of nonignorable missing responses. These are based on the inverse probability weighted (IPW) method, the augmented IPW (AIPW) method and the imputation technique. A logistic regression model is adopted to specify the propensity score. Maximum likelihood estimation is used to estimate parameters in the propensity score by combining the idea of importance sampling and imputing estimating equations. Under some regularity conditions, we obtain the asymptotic properties of the maximum EL estimators of these unknown parameters. Simulation studies are conducted to investigate the finite sample performance of our proposed estimation procedures. Empirical results provide evidence that the AIPW procedure exhibits better performance than the other two procedures. Data from a survey conducted in 2002 are used to illustrate the proposed estimation procedure. The Canadian Journal of Statistics 48: 386–416; 2020 © 2020 Statistical Society of Canada     

The IPW estimator for the joint distribution function of the gap times from recurrent event data

Lee et al. in 2016 proposed a nonparametric estimator of the joint distribution of the gap time between transplant and the first infection and the following gap times between consecutive infections. In this article, we propose an alternative estimator based on the inverse-probability weighted (IPW) approach. Asymptotic properties of the proposed estimator are established . Simulation results indicate that the IPW estimator performs as well as the estimator proposed by Lee et al. We also propose an IPW estimator for estimating the joint distribution function of the gap times between consecutive recurrent events beyond the first episode.     

Efficient inverse probability weighting method for quantile regression with nonignorable missing data

Quantitle regression (QR) is a popular approach to estimate functional relations between variables for all portions of a probability distribution. Parameter estimation in QR with missing data is one of the most challenging issues in statistics. Regression quantiles can be substantially biased when observations are subject to missingness. We study several inverse probability weighting (IPW) estimators for parameters in QR when covariates or responses are subject to missing not at random. Maximum likelihood and semiparametric likelihood methods are employed to estimate the respondent probability function. To achieve nice efficiency properties, we develop an empirical likelihood (EL) approach to QR with the auxiliary information from the calibration constraints. The proposed methods are less sensitive to misspecified missing mechanisms. Asymptotic properties of the proposed IPW estimators are shown under general settings. The efficiency gain of EL-based IPW estimator is quantified theoretically. Simulation studies and a data set on the work limitation of injured workers from Canada are used to illustrated our proposed methodologies.     

Semiparametric analysis of transformation models with dependently left-truncated and right-censored data

In transplant studies, the patients must survive long enough to receive a transplant, which induces left-truncation. The assumption of independence between failure and truncation times may not hold since a longer transplant waiting time can be associated with a worse survivorship. To take dependence into consideration, we utilize a semiparametric transformation model, where the truncation time is both a truncated variable and a predictor of the time to failure. Using the inverse-probability-weighted (IPW) approach, we propose an IPW estimator of the marginal distribution of waiting time. Simulation studies are conducted to investigate finite sample performance of the proposed estimator. We also apply our methods to bone marrow and heart transplant data.     

Estimation of the joint survival function for successive duration times

In incident cohort studies, survival data often include subjects who have experienced an initiate event but have not experienced a subsequent event at the calendar time of recruitment. During the follow-up periods, subjects may undergo a series of successive events. Since the second/third duration process becomes observable only if the first/second event has occurred, the data are subject to left-truncation and dependent censoring. In this article, using the inverse-probability-weighted (IPW) approach, we propose nonparametric estimators for the estimation of the joint survival function of three successive duration times. The asymptotic properties of the proposed estimators are established. The simple bootstrap methods are used to estimate standard deviations and construct interval estimators. A simulation study is conducted to investigate the finite sample properties of the proposed estimators.     

An inverse-probability-weighted approach to estimation of the bivariate survival function under left-truncation and right-censoring

In this note, we consider estimating the bivariate survival function when both survival times are subject to random left truncation and one of the survival times is subject to random right censoring. Motivated by Satten and Datta [2001. The Kaplan–Meier estimator as an inverse-probability-of-censoring weighted average. Amer. Statist. 55, 207–210], we propose an inverse-probability-weighted (IPW) estimator. It involves simultaneous estimation of the bivariate survival function of the truncation variables and that of the censoring variable and the truncation variable of the uncensored components. We prove that (i) when there is no censoring, the IPW estimator reduces to NPMLE of van der Laan [1996a. Nonparametric estimation of the bivariate survival function with truncated data. J. Multivariate Anal. 58, 107–131] and (ii) when there is random left truncation and right censoring on only one of the components and the other component is always observed, the IPW estimator reduces to the estimator of Gijbels and Gürler [1998. Covariance function of a bivariate distribution function estimator for left truncated and right censored data. Statist. Sin. 1219–1232]. Based on Theorem 3.1 of van der Laan [1996a. Nonparametric estimation of the bivariate survival function with truncated data. J. Multivariate Anal. 58, 107–131, 1996b. Efficient estimation of the bivariate censoring model and repairing NPMLE. Ann. Statist. 24, 596–627], we prove that the IPW estimator is consistent under certain conditions. Finally, we examine the finite sample performance of the IPW estimator in some simulation studies. For the special case that censoring time is independent of truncation time, a simulation study is conducted to compare the performances of the IPW estimator against that of the estimator proposed by van der Laan [1996a. Nonparametric estimation of the bivariate survival function with truncated data. J. Multivariate Anal. 58, 107–131, 1996b. Efficient estimation of the bivariate censoring model and repairing NPMLE. Ann. Statist. 24, 596–627]. For the special case (i), a simulation study is conducted to compare the performances of the IPW estimator against that of the estimator proposed by Huang et al. (2001. Nonnparametric estimation of marginal distributions under bivariate truncation with application to testing for age-of-onset application. Statist. Sin. 11, 1047–1068).     

Estimation of conditional cumulative incidence functions under generalized semiparametric regression models with missing covariates,with application to analysis of biomarker correlates in vaccine trials

This article presents generalized semiparametric regression models for conditional cumulative incidence functions with competing risks data when covariates are missing by sampling design or happenstance. A doubly robust augmented inverse probability weighted (AIPW) complete-case approach to estimation and inference is investigated. This approach modifies IPW complete-case estimating equations by exploiting the key features in the relationship between the missing covariates and the phase-one data to improve efficiency. An iterative numerical procedure is derived to solve the nonlinear estimating equations. The asymptotic properties of the proposed estimators are established. A simulation study examining the finite-sample performances of the proposed estimators shows that the AIPW estimators are more efficient than the IPW estimators. The developed method is applied to the RV144 HIV-1 vaccine efficacy trial to investigate vaccine-induced IgG binding antibodies to HIV-1 as correlates of acquisition of HIV-1 infection while taking account of whether the HIV-1 sequences are near or far from the HIV-1 sequences represented in the vaccine construct.     

Nonparametric Estimation of the Bivariate Distribution Function with Doubly Truncated Data

Doubly truncated data play an important role in the statistical analysis of astronomical observations as well as in survival analysis. In this article, using inverse-probability-weighted (IPW) approaches, we derive the nonparametric maximum likelihood estimator (NPMLE) of joint distribution function with bivariate doubly truncated data. The asymptotic properties of the NPMLE are established. A simulation study is conducted to investigate the performance of the NPMLE.     

Using Inverse Probability Weighting Estimators to Evaluate Various Propensity Scores When Treatment Switching Exists

In this paper, we conduct a Monte Carlo simulation study to evaluate three propensity score (PS) scenarios for estimating an average treatment effect (ATE) in observational studies when treatment switching exists: (a) ignoring treatment switching in subjects (UPS), (b) removing subjects with treatment switching (RPS), and (c) adjusting for treatment switching effect (APS) with two inverse probability weighting estimators, IPW1 and IPW2. We evaluate these six estimators in terms of bias, mean squared error (MSE), empirical standard error (ESE), and coverage probability (CP) under various simulation scenarios. Simulation results show that the IPW2 estimator with RPS has relatively good performance.     

Mark-specific hazard ratio model with missing multivariate marks

An objective of randomized placebo-controlled preventive HIV vaccine efficacy (VE) trials is to assess the relationship between vaccine effects to prevent HIV acquisition and continuous genetic distances of the exposing HIVs to multiple HIV strains represented in the vaccine. The set of genetic distances, only observed in failures, is collectively termed the ‘mark.’ The objective has motivated a recent study of a multivariate mark-specific hazard ratio model in the competing risks failure time analysis framework. Marks of interest, however, are commonly subject to substantial missingness, largely due to rapid post-acquisition viral evolution. In this article, we investigate the mark-specific hazard ratio model with missing multivariate marks and develop two inferential procedures based on (i) inverse probability weighting (IPW) of the complete cases, and (ii) augmentation of the IPW estimating functions by leveraging auxiliary data predictive of the mark. Asymptotic properties and finite-sample performance of the inferential procedures are presented. This research also provides general inferential methods for semiparametric density ratio/biased sampling models with missing data. We apply the developed procedures to data from the HVTN 502 ‘Step’ HIV VE trial.     

缺失数据下的逆概率多重加权分位回归估计及其应用

数据缺失问题普遍存在于应用研究中。在随机缺失机制假定下,本文从模型推断角度出发,针对线性缺失分位回归模型,提出一种新的有效估计方法——逆概率多重加权（IPMW）估计。该方法是在逆概率加权（IPW）估计的基础上,结合倾向得分匹配及模型平均思想,经过多次估计,加权确定最终参数估计结果。该方法适用于响应变量是独立同分布或独立非同分布的情形,并适用于绝大多数缺失场景。经过理论推导及模拟研究发现,IPMW估计量在继承IPW估计量的优势上具有更稳健的性质。最后,将该方法应用于含有缺失数据的微观调查数据中,研究了经济较发达的准一线城市中等收入群体消费水平的影响因素,对比两种估计方法的估计结果及置信带,发现逆概率多重加权估计量的标准偏差更小,估计结果更稳健。     

Efficient Augmented Inverse Probability Weighted Estimation in Missing Data Problems

When analyzing data with missing data, a commonly used method is the inverse probability weighting (IPW) method, which reweights estimating equations with propensity scores. The popularity of the IPW method is due to its simplicity. However, it is often being criticized for being inefficient because most of the information from the incomplete observations is not used. Alternatively, the regression method is known to be efficient but is nonrobust to the misspecification of the regression function. In this article, we propose a novel way of optimally combining the propensity score function and the regression model. The resulting estimating equation enjoys the properties of robustness against misspecification of either the propensity score or the regression function, as well as being locally semiparametric efficient. We demonstrate analytically situations where our method leads to a more efficient estimator than some of its competitors. In a simulation study, we show the new method compares favorably with its competitors in finite samples. Supplementary materials for this article are available online.     

Weighted Likelihood for Semiparametric Models and Two-phase Stratified Samples, with Application to Cox Regression

Abstract.  We consider semiparametric models for which solution of Horvitz–Thompson or inverse probability weighted (IPW) likelihood equations with two-phase stratified samples leads to consistent and asymptotically Gaussian estimators of both Euclidean and non-parametric parameters. For Bernoulli (independent and identically distributed) sampling, standard theory shows that the Euclidean parameter estimator is asymptotically linear in the IPW influence function. By proving weak convergence of the IPW empirical process, and borrowing results on weighted bootstrap empirical processes, we derive a parallel asymptotic expansion for finite population stratified sampling. Several of our key results have been derived already for Cox regression with stratified case–cohort and more general survey designs. This paper is intended to help interpret this previous work and to pave the way towards a general Horvitz–Thompson approach to semiparametric inference with data from complex probability samples.     

Nonparametric Estimators of the Distribution Function for One Modified Model of Current Status Data

In this article, we consider the estimation of distribution function for one modified form of current status data. An inverse-probability-weighted (IPW) estimator and a self-consistent estimator (SCE) are proposed. The asymptotic properties of the IPW estimator are derived. A simulation study is conducted to compare the performances among the IPW estimator, SCE, and the product-limit estimator proposed by Patilea and Rolin (2006 Patilea , V. , Rolin , J.-M. (2006). Product-limit estimators of the survival function for two modified forms of current-status data. Bernoulli 12(5):801–819.[Crossref], [Web of Science ®] , [Google Scholar]). Simulation results indicate that when right censoring is light and left censoring is heavy, both IPW estimator and SCE can outperform the product-limit estimator. The performances of the IPW estimator and SCE are close to each other.     

Estimation and inference of combining quantile and least-square regressions with missing data

In this paper, we consider how to incorporate quantile information to improve estimator efficiency for regression model with missing covariates. We combine the quantile information with least-squares normal equations and construct an unbiased estimating equations (EEs). The lack of smoothness of the objective EEs is overcome by replacing them with smooth approximations. The maximum smoothed empirical likelihood (MSEL) estimators are established based on inverse probability weighted (IPW) smoothed EEs and their asymptotic properties are studied under some regular conditions. Moreover, we develop two novel testing procedures for the underlying model. The finite-sample performance of the proposed methodology is examined by simulation studies. A real example is used to illustrate our methods.     

Combining Inverse Probability Weighting and Multiple Imputation to Improve Robustness of Estimation

Inverse probability weighting (IPW) and multiple imputation are two widely adopted approaches dealing with missing data. The former models the selection probability, and the latter models data distribution. Consistent estimation requires correct specification of corresponding models. Although the augmented IPW method provides an extra layer of protection on consistency, it is usually not sufficient in practice as the true data‐generating process is unknown. This paper proposes a method combining the two approaches in the same spirit of calibration in sampling survey literature. Multiple models for both the selection probability and data distribution can be simultaneously accounted for, and the resulting estimator is consistent if any model is correctly specified. The proposed method is within the framework of estimating equations and is general enough to cover regression analysis with missing outcomes and/or missing covariates. Results on both theoretical and numerical investigation are provided.     

Non parametric analysis of gap times for bivariate recurrent event data with cure fraction

Recurrent event data often arise in longitudinal studies. In many applications, subjects may experience two different types of events alternatively over time or a pair of subjects may experience recurrent events of the same type. Medical advances have made it possible for some patients to be cured such that the disease of interest does not recur. In this article, we consider non parametric analysis of bivariate recurrent event data with cure fraction. Using the inverse-probability weighted (IPW) approach, we propose non parametric estimators for the proportion of cured patients and for the joint distribution functions of bivariate recurrence times of the uncured ones. The asymptotic properties of the proposed estimators are established. Simulation study indicates that the proposed estimators perform well in finite samples.