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.     

Pareto Sampling versus Sampford and Conditional Poisson Sampling

Abstract.  Pareto sampling was introduced by Rosén in the late 1990s. It is a simple method to get a fixed size π ps sample though with inclusion probabilities only approximately as desired. Sampford sampling, introduced by Sampford in 1967, gives the desired inclusion probabilities but it may take time to generate a sample. Using probability functions and Laplace approximations, we show that from a probabilistic point of view these two designs are very close to each other and asymptotically identical. A Sampford sample can rapidly be generated in all situations by letting a Pareto sample pass an acceptance–rejection filter. A new very efficient method to generate conditional Poisson ( CP ) samples appears as a byproduct. Further, it is shown how the inclusion probabilities of all orders for the Pareto design can be calculated from those of the CP design. A new explicit very accurate approximation of the second-order inclusion probabilities, valid for several designs, is presented and applied to get single sum type variance estimates of the Horvitz–Thompson estimator.     

Multistage Sampling Designs and Estimating Equations

In some applications it is cost efficient to sample data in two or more stages. In the first stage a simple random sample is drawn and then stratified according to some easily measured attribute. In each subsequent stage a random subset of previously selected units is sampled for more detailed and costly observation, with a unit's sampling probability determined by its attributes as observed in the previous stages. This paper describes multistage sampling designs and estimating equations based on the resulting data. Maximum likelihood estimates (MLEs) and their asymptotic variances are given for designs using parametric models. Horvitz–Thompson estimates are introduced as alternatives to MLEs, their asymptotic distributions are derived and their strengths and weaknesses are evaluated. The designs and the estimates are illustrated with data on corn production.     

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.     

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.     

Pseudo‐empirical likelihood ratio confidence intervals for complex surveys

The authors show how an adjusted pseudo‐empirical likelihood ratio statistic that is asymptotically distributed as a chi‐square random variable can be used to construct confidence intervals for a finite population mean or a finite population distribution function from complex survey samples. They consider both non‐stratified and stratified sampling designs, with or without auxiliary information. They examine the behaviour of estimates of the mean and the distribution function at specific points using simulations calling on the Rao‐Sampford method of unequal probability sampling without replacement. They conclude that the pseudo‐empirical likelihood ratio confidence intervals are superior to those based on the normal approximation, whether in terms of coverage probability, tail error rates or average length of the intervals.     

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     

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.     

Weighted empirical likelihood inference for multiple samples

We propose a weighted empirical likelihood approach to inference with multiple samples, including stratified sampling, the estimation of a common mean using several independent and non-homogeneous samples and inference on a particular population using other related samples. The weighting scheme and the basic result are motivated and established under stratified sampling. We show that the proposed method can ideally be applied to the common mean problem and problems with related samples. The proposed weighted approach not only provides a unified framework for inference with multiple samples, including two-sample problems, but also facilitates asymptotic derivations and computational methods. A bootstrap procedure is also proposed in conjunction with the weighted approach to provide better coverage probabilities for the weighted empirical likelihood ratio confidence intervals. Simulation studies show that the weighted empirical likelihood confidence intervals perform better than existing ones.     

Make assurance double sure: combination of two disclosure limitation methods and estimation of general regression models

In order to guarantee confidentiality and privacy of firm-level data, statistical offices apply various disclosure limitation techniques. However, each anonymization technique has its protection limits such that the probability of disclosing the individual information for some observations is not minimized. To overcome this problem, we propose combining two separate disclosure limitation techniques, blanking and multiplication of independent noise, in order to protect the original dataset. The proposed approach yields a decrease in the probability of reidentifying/disclosing individual information and can be applied to linear and nonlinear regression models. We show how to combine the blanking method with the multiplicative measurement error method and how to estimate the model by combining the multiplicative Simulation-Extrapolation (M-SIMEX) approach from Nolte (, 2007) on the one side with the Inverse Probability Weighting (IPW) approach going back to Horwitz and Thompson (J. Am. Stat. Assoc. 47:663–685, 1952) and on the other side with matching methods, as an alternative to IPW, like the semiparametric M-Estimator proposed by Flossmann (, 2007). Based on Monte Carlo simulations, we show that multiplicative measurement error combined with blanking as a masking procedure does not necessarily lead to a severe reduction in the estimation quality, provided that its effects on the data generating process are known.     

Marginal regression models with a time to event outcome and discrete multiple source predictors

Information from multiple informants is frequently used to assess psychopathology. We consider marginal regression models with multiple informants as discrete predictors and a time to event outcome. We fit these models to data from the Stirling County Study; specifically, the models predict mortality from self report of psychiatric disorders and also predict mortality from physician report of psychiatric disorders. Previously, Horton et al. found little relationship between self and physician reports of psychopathology, but that the relationship of self report of psychopathology with mortality was similar to that of physician report of psychopathology with mortality. Generalized estimating equations (GEE) have been used to fit marginal models with multiple informant covariates; here we develop a maximum likelihood (ML) approach and show how it relates to the GEE approach. In a simple setting using a saturated model, the ML approach can be constructed to provide estimates that match those found using GEE. We extend the ML technique to consider multiple informant predictors with missingness and compare the method to using inverse probability weighted (IPW) GEE. Our simulation study illustrates that IPW GEE loses little efficiency compared with ML in the presence of monotone missingness. Our example data has non-monotone missingness; in this case, ML offers a modest decrease in variance compared with IPW GEE, particularly for estimating covariates in the marginal models. In more general settings, e.g., categorical predictors and piecewise exponential models, the likelihood parameters from the ML technique do not have the same interpretation as the GEE. Thus, the GEE is recommended to fit marginal models for its flexibility, ease of interpretation and comparable efficiency to ML in the presence of missing data.     

Asymptotic results for fitting marginal hazards models from stratified case-cohort studies with multiple disease outcomes

In stratified case-cohort designs, samplings of case-cohort samples are conducted via a stratified random sampling based on covariate information available on the entire cohort members. In this paper, we extended the work of Kang & Cai (2009) to a generalized stratified case-cohort study design for failure time data with multiple disease outcomes. Under this study design, we developed weighted estimating procedures for model parameters in marginal multiplicative intensity models and for the cumulative baseline hazard function. The asymptotic properties of the estimators are studied using martingales, modern empirical process theory, and results for finite population sampling.     

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.     

Probability density estimation with data missing at random when covariables are present

This paper addresses the problem of the probability density estimation in the presence of covariates when data are missing at random (MAR). The inverse probability weighted method is used to define a nonparametric and a semiparametric weighted probability density estimators. A regression calibration technique is also used to define an imputed estimator. It is shown that all the estimators are asymptotically normal with the same asymptotic variance as that of the inverse probability weighted estimator with known selection probability function and weights. Also, we establish the mean squared error (MSE) bounds and obtain the MSE convergence rates. A simulation is carried out to assess the proposed estimators in terms of the bias and standard error.     

Influence analysis based on the case sensitivity function

The case sensitivity function approach to influence analysis is introduced as a natural smooth extension of influence curve methodology in which both the insights of geometry and the power of (convex) analysis are available. In it, perturbation is defined as movement between probability vectors defining weighted empirical distributions. A Euclidean geometry is proposed giving such perturbations both size and direction. The notion of the salience of a perturbation is emphasized. This approach has several benefits. A general probability case weight analysis results. Answers to a number of outstanding questions follow directly. Rescaled versions of the three usual finite sample influence curve measures—seen now to be required for comparability across different-sized subsets of cases—are readily available. These new diagnostics directly measure the salience of the (infinitesimal) perturbations involved. Their essential unity, both within and between subsets, is evident geometrically. Finally it is shown how a relaxation strategy, in which a high dimensional ( O ( ⁿC_m )) discrete problem is replaced by a low dimensional ( O ( n )) continuous problem, can combine with (convex) optimization results to deliver better performance in challenging multiple-case influence problems. Further developments are briefly indicated.     

Nonparametric maximum likelihood estimation of population size based on the counting distribution

Summary.  The paper discusses the estimation of an unknown population size n . Suppose that an identification mechanism can identify n _obs cases. The Horvitz–Thompson estimator of n adjusts this number by the inverse of 1− p ₀, where the latter is the probability of not identifying a case. When repeated counts of identifying the same case are available, we can use the counting distribution for estimating p ₀ to solve the problem. Frequently, the Poisson distribution is used and, more recently, mixtures of Poisson distributions. Maximum likelihood estimation is discussed by means of the EM algorithm. For truncated Poisson mixtures, a nested EM algorithm is suggested and illustrated for several application cases. The algorithmic principles are used to show an inequality, stating that the Horvitz–Thompson estimator of n by using the mixed Poisson model is always at least as large as the estimator by using a homogeneous Poisson model. In turn, if the homogeneous Poisson model is misspecified it will, potentially strongly, underestimate the true population size. Examples from various areas illustrate this finding.     

Weighted quantile regression with missing covariates using empirical likelihood

This paper proposes an empirical likelihood-based weighted (ELW) quantile regression approach for estimating the conditional quantiles when some covariates are missing at random. The proposed ELW estimator is computationally simple and achieves semiparametric efficiency if the probability of missingness is correctly specified. The limiting covariance matrix of the ELW estimator can be estimated by a resampling technique, which does not involve nonparametric density estimation or numerical derivatives. Simulation results show that the ELW method works remarkably well in finite samples. A real data example is used to illustrate the proposed ELW method.     

Semiparametric estimation for weighted average derivatives with responses missing at random

When responses are missing at random, we propose a semiparametric direct estimator for the missing probability and density-weighted average derivatives of a general nonparametric multiple regression function. An estimator for the normalized version of the weighted average derivatives is constructed as well using instrumental variables regression. The proposed estimators are computationally simple and asymptotically normal, and provide a solution to the problem of estimating index coefficients of single-index models with responses missing at random. The developed theory generalizes the method of the density-weighted average derivatives estimation of Powell et al. (1989) for the non-missing data case. Monte Carlo simulation studies are conducted to study the performance of the methods.     

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.     

Empirical likelihood weighted composite quantile regression with partially missing covariates

This paper develops a novel weighted composite quantile regression (CQR) method for estimation of a linear model when some covariates are missing at random and the probability for missingness mechanism can be modelled parametrically. By incorporating the unbiased estimating equations of incomplete data into empirical likelihood (EL), we obtain the EL-based weights, and then re-adjust the inverse probability weighted CQR for estimating the vector of regression coefficients. Theoretical results show that the proposed method can achieve semiparametric efficiency if the selection probability function is correctly specified, therefore the EL weighted CQR is more efficient than the inverse probability weighted CQR. Besides, our algorithm is computationally simple and easy to implement. Simulation studies are conducted to examine the finite sample performance of the proposed procedures. Finally, we apply the new method to analyse the US news College data.