Homogeneity test, sample size determination and interval construction of difference of two proportions in stratified bilateral-sample designs

In stratified otolaryngologic (or ophthalmologic) studies, the misleading results may be obtained when ignoring the confounding effect and the correlation between responses from two ears. Score statistic and Wald-type statistic are presented to test equality in a stratified bilateral-sample design, and their corresponding sample size formulae are given. Score statistic for testing homogeneity of difference between two proportions and score confidence interval of the common difference of two proportions in a stratified bilateral-sample design are derived. Empirical results show that (1) score statistic and Wald-type statistic based on dependence model assumption outperform other statistics in terms of the type I error rates; (2) score confidence interval demonstrates reasonably good coverage property; (3) sample size formula via Wald-type statistic under dependence model assumption is rather accurate. A real example is used to illustrate the proposed methodologies.     

Testing equality of correlation coefficients for paired binary data from multiple groups

Paired binary data arise naturally when paired body parts are investigated in clinical trials. One of the widely used models for dealing with this kind of data is the equal correlation coefficients model. Before using this model, it is necessary to test whether the correlation coefficients in each group are actually equal. In this paper, three test statistics (likelihood ratio test, Wald-type test, and Score test) are derived for this purpose. The simulation results show that the Score test statistic maintains type I error rate and has satisfactory power, and therefore is recommended among the three methods. The likelihood ratio test is over conservative in most cases, and the Wald-type statistic is not robust with respect to empirical type I error. Three real examples, including a multi-centre Phase II double-blind placebo randomized controlled trial, are given to illustrate the three proposed test statistics.     

A heteroscedastic structural errors-in-variables model with equation error

It is not uncommon with astrophysical and epidemiological data sets that the variances of the observations are accessible from an analytical treatment of the data collection process. Moreover, in a regression model, heteroscedastic measurement errors and equation errors are common situations when modelling such data. This article deals with the limiting distribution of the maximum-likelihood and method-of-moments estimators for the line parameters of the regression model. We use the delta method to achieve it, making it possible to build joint confidence regions and hypothesis testing. This technique produces closed expressions for the asymptotic covariance matrix of those estimators. In the moment approach we do not assign any distribution for the unobservable covariate while with the maximum-likelihood approach, we assume a normal distribution. We also conduct simulation studies of rejection rates for Wald-type statistics in order to verify the test size and power. Practical applications are reported for a data set produced by the Chandra observatory and also from the WHO MONICA Project on cardiovascular disease.     

On testing homogeneity of variances for nonnormal models using entropy

This paper deals with testing equality of variances of observations in the different treatment groups assuming treatment effects are fixed. We study the distribution of a test statistic which is known to perform comparably well with other statistics for the same purpose under normality. The statistic we consider is based on Shannon’s entropy for a distribution function. We will derive the asymptotic expansion for the distribution of the test statistic based on Shannon’s entropy under nonnormality and numerically examine its performance in comparison with the modified likelihood ratio criteria for normal and some nonnormal populations.      

Statistical analysis of non-inferiority via non-zero risk difference in stratified matched-pair studies

A stratified study is often designed for adjusting several independent trials in modern medical research. We consider the problem of non-inferiority tests and sample size determinations for a nonzero risk difference in stratified matched-pair studies, and develop the likelihood ratio and Wald-type weighted statistics for testing a null hypothesis of non-zero risk difference for each stratum in stratified matched-pair studies on the basis of (1) the sample-based method and (2) the constrained maximum likelihood estimation (CMLE) method. Sample size formulae for the above proposed statistics are derived, and several choices of weights for Wald-type weighted statistics are considered. We evaluate the performance of the proposed tests according to type I error rates and empirical powers via simulation studies. Empirical results show that (1) the likelihood ratio and the Wald-type CMLE test based on harmonic means of the stratum-specific sample size (SSIZE) weight (the Cochran's test) behave satisfactorily in the sense that their significance levels are much closer to the prespecified nominal level; (2) the likelihood ratio test is better than Nam's [2006. Non-inferiority of new procedure to standard procedure in stratified matched-pair design. Biometrical J. 48, 966–977] score test; (3) the sample sizes obtained by using SSIZE weight are smaller than other weighted statistics in general; (4) the Cochran's test statistic is generally much better than other weighted statistics with CMLE method. A real example from a clinical laboratory study is used to illustrate the proposed methodologies.     

Homogeneity Test of Risk Differences of Marginal and Conditional Probabilities in Several Incomplete Correlated 2 × 2 Tables

This article considers K pairs of incomplete correlated 2 × 2 tables in which the interesting measurement is the risk difference between marginal and conditional probabilities. A Wald-type statistic and a score-type statistic are presented to test the homogeneity hypothesis about risk differences across strata. Powers and sample size formulae based on the above two statistics are deduced. Figures about sample size against risk difference (or marginal probability) are given. A real example is used to illustrate the proposed methods.     

Robust tests for the common principal components model

When dealing with several populations, the common principal components (CPC) model assumes equal principal axes but different variances along them. In this paper, a robust log-likelihood ratio statistic allowing to test the null hypothesis of a CPC model versus no restrictions on the scatter matrices is introduced. The proposal plugs into the classical log-likelihood ratio statistic robust scatter estimators. Using the same idea, a robust log-likelihood ratio and a robust Wald-type statistic for testing proportionality against a CPC model are considered. Their asymptotic distributions under the null hypothesis and their partial influence functions are derived. A small simulation study allows to compare the behavior of the classical and robust tests, under normal and contaminated data.     

Performance of confidence interval tests for the ratio of two lognormal means applied to Weibull and gamma distribution data

Five estimation approaches have been developed to compute the confidence interval (CI) for the ratio of two lognormal means: (1) T, the CI based on the t-test procedure; (2) ML, a traditional maximum likelihood-based approach; (3) BT, a bootstrap approach; (4) R, the signed log-likelihood ratio statistic; and (5) R*, the modified signed log-likelihood ratio statistic. The purpose of this study was to assess the performance of these five approaches when applied to distributions other than lognormal distribution, for which they were derived. Performance was assessed in terms of average length and coverage probability of the CIs for each estimation approaches (i.e., T, ML, BT, R, and R*) when data followed a Weibull or gamma distribution. Four models were discussed in this study. In Model 1, the sample sizes and variances were equal within the two groups. In Model 2, the sample sizes were equal but variances were different within the two groups. In Model 3, the variances were different within the two groups and the larger variance was paired with the larger sample size. In Model 4, the variances were different within the two groups and the larger variance was paired with the smaller sample size. The results showed that when the variances of the two groups were equal, the t-test performed well, no matter what the underlying distribution was and how large the variances of the two groups were. The BT approach performed better than the others when the underlying distribution was not lognormal distribution, although it was inaccurate when the variances were large. The R* test did not perform well when the underlying distribution was Weibull or gamma distributed data, but it performed best when the data followed a lognormal distribution.     

Diagnosing explainable heterogeneity of variance in random‐effects models

Data‐analytic tools for models other than the normal linear regression model are relatively rare. Here we develop plots and diagnostic statistics for nonconstant variance for the random‐effects model (REM). REMs for longitudinal data include both within‐ and between‐subject variances. A basic assumption is that the two variance terms are constant across subjects. However, we often find that these variances are functions of covariates, and the data set has what we call explainable heterogeneity, which needs to be allowed for in the model. We characterize several types of heterogeneity of variance in REMs and develop three diagnostic tests using the score statistic: one for each of the two variance terms, and the third for a form of multivariate nonconstant variance. For each test we present an adjusted residual plot which can identify cases that are unusually influential on the outcome of the test.     

Skew-normal factor analysis models with incomplete data

Traditional factor analysis (FA) rests on the assumption of multivariate normality. However, in some practical situations, the data do not meet this assumption; thus, the statistical inference made from such data may be misleading. This paper aims at providing some new tools for the skew-normal (SN) FA model when missing values occur in the data. In such a model, the latent factors are assumed to follow a restricted version of multivariate SN distribution with additional shape parameters for accommodating skewness. We develop an analytically feasible expectation conditional maximization algorithm for carrying out parameter estimation and imputation of missing values under missing at random mechanisms. The practical utility of the proposed methodology is illustrated with two real data examples and the results are compared with those obtained from the traditional FA counterparts.     

Testing the rate ratio under inverse sampling based on gradient statistic

Inverse sampling is widely applied in studies with dichotomous outcomes, especially when the subjects arrive sequentially or the response of interest is difficult to obtain. In this paper, we investigate the rate ratio test problem under inverse sampling based on gradient statistic with the asymptotic method and parametric bootstrap technique. The gradient statistic has many advantages, for example, it is simple to calculate and competitive with Wald-type, score and likelihood ratio tests in terms of local power. Numerical studies are carried out to evaluate the performance of our gradient test and the existing tests, namely Wald-type, score and likelihood ratio tests. The simulation results suggest that the gradient test based on the parametric bootstrap method has excellent type I error control and large powers even in small sample design. Two real examples, from a heart disease study and a drug comparison study, are applied to illustrate our methods.     

An Optimal Semiparametric Method for Two‐group Classification

In the classical discriminant analysis, when two multivariate normal distributions with equal variance–covariance matrices are assumed for two groups, the classical linear discriminant function is optimal with respect to maximizing the standardized difference between the means of two groups. However, for a typical case‐control study, the distributional assumption for the case group often needs to be relaxed in practice. Komori et al. (Generalized t ‐statistic for two‐group classification. Biometrics 2015, 71: 404–416) proposed the generalized t ‐statistic to obtain a linear discriminant function, which allows for heterogeneity of case group. Their procedure has an optimality property in the class of consideration. We perform a further study of the problem and show that additional improvement is achievable. The approach we propose does not require a parametric distributional assumption on the case group. We further show that the new estimator is efficient, in that no further improvement is possible to construct the linear discriminant function more efficiently. We conduct simulation studies and real data examples to illustrate the finite sample performance and the gain that it produces in comparison with existing methods.     

Methods to test for equality of two normal distributions

Statistical tests for two independent samples under the assumption of normality are applied routinely by most practitioners of statistics. Likewise, presumably each introductory course in statistics treats some statistical procedures for two independent normal samples. Often, the classical two-sample model with equal variances is introduced, emphasizing that a test for equality of the expected values is a test for equality of both distributions as well, which is the actual goal. In a second step, usually the assumption of equal variances is discarded. The two-sample t test with Welch correction and the F test for equality of variances are introduced. The first test is solely treated as a test for the equality of central location, as well as the second as a test for the equality of scatter. Typically, there is no discussion if and to which extent testing for equality of the underlying normal distributions is possible, which is quite unsatisfactorily regarding the motivation and treatment of the situation with equal variances. It is the aim of this article to investigate the problem of testing for equality of two normal distributions, and to do so using knowledge and methods adequate to statistical practitioners as well as to students in an introductory statistics course. The power of the different tests discussed in the article is examined empirically. Finally, we apply the tests to several real data sets to illustrate their performance. In particular, we consider several data sets arising from intelligence tests since there is a large body of research supporting the existence of sex differences in mean scores or in variability in specific cognitive abilities.     

Some Properties of the Pivotal Statistic Based on the Asymptotically Distribution-Free Theory in Structural Equation Modeling

For normally distributed data, the asymptotic bias and skewness of the pivotal statistic Studentized by the asymptotically distribution-free standard error are shown to be the same as those given by the normal theory in structural equation modeling. This gives the same asymptotic null distributions of the two pivotal statistics up to the next order beyond the usual normal approximation under normality. With an alternative hypothesis, the asymptotic variances of the two statistics under normality/non normality are also derived. It is, however, shown that the asymptotic variances of the non null distributions of the statistics are generally different even under normality.     

Nonparametric tests for right-censored data with biased sampling

Testing the equality of two survival distributions can be difficult in a prevalent cohort study when non random sampling of subjects is involved. Due to the biased sampling scheme, independent censoring assumption is often violated. Although the issues about biased inference caused by length-biased sampling have been widely recognized in statistical, epidemiological and economical literature, there is no satisfactory solution for efficient two-sample testing. We propose an asymptotic most efficient nonparametric test by properly adjusting for length-biased sampling. The test statistic is derived from a full likelihood function, and can be generalized from the two-sample test to a k-sample test. The asymptotic properties of the test statistic under the null hypothesis are derived using its asymptotic independent and identically distributed representation. We conduct extensive Monte Carlo simulations to evaluate the performance of the proposed test statistics and compare them with the conditional test and the standard logrank test for different biased sampling schemes and right-censoring mechanisms. For length-biased data, empirical studies demonstrated that the proposed test is substantially more powerful than the existing methods. For general left-truncated data, the proposed test is robust, still maintains accurate control of type I error rate, and is also more powerful than the existing methods, if the truncation patterns and right-censoring patterns are the same between the groups. We illustrate the methods using two real data examples.     

Improvement of the quality of the chi-square approximation for the ADF test on a covariance matrix with a linear structure

The asymptotically distribution-free (ADF) test statistic was proposed by Browne (1984). It is known that the null distribution of the ADF test statistic is asymptotically distributed according to the chi-square distribution. This asymptotic property is always satisfied, even under nonnormality, although the null distributions of other famous test statistics, e.g., the maximum likelihood test statistic and the generalized least square test statistic, do not converge to the chi-square distribution under nonnormality. However, many authors have reported numerical results which indicate that the quality of the chi-square approximation for the ADF test is very poor, even when the sample size is large and the population distribution is normal. In this paper, we try to improve the quality of the chi-square approximation to the ADF test for a covariance matrix with a linear structure by using the Bartlett correction applicable under the assumption of normality. By conducting numerical studies, we verify that the obtained Bartlett correction can perform well even when the assumption of normality is violated.     

A Wald-type test statistic based on robust modified median estimator in logistic regression models

In this paper a new robust estimator, modified median estimator, is introduced and studied for the logistic regression model. This estimator is based on the median estimator considered in Hobza et al. [Robust median estimator in logistic regression. J Stat Plan Inference. 2008;138:3822–3840]. Its asymptotic distribution is obtained. Using the modified median estimator, we also consider a Wald-type test statistic for testing linear hypotheses in the logistic regression model and we obtain its asymptotic distribution under the assumption of random regressors. An extensive simulation study is presented in order to analyse the efficiency as well as the robustness of the modified median estimator and Wald-type test based on it.     

A class of goodness-of-fit test for the additive hazards model with case-cohort data

The case-cohort design is commonly used in epidemiological studies due to its cost-effectiveness. The additive hazards model is widely used in survival analysis when the hazards difference is constant. In this article, we propose a class of goodness-of-fit test statistics for the assumption of the additive hazards model with case-cohort data through a class of asymptotically mean-zero multiparameter stochastic processes. We also establish the asymptotic theory of the proposed test statistics and a resampling scheme is adopted to approximate its asymptotic distribution. The performance of the proposed test statistics is evaluated through simulation studies and a real dataset is analyzed to illustrate the proposed method.     

Testing the Equality of Two Proportions for Combined Unilateral and Bilateral Data

In many medical comparative studies (e.g., comparison of two treatments in an otolaryngological study), subjects may produce either bilateral (e.g., responses from a pair of ears) or unilateral (response from only one ear) data. For bilateral cases, it is meaningful to assume that the information between the two ears from the same subject are generally highly correlated. In this article, we would like to test the equality of the successful cure rates between two treatments with the presence of combined unilateral and bilateral data. Based on the dependence and independence models, we study ten test statistics which utilize both the unilateral and bilateral data. The performance of these statistics will be evaluated with respect to their empirical Type I error rates and powers under different configurations. We find that both Rosner's and Wald-type statistics based on the dependence model and constrained maximum likelihood estimates (under the null hypothesis) perform satisfactorily for small to large samples and are hence recommended. We illustrate our methodologies with a real data set from an otolaryngology study.     

Linearly admissible estimators of mean vector with respect to balanced loss function in multivariate statistics

The authors discuss the bias of the estimate of the variance of the overall effect synthesized from individual studies by using the variance weighted method. This bias is proven to be negative. Furthermore, the conditions, the likelihood of underestimation and the bias from this conventional estimate are studied based on the assumption that the estimates of the effect are subject to normal distribution with common mean. The likelihood of underestimation is very high (e.g. it is greater than 85% when the sample sizes in two combined studies are less than 120). The alternative less biased estimates for the cases with and without the homogeneity of the variances are given in order to adjust for the sample size and the variation of the population variance. In addition, the sample size weight method is suggested if the consistence of the sample variances is violated Finally, a real example is presented to show the difference by using the above three estimate methods.