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.     

Inference on effect size indices from several two-armed experiments

The statistical inference problem on effect size indices is addressed using a series of independent two-armed experiments from k arbitrary populations. The effect size parameter simply quantifies the difference between two groups. It is a meaningful index to be used when data are measured on different scales. In the context of bivariate statistical models, we define estimators of the effect size indices and propose large sample testing procedures to test the homogeneity of these indices. The null and non-null distributions of the proposed testing procedures are derived and their performance is evaluated via Monte Carlo simulation. Further, three types of interval estimation of the proposed indices are considered for both combined and uncombined data. Lower and upper confidence limits for the actual effect size indices are obtained and compared via bootstrapping. It is found that the length of the intervals based on the combined effect size estimator are almost half the length of the intervals based on the uncombined effect size estimators. Finally, we illustrate the proposed procedures for hypothesis testing and interval estimation using a real data set.     

An exact test for comparing two predictive values in small‐size clinical trials

Positive and negative predictive values describe the performance of a diagnostic test. There are several methods to test the equality of predictive values in paired designs. However, these methods were premised on large sample theory, and they may not be suitable for small‐size clinical trials because of inflation of the type 1 error rate. In this study, we propose an exact test to control the type 1 error rate strictly for conducting a small‐size clinical trial that investigates the equality of predictive values in paired designs. In addition, we execute simulation studies to evaluate the performance of the proposed exact test and existing methods in small‐size clinical trials. The proposed test can calculate the exact P value, and as a result of simulations, the empirical type 1 error rate for the proposed test did not exceed the significance level regardless of the setting, and the empirical power for the proposed test is not much different from the other methods based on large‐sample theory. Therefore, it is considered that the proposed exact test is useful when the type 1 error rate needs to be controlled strictly.     

C55. Quick and accurate rule of thumb for determining the critical value of the coefficient of correlation

Life table analysis techniques in epidemiology depend upon the asymptotic properties of the statistical test methods employed. In some instances, the statistical procedures indicate highly significant results which are, in reality, unjustified. The phenomenon may occur when the asymptotic methods are applied in situations where the cases of interest are few in number. This situation is illustrated by the 20 multiple myeloma deaths observed in the RERF Life Span Study cohort. A permutation test is applied to the life table data, although the test requires the false assumption that the censoring distribution is independent of the radiation dose. A simulation test is developed which does not require equal censoring, which has the same asymptotics as the usual test methods, and which is less likely to overestimate significance in small samples. It is found that both of these small-sample tests provide reasonable numerical solutions. In addition, the simulation test is recommended in general for analyzing life table data with unequal censoring. Finally, by using the small-sample tests, the frequency of death from multiple myeloma is shown to be positively associated with radiation dose (P<0.01).     

A comparison of several adaptive tests for paired data

In the last few years, two adaptive tests for paired data have been proposed. One test proposed by Freidlin et al. [On the use of the Shapiro–Wilk test in two-stage adaptive inference for paired data from moderate to very heavy tailed distributions, Biom. J. 45 (2003), pp. 887–900] is a two-stage procedure that uses a selection statistic to determine which of three rank scores to use in the computation of the test statistic. Another statistic, proposed by O'Gorman [Applied Adaptive Statistical Methods: Tests of Significance and Confidence Intervals, Society for Industrial and Applied Mathematics, Philadelphia, 2004], uses a weighted t-test with the weights determined by the data. These two methods, and an earlier rank-based adaptive test proposed by Randles and Hogg [Adaptive Distribution-free Tests, Commun. Stat. 2 (1973), pp. 337–356], are compared with the t-test and to Wilcoxon's signed-rank test. For sample sizes between 15 and 50, the results show that the adaptive test proposed by Freidlin et al. and the adaptive test proposed by O'Gorman have higher power than the other tests over a range of moderate to long-tailed symmetric distributions. The results also show that the test proposed by O'Gorman has greater power than the other tests for short-tailed distributions. For sample sizes greater than 50 and for small sample sizes the adaptive test proposed by O'Gorman has the highest power for most distributions.     

An extensive power evaluation of a novel two-sample density-based empirical likelihood ratio test for paired data with an application to a treatment study of attention-deficit/hyperactivity disorder and severe mood dysregulation

In many case-control studies, it is common to utilize paired data when treatments are being evaluated. In this article, we propose and examine an efficient distribution-free test to compare two independent samples, where each is based on paired observations. We extend and modify the density-based empirical likelihood ratio test presented by Gurevich and Vexler [7] to formulate an appropriate parametric likelihood ratio test statistic corresponding to the hypothesis of our interest and then to approximate the test statistic nonparametrically. We conduct an extensive Monte Carlo study to evaluate the proposed test. The results of the performed simulation study demonstrate the robustness of the proposed test with respect to values of test parameters. Furthermore, an extensive power analysis via Monte Carlo simulations confirms that the proposed method outperforms the classical and general procedures in most cases related to a wide class of alternatives. An application to a real paired data study illustrates that the proposed test can be efficiently implemented in practice.     

Simultaneous confidence interval construction for many-to-one comparisons of proportion differences based on correlated paired data

In some medical researches such as ophthalmological, orthopaedic and otolaryngologic studies, it is often of interest to compare multiple groups with a control using data collected from paired organs of patients. The major difficulty in performing the data analysis is to adjust the multiplicity between the comparison of multiple groups, and the correlation within the same patient''s paired organs. In this article, we construct asymptotic simultaneous confidence intervals (SCIs) for many-to-one comparisons of proportion differences adjusting for multiplicity and the correlation. The coverage probabilities and widths of the proposed CIs are evaluated by Monte Carlo simulation studies. The methods are illustrated by a real data example.     

A Cautionary Note on the Analysis of Extreme Data with Cox Regression

This article analyzes a small censored data set to demonstrate the potential dangers of using statistical computing packages without understanding the details of statistical methods. The data, consisting of censored response times with heavy ties in one time point, were analyzed with a Cox regression model utilizing SAS PHREG and BMDP2L procedures. The p values, reported from both SAS PHREG and BMDP2L procedures, for testing the equality of two treatments vary considerably. This article illustrates that (1) the Breslow likelihood used in both BMDP2L and SAS PHREG procedures is too conservative and can have a critical effect on an extreme data set, (2) Wald's test in the SAS PHREG procedure may yield absurd results from most likelihood models, and (3) BMDP2L needs to include more than just the Breslow likelihood in future development.     

Positive quadrant dependence tests for copulas

In this paper the interest is in testing the null hypothesis of positive quadrant dependence (PQD) between two random variables. Such a testing problem is important since prior knowledge of PQD is a qualitative restriction that should be taken into account in further statistical analysis, for example, when choosing an appropriate copula function to model the dependence structure. The key methodology of the proposed testing procedures consists of evaluating a “distance” between a nonparametric estimator of a copula and the independence copula, which serves as a reference case in the whole set of copulas having the PQD property. Choices of appropriate distances and nonparametric estimators of copula are discussed, and the proposed methods are compared with testing procedures based on bootstrap and multiplier techniques. The consistency of the testing procedures is established. In a simulation study the authors investigate the finite sample size and power performances of three types of test statistics, Kolmogorov–Smirnov, Cramér–von‐Mises, and Anderson–Darling statistics, together with several nonparametric estimators of a copula, including recently developed kernel type estimators. Finally, they apply the testing procedures on some real data. The Canadian Journal of Statistics 38: 555–581; 2010 © 2010 Statistical Society of Canada     

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.     

Testing for monotonic trend in time series based on resampling methods

In this paper, we propose several tests for monotonic trend based on the Brillinger's test statistic (1989, Biometrika, 76, 23–30). When there are highly correlated residuals or short record lengths, Brillinger's test procedure tends to have significance level much higher than the nominal level. It is found that this could be related to the discrepancy between the empirical distribution of the test statistic and the asymptotic normal distribution. Hence, in this paper, we propose three bootstrap-based procedures based on the Brillinger's test statistic to test for monotonic trend. The performance of the proposed test procedures is evaluated through an extensive Monte Carlo simulation study, and is compared to other trend test procedures in the literature. It is shown that the proposed bootstrap-based Brillinger test procedures can well control the significance levels and provide satisfactory power performance in testing the monotonic trend under different scenarios.     

基于平均自下而上时间的两种分类方法的比较

内容提要：诸如疾病分类系统的预后预测和分类方法,常可用于帮助进行临床管理决策。同一疾病总体常可得到多种分类方法,因此有必要比较这些方法以确定最优分类,或者寻找不逊于最优分类的替代方法。本文基于约束平均寿命引入分离度指标来度量分类方法的预后分类效率,这个指标可用来比较以生存时间为结局的两种分类方法的功效,特别是用于非劣性和等效性检验。我们给出了基于配对数据的两个分离度的估计与检验方法。模拟结果提示,检验方法在适当的样本量条件下能够控制第一类错误,两个实例表明在医学临床中的应用。     

Estimating and testing effect size from an arbitrary population

In this article, we develop inference tools for an effect size parameter in a paired experiment. A class of estimators is defined that includes natural, shrinkage and shrinkage preliminary test estimators. The shrinkage and preliminary test methods incorporate uncertain prior information on the parameter. This information may be available in the form of a realistic guess on the basis of the experimenter’s knowledge and experience, which can be incorporated into the estimation process to increase the efficiency of the estimator. Asymptotic properties of the proposed estimators are investigated both analytically and computationally. A simulation study is also conducted to assess the performance of the estimators for moderate and large samples. For illustration purposes, the method is applied to a data set.     

Testing equality of variances for several normal populations

In this study, we develop a test based on computational approach for the equality of variances of several normal populations. The proposed method is numerically compared with the existing methods. The numeric results demonstrate that the proposed method performs very well in terms of type I error rate and power of test. Furthermore we study the robustness of the tests by using simulation study when the underlying data are from t, exponential and uniform distributions. Finally we analyze a real dataset that motivated our study using the proposed test.     

Tests for a simple tree order restriction with application to dose–response studies

Summary.  We propose 'Dunnett-type' test procedures to test for simple tree order restrictions on the means of p independent normal populations. The new tests are based on the estimation procedures that were introduced by Hwang and Peddada and later by Dunbar, Conaway and Peddada. The procedures proposed are also extended to test for 'two-sided' simple tree order restrictions. For non-normal data, nonparametric versions based on ranked data are also suggested. Using computer simulations, we compare the proposed test procedures with some existing test procedures in terms of size and power. Our simulation study suggests that the procedures compete well with the existing procedures for both one-sided and two-sided simple tree alternatives. In some instances, especially in the case of two-sided alternatives or for non-normally distributed data, the gains in power due to the procedures proposed can be substantial.     

Rapid penalized likelihood-based outlier detection via heteroskedasticity test

Outlier detection is fundamental to statistical modelling. When there are multiple outliers, many traditional approaches in use are stepwise detection procedures, which can be computationally expensive and ignore stochastic error in the outlier detection process. Outlier detection can be performed by a heteroskedasticity test. In this article, a rapid outlier detection method via multiple heteroskedasticity test based on penalized likelihood approaches is proposed to handle these kinds of problems. The proposed method detects the heteroskedasticity of all data only by one step and estimate coefficients simultaneously. The proposed approach is distinguished from others in that a rapid modelling approach uses a weighted least squares formulation coupled with nonconvex sparsity-including penalization. Furthermore, the proposed approach does not need to construct test statistics and calculate their distributions. A new algorithm is proposed for optimizing penalized likelihood functions. Favourable theoretical properties of the proposed approach are obtained. Our simulation studies and real data analysis show that the newly proposed methods compare favourably with other traditional outlier detection techniques.     

Notes on Kolassa’s Method for Estimating the Power of Wilcoxon–Mann–Whitney Test

The Kolassa method implemented in the nQuery Advisor software has been widely used for approximating the power of the Wilcoxon–Mann–Whitney (WMW) test for ordered categorical data, in which Edgeworth approximation is used to estimate the power of an unconditional test based on the WMW U statistic. When the sample size is small or when the sizes in the two groups are unequal, Kolassa’s method may yield quite poor approximation to the power of the conditional WMW test that is commonly implemented in statistical packages. Two modifications of Kolassa’s formula are proposed and assessed by simulation studies.     

Goodness‐of‐fit tests for distributions estimated from complex survey data

We propose nonparametric procedures for comparing the empirical distribution function of data from a complex survey with a hypothesized parametric reference distribution. The hypothesized distribution may be fully specified, or it may be a family with the parameters to be estimated from the data. Of the procedures studied, a modification of the Cramér–von Mises test proposed by Lockhart, Spinelli & Stephens [Lockhart, Spinelli and Stephens, The Canadian Journal of Statistics 2007; 35, 125–133] is supported theoretically and performs well in two simulation studies. The methods are applied to examine the distribution of body mass index in the U.S. National Health and Nutrition Examination Survey. The Canadian Journal of Statistics 47: 409–425; 2019 © 2019 Statistical Society of Canada     

Notes on Test Homogeneity of the Odds Ratio in Matched Pairs Under Stratified Sampling: A Monte Carlo Evaluation

Using Monte Carlo simulation, we compare the performance of five asymptotic test procedures and a randomized permutation test procedure for testing the homogeneity of odds ratio under the stratified matched-pair design. We note that the weighted-least-square test procedure is liberal, while Pearson's goodness-of-fit (PGF) test procedure with the continuity correction is conservative. We note that PGF without the continuity correction, the conditional likelihood ratio test procedure, and the randomized permutation test procedure can generally perform well with respect to Type I error. We use the data taken from a case–control study regarding the endometrial cancer incidence published elsewhere to illustrate the use of these test procedures.     

Efficient tests for one sample correlated binary data with applications

Four testing procedures are considered for testing the response rate of one sample correlated binary data with a cluster size of one or two, which often occurs in otolaryngologic and ophthalmologic studies. Although an asymptotic approach is often used for statistical inference, it is criticized for unsatisfactory type I error control in small sample settings. An alternative to the asymptotic approach is an unconditional approach. The first unconditional approach is the one based on estimation, also known as parametric bootstrap (Lee and Young in Stat Probab Lett 71(2):143–153, 2005). The other two unconditional approaches considered in this article are an approach based on maximization (Basu in J Am Stat Assoc 72(358):355–366, 1977), and an approach based on estimation and maximization (Lloyd in Biometrics 64(3):716–723, 2008a). These two unconditional approaches guarantee the test size and are generally more reliable than the asymptotic approach. We compare these four approaches in conjunction with a test proposed by Lee and Dubin (Stat Med 13(12):1241–1252, 1994) and a likelihood ratio test derived in this article, in regards to type I error rate and power for sample sizes from small to medium. An example from an otolaryngologic study is provided to illustrate the various testing procedures. The unconditional approach based on estimation and maximization using the test in Lee and Dubin (Stat Med 13(12):1241–1252, 1994) is preferable due to the power advantageous.