Tests of marginal homogeneity and special cases

The analysis of two‐way contingency tables is common in clinical studies. In addition to summary counts and percentages, statistical tests or summary measures are often desired. If the data can be viewed as two categorical measurements on the same experimental unit (matched pair data) then a test of marginal homogeneity may be appropriate. The most common clinical example is the so called ‘shift table’ whereby a quantity is tested for change between two time points. The two principal marginal homogeneity tests are the Stuart Maxwell and Bhapkar tests. At present, SAS software does not compute either test directly (for tables with more than two categories) and a programmatic solution is required. Two examples of programmatic SAS code are found in the current literature. Although accurate in most instances, they fail to produce output for certain tables (‘special cases’). After summarizing the mathematics behind the two tests, a SAS macro is presented, which produces correct output for all tables. Finally, several examples are coded and presented with resultant output. Copyright © 2013 John Wiley & Sons, Ltd.     

Nenparametric Two-Group Classification: Concepts and a SAS-Based Software Package

This article introduces BestClass, a set of SAS macros, available in the mainframe and workstation environment, designed for solving two-group classification problems using a class of recently developed nonparametric classification methods. The criteria used to estimate the classification function are based on either minimizing a function of the absolute deviations from the surface which separates the groups, or directly minimizing a function of the number of misclassified entities in the training sample. The solution techniques used by BestClass to estimate the classification rule use the mathematical programming routines of the SAS/OR software. Recently, a number of research studies have reported that under certain data conditions this class of classification methods can provide more accurate classification results than existing methods, such as Fisher's linear discriminant function and logistic regression. However, these robust classification methods have not yet been implemented in the major statistical packages, and hence are beyond the reach of those statistical analysts who are unfamiliar with mathematical programming techniques. We use a limited simulation experiment and an example to compare and contrast properties of the methods included in Best-Class with existing parametric and nonparametric methods. We believe that BestClass contributes significantly to the field of nonparametric classification analysis, in that it provides the statistical community with convenient access to this recently developed class of methods. BestClass is available from the authors.     

Analysis of means: a generalized approach using R

Papers on the analysis of means (ANOM) have been circulating in the quality control literature for decades, routinely describing it as a statistical stand-alone concept. Therefore, we clarify that ANOM should rather be regarded as a special case of a much more universal approach known as multiple contrast tests (MCTs). Perceiving ANOM as a grand-mean-type MCT paves the way for implementing it in the open-source software R. We give a brief tutorial on how to exploit R's versatility and introduce the R package ANOM for drawing the familiar decision charts. Beyond that, we illustrate two practical aspects of data analysis with ANOM: firstly, we compare merits and drawbacks of ANOM-type MCTs and ANOVA F-test and assess their respective statistical powers, and secondly, we show that the benefit of using critical values from multivariate t-distributions for ANOM instead of simple Bonferroni quantiles is oftentimes negligible.     

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.     

Estimating the reliability of repeatedly measured endpoints based on linear mixed‐effects models. A tutorial

There are various settings in which researchers are interested in the assessment of the correlation between repeated measurements that are taken within the same subject (i.e., reliability). For example, the same rating scale may be used to assess the symptom severity of the same patients by multiple physicians, or the same outcome may be measured repeatedly over time in the same patients. Reliability can be estimated in various ways, for example, using the classical Pearson correlation or the intra‐class correlation in clustered data. However, contemporary data often have a complex structure that goes well beyond the restrictive assumptions that are needed with the more conventional methods to estimate reliability. In the current paper, we propose a general and flexible modeling approach that allows for the derivation of reliability estimates, standard errors, and confidence intervals – appropriately taking hierarchies and covariates in the data into account. Our methodology is developed for continuous outcomes together with covariates of an arbitrary type. The methodology is illustrated in a case study, and a Web Appendix is provided which details the computations using the R package CorrMixed and the SAS software. Copyright © 2016 John Wiley & Sons, Ltd.     

Generalized run tests for statistical process control

ABSTRACT

In a sequence of elements, a run is defined as a maximal subsequence of like elements. The number of runs or the length of the longest run has been widely used to test the randomness of an ordered sequence. Based on two different sampling methods and two types of test statistics used, run tests can be classified into one of four cases. Numerous researchers have derived the probability distributions in many different ways, treating each case separately. In the paper, we propose a unified approach which is based on recurrence arguments of two mutually exclusive sub-sequences. We also consider the sequence of nominal data that has more than two classes. Thus, the traditional run tests for a binary sequence are special cases of our generalized run tests. We finally show that the generalized run tests can be applied to many quality management areas, such as testing changes in process variation, developing non-parametric multivariate control charts, and comparing the shapes and locations of more than two process distributions.     

Parameter estimation of nonlinear mixed-effects models using first-order conditional linearization and the EM algorithm

Nonlinear mixed-effects (NLME) models are flexible enough to handle repeated-measures data from various disciplines. In this article, we propose both maximum-likelihood and restricted maximum-likelihood estimations of NLME models using first-order conditional expansion (FOCE) and the expectation–maximization (EM) algorithm. The FOCE-EM algorithm implemented in the ForStat procedure SNLME is compared with the Lindstrom and Bates (LB) algorithm implemented in both the SAS macro NLINMIX and the S-Plus/R function nlme in terms of computational efficiency and statistical properties. Two realworld data sets an orange tree data set and a Chinese fir (Cunninghamia lanceolata) data set, and a simulated data set were used for evaluation. FOCE-EM converged for all mixed models derived from the base model in the two realworld cases, while LB did not, especially for the models in which random effects are simultaneously considered in several parameters to account for between-subject variation. However, both algorithms had identical estimated parameters and fit statistics for the converged models. We therefore recommend using FOCE-EM in NLME models, particularly when convergence is a concern in model selection.     

Heteroscedasticity diagnostics in two-phase linear regression models

In two-phase linear regression models, it is a standard assumption that the random errors of two phases have constant variances. However, this assumption is not necessarily appropriate. This paper is devoted to the tests for variance heterogeneity in these models. We initially discuss the simultaneous test for variance heterogeneity of two phases. When the simultaneous test shows that significant heteroscedasticity occurs in the whole model, we construct two individual tests to investigate whether or not both phases or one of them have/has significant heteroscedasticity. Several score statistics and their adjustments based on Cox and Reid [D. R. Cox and N. Reid, Parameter orthogonality and approximate conditional inference. J. Roy. Statist. Soc. Ser. B 49 (1987), pp. 1–39] are obtained and illustrated with Australian onion data. The simulated powers of test statistics are investigated through Monte Carlo methods.     

Small-sample likelihood inference in extreme-value regression models

We deal with a general class of extreme-value regression models introduced by Barreto-Souza and Vasconcellos [Bias and skewness in a general extreme-value regression model, Comput. Statist. Data Anal. 55 (2011), pp. 1379–1393]. Our goal is to derive an adjusted likelihood ratio statistic that is approximately distributed as χ² with a high degree of accuracy. Although the adjusted statistic requires more computational effort than its unadjusted counterpart, it is shown that the adjustment term has a simple compact form that can be easily implemented in standard statistical software. Further, we compare the finite-sample performance of the three classical tests (likelihood ratio, Wald, and score), the gradient test that has been recently proposed by Terrell [The gradient statistic, Comput. Sci. Stat. 34 (2002), pp. 206–215], and the adjusted likelihood ratio test obtained in this article. Our simulations favour the latter. Applications of our results are presented.     

Multivariate two‐sample permutation tests for trials with multiple time‐to‐event outcomes

Clinical trials involving multiple time‐to‐event outcomes are increasingly common. In this paper, permutation tests for testing for group differences in multivariate time‐to‐event data are proposed. Unlike other two‐sample tests for multivariate survival data, the proposed tests attain the nominal type I error rate. A simulation study shows that the proposed tests outperform their competitors when the degree of censored observations is sufficiently high. When the degree of censoring is low, it is seen that naive tests such as Hotelling's T² outperform tests tailored to survival data. Computational and practical aspects of the proposed tests are discussed, and their use is illustrated by analyses of three publicly available datasets. Implementations of the proposed tests are available in an accompanying R package.     

%Gra: an SAS macro for generalized redundancy analysis

In the framework of redundancy analysis and reduced rank regression, the extended redundancy analysis model managed to account for more than two blocks of manifest variables in its specification. A further extension, the generalized redundancy analysis (GRA), has been recently proposed in literature, with the aim of incorporating external covariates into the model, thanks to a new estimation algorithm that manages to separate all the contributions of the exogenous and external covariates in the formation of the latent composites. At present, software to estimate GRA models is not available. In this paper, we provide an SAS macro, %GRA, to specify and fit structural relationships, with an application to illustrate the use of the macro.     

Testing the equality of several means when the population variances are unequal

A comparative study is made of three tests, developed by James (1951), Welch (1951) and Brown & Forsythe (1974). James presented two methods of which only one is considered in this paper. It is shown that this method gives better control over the size than the other two tests. None of these methods is uniformly more powerful than the other two. In some cases the tests of James and Welch reject a false null hypothesis more often than the test of Brown & Forsythe, but there are also situations in which it is the other way around.

We conclude that for implementation in a statistical software package the very complicated test of James is the most attractive. A practical disadvantage of this method can be overcome by a minor modification.     

Combining the t test and Wilcoxon's rank-sum test

In the two-sample location-shift problem, Student's t test or Wilcoxon's rank-sum test are commonly applied. The latter test can be more powerful for non-normal data. Here, we propose to combine the two tests within a maximum test. We show that the constructed maximum test controls the type I error rate and has good power characteristics for a variety of distributions; its power is close to that of the more powerful of the two tests. Thus, irrespective of the distribution, the maximum test stabilizes the power. To carry out the maximum test is a more powerful strategy than selecting one of the single tests. The proposed test is applied to data of a clinical trial.     

Levene type tests for the ratio of two scales

Tests for the equality of variances are of interest in many areas such as quality control, agricultural production systems, experimental education, pharmacology, biology, as well as a preliminary to the analysis of variance, dose–response modelling or discriminant analysis. The literature is vast. Traditional non-parametric tests are due to Mood, Miller and Ansari–Bradley. A test which usually stands out in terms of power and robustness against non-normality is the W50 Brown and Forsythe [Robust tests for the equality of variances, J. Am. Stat. Assoc. 69 (1974), pp. 364–367] modification of the Levene test [Robust tests for equality of variances, in Contributions to Probability and Statistics, I. Olkin, ed., Stanford University Press, Stanford, 1960, pp. 278–292]. This paper deals with the two-sample scale problem and in particular with Levene type tests. We consider 10 Levene type tests: the W50, the M50 and L50 tests [G. Pan, On a Levene type test for equality of two variances, J. Stat. Comput. Simul. 63 (1999), pp. 59–71], the R-test [R.G. O'Brien, A general ANOVA method for robust tests of additive models for variances, J. Am. Stat. Assoc. 74 (1979), pp. 877–880], as well as the bootstrap and permutation versions of the W50, L50 and R tests. We consider also the F-test, the modified Fligner and Killeen [Distribution-free two-sample tests for scale, J. Am. Stat. Assoc. 71 (1976), pp. 210–213] test, an adaptive test due to Hall and Padmanabhan [Adaptive inference for the two-sample scale problem, Technometrics 23 (1997), pp. 351–361] and the two tests due to Shoemaker [Tests for differences in dispersion based on quantiles, Am. Stat. 49(2) (1995), pp. 179–182; Interquantile tests for dispersion in skewed distributions, Commun. Stat. Simul. Comput. 28 (1999), pp. 189–205]. The aim is to identify the effective methods for detecting scale differences. Our study is different with respect to the other ones since it is focused on resampling versions of the Levene type tests, and many tests considered here have not ever been proposed and/or compared. The computationally simplest test found robust is W50. Higher power, while preserving robustness, is achieved by considering the resampling version of Levene type tests like the permutation R-test (recommended for normal- and light-tailed distributions) and the bootstrap L50 test (recommended for heavy-tailed and skewed distributions). Among non-Levene type tests, the best one is the adaptive test due to Hall and Padmanabhan.     

Testing Inference in Inflated Beta Regressions under Model Misspecification

We consider testing inference in inflated beta regressions subject to model misspecification. In particular, quasi-z tests based on sandwich covariance matrix estimators are described and their finite sample behavior is investigated via Monte Carlo simulations. The numerical evidence shows that quasi-z testing inference can be considerably more accurate than inference made through the usual z tests, especially when there is model misspecification. Interval estimation is also considered. We also present an empirical application that uses real (not simulated) data.     

Testing regression lack of fit without replication: a tutorial around Minitab's XLOF procedures

The tutorial is concerned with two types of test for the general lack of fit of a linear regression model, as found in the Minitab software package, and which do not require replicated observations. They aim to identify non-specified curvature and interaction in predictors, by comparing fits over the predictor region divided into two parts. Minitab's regression subcommand XLOF which gives the tests is only briefly documented in the manual and, unlike virtually all other statistical procedures in the software, it is not standard and cannot be readily found in textbooks. The two types of test are described here; they concern the predictors one-at-a-time and the predictors all-at-once. An example of their use is given. A suite of macros is available which reproduces the results of the XLOF tests in much more detail than is given by the XLOF subcommand.     

The analysis of multicentre clinical trials when there is heterogeneity between centres

Two-treatment multicentre clinical trials are very common in practice. In cases where a non-parametric analysis is appropriate, a rank-sum test for grouped data called the van Elteren test can be applied. As an alternative approach, one may apply a combination test such as Fisher's combination test or the inverse normal combination test (also called Liptak's method) in order to combine centre-specific P-values. If there are no ties and no differences between centres with regard to the groups’ sample sizes, the inverse normal combination test using centre-specific Wilcoxon rank-sum tests is equivalent to the van Elteren test. In this paper, the van Elteren test is compared with Fisher's combination test based on Wilcoxon rank-sum tests. Data from two multicentre trials as well as simulated data indicate that Fisher's combination of P-values is more powerful than the van Elteren test in realistic scenarios, i.e. when there are large differences between the centres’ P-values, some quantitative interaction between treatment and centre, and/or heterogeneity in variability. The combination approach opens the possibility of using statistics other than the rank sum, and it is also a suitable method for more complicated designs, e.g. when covariates such as age or gender are included in the analysis.     

Hypothesis testing in smoothing spline models

Nonparametric regression models are often used to check or suggest a parametric model. Several methods have been proposed to test the hypothesis of a parametric regression function against an alternative smoothing spline model. Some tests such as the locally most powerful (LMP) test by Cox et al. (Cox, D., Koh, E., Wahba, G. and Yandell, B. (1988). Testing the (parametric) null model hypothesis in (semiparametric) partial and generalized spline models. Ann. Stat., 16, 113–119.), the generalized maximum likelihood (GML) ratio test and the generalized cross validation (GCV) test by Wahba (Wahba, G. (1990). Spline models for observational data. CBMS-NSF Regional Conference Series in Applied Mathematics, SIAM.) were developed from the corresponding Bayesian models. Their frequentist properties have not been studied. We conduct simulations to evaluate and compare finite sample performances. Simulation results show that the performances of these tests depend on the shape of the true function. The LMP and GML tests are more powerful for low frequency functions while the GCV test is more powerful for high frequency functions. For all test statistics, distributions under the null hypothesis are complicated. Computationally intensive Monte Carlo methods can be used to calculate null distributions. We also propose approximations to these null distributions and evaluate their performances by simulations.