Large sample results for tests of association ii. multinomial and stratified sampling

This paper deals with the asymptotics of a class of tests for association in 2-way contingency tables based on square forms in cell frequencies, given the total number of observations (multinomial sampling) or one set of marginal totals (stratified sampling). The case when both row and column marginal totals are fixed (hypergeometric sampling) was studied in Kulinskaya (1994), The class of tests under consideration includes a number of classical measures for association, Its two subclasses are the tests based on statistics using centralized cell frequencies (asymptotically distributed as weighted sums of central chi-squares) and those using the non-centralized cell frequencies (asymptotically normal). The parameters of asymptotic distributions depend on the sampling model and on true marginal probabilities. Maximum efficiency for asymptotically normal statistics is achieved under hypergeometric sampling, If the cell frequencies or the statistic as a whole are centralized using marginal proportions as estimates for marginal probabilities, the asymptotic distribution does not differ much between models and it is equivalent to that under hypergeometric sampling. These findings give an extra justification for the use of permutation tests for association (which are based on hypergeometric sampling). As an application, several well known measures of association are analysed.     

Log-linear models for correlated marginal totals of a contingency table

The marginal totals of a contingency table can be rearranged to form a new table. If at least twoof these totals include the same cell of the original table, the new table cannot be treated as anordinary contingency table. An iterative method is proposed to calculate maximum likelihood estimators for the expected cell frequencies of the original table under the assumption that some marginal totals (or more generally, some linear functions) of these expected frequencies satisfy a log-linear model.In some cases, a table of correlated marginal totals is treated as if it was an ordinary contingency table. The effects of ignoring the special structure of the marginal table on thedistributionof the goodness-of-fit test statistics are discussed and illustrated, with special reference to stationary Markov chains.     

Exact Poly-K Test

For animal carcinogenicity study with multiple dose groups, positive trend test and pairwise comparisons of treated groups with control are generally performed using the Cochran-Armitage, Peto test, or Poly-K test. These tests are asymptotically normal. The exact version of Cochran-Armitage and Peto tests are available based on the permutation test assuming fixed column and row totals. For Poly-K test column totals depend on the mortality pattern of the animals and can not be kept fixed over the permutations of the animals. In this work a modification of the permutation test is suggested that can be applied on exact Poly-K test.     

A comparison of some conditional and unconditional exact tests for 2x2 contingency tables

In 1935, R.A. Fisher published his well-known “exact” test for 2x2 contingency tables. This test is based on the conditional distribution of a cell entry when the rows and columns marginal totals are held fixed. Tocher (1950) and Lehmann (1959) showed that Fisher s test, when supplemented by randomization, is uniformly most powerful among all the unbiased tests UMPU). However, since all the practical tests for 2x2 tables are nonrandomized - and therefore biased the UMPU test is not necessarily more powerful than other tests of the same or lower size. Inthis work, the two-sided Fisher exact test and the UMPU test are compared with six nonrandomized unconditional exact tests with respect to their power. In both the two-binomial and double dichotomy models, the UMPU test is often less powerful than some of the unconditional tests of the same (or even lower) size. Thus, the assertion that the Tocher-Lehmann modification of Fisher's conditional test is the optimal test for 2x2 tables is unjustified.     

Generalized Mann–Whitney Type Tests for Microarray Experiments

New statistical procedures are introduced to analyse typical microRNA expression data sets. For each separate microRNA expression, the null hypothesis to be tested is that there is no difference between the distributions of the expression in different groups. The test statistics are then constructed having certain type of alternatives in mind. To avoid strong (parametric) distributional assumptions, the alternatives are formulated using probabilities of different orders of pairs or triples of observations coming from different groups, and the test statistics are then constructed using corresponding several‐sample U‐statistics, natural estimates of these probabilities. Classical several‐sample rank test statistics, such as the Kruskal–Wallis and Jonckheere–Terpstra tests, are special cases in our approach. Also, as the number of variables (microRNAs) is huge, we confront a serious simultaneous testing problem. Different approaches to control the family‐wise error rate or the false discovery rate are shortly discussed, and it is shown how the Chen–Stein theorem can be used to show that family‐wise error rate can be controlled for cluster‐dependent microRNAs under weak assumptions. The theory is illustrated with an analysis of real data, a microRNA expression data set on Finnish (aggressive and non‐aggressive) prostate cancer patients and their controls.     

Some solutions to the multivariate Behrens–Fisher problem for dissimilarity‐based analyses

The essence of the generalised multivariate Behrens–Fisher problem (BFP) is how to test the null hypothesis of equality of mean vectors for two or more populations when their dispersion matrices differ. Solutions to the BFP usually assume variables are multivariate normal and do not handle high‐dimensional data. In ecology, species' count data are often high‐dimensional, non‐normal and heterogeneous. Also, interest lies in analysing compositional dissimilarities among whole communities in non‐Euclidean (semi‐metric or non‐metric) multivariate space. Hence, dissimilarity‐based tests by permutation (e.g., PERMANOVA, ANOSIM) are used to detect differences among groups of multivariate samples. Such tests are not robust, however, to heterogeneity of dispersions in the space of the chosen dissimilarity measure, most conspicuously for unbalanced designs. Here, we propose a modification to the PERMANOVA test statistic, coupled with either permutation or bootstrap resampling methods, as a solution to the BFP for dissimilarity‐based tests. Empirical simulations demonstrate that the type I error remains close to nominal significance levels under classical scenarios known to cause problems for the un‐modified test. Furthermore, the permutation approach is found to be more powerful than the (more conservative) bootstrap for detecting changes in community structure for real ecological datasets. The utility of the approach is shown through analysis of 809 species of benthic soft‐sediment invertebrates from 101 sites in five areas spanning 1960 km along the Norwegian continental shelf, based on the Jaccard dissimilarity measure.     

Editor's Report

There are two common methods for statistical inference on 2 × 2 contingency tables. One is the widely taught Pearson chi-square test, which uses the well-known χ²statistic. The chi-square test is appropriate for large sample inference, and it is equivalent to the Z-test that uses the difference between the two sample proportions for the 2 × 2 case. Another method is Fisher’s exact test, which evaluates the likelihood of each table with the same marginal totals. This article mathematically justifies that these two methods for determining extreme do not completely agree with each other. Our analysis obtains one-sided and two-sided conditions under which a disagreement in determining extreme between the two tests could occur. We also address the question whether or not their discrepancy in determining extreme would make them draw different conclusions when testing homogeneity or independence. Our examination of the two tests casts light on which test should be trusted when the two tests draw different conclusions.     

Effective directed tests for models with ordered categorical data

This paper offers a new method for testing one‐sided hypotheses in discrete multivariate data models. One‐sided alternatives mean that there are restrictions on the multidimensional parameter space. The focus is on models dealing with ordered categorical data. In particular, applications are concerned with R×C contingency tables. The method has advantages over other general approaches. All tests are exact in the sense that no large sample theory or large sample distribution theory is required. Testing is unconditional although its execution is done conditionally, section by section, where a section is determined by marginal totals. This eliminates any potential nuisance parameter issues. The power of the tests is more robust than the power of the typical linear tests often recommended. Furthermore, computer programs are available to carry out the tests efficiently regardless of the sample sizes or the order of the contingency tables. Both censored data and uncensored data models are discussed.     

Shift in re‐randomization distribution with conditional randomization test

Proschan, Brittain, and Kammerman made a very interesting observation that for some examples of the unequal allocation minimization, the mean of the unconditional randomization distribution is shifted away from 0. Kuznetsova and Tymofyeyev linked this phenomenon to the variations in the allocation ratio from allocation to allocation in the examples considered in the paper by Proschan et al. and advocated the use of unequal allocation procedures that preserve the allocation ratio at every step. In this paper, we show that the shift phenomenon extends to very common settings: using conditional randomization test in a study with equal allocation. This phenomenon has the same cause: variations in the allocation ratio among the allocation sequences in the conditional reference set, not previously noted. We consider two kinds of conditional randomization tests. The first kind is the often used randomization test that conditions on the treatment group totals; we describe the variations in the conditional allocation ratio with this test on examples of permuted block randomization and biased coin randomization. The second kind is the randomization test proposed by Zheng and Zelen for a multicenter trial with permuted block central allocation that conditions on the within‐center treatment totals. On the basis of the sequence of conditional allocation ratios, we derive the value of the shift in the conditional randomization distribution for specific vector of responses and the expected value of the shift when responses are independent identically distributed random variables. We discuss the asymptotic behavior of the shift for the two types of tests. Copyright © 2013 John Wiley & Sons, Ltd.     

SOME ISSUES ARISING FROM THE ANALYSIS OF 2×2 CONTINGENCY TABLES

We review the 40 year old controversy over the correct analysis of 2×2 tables. It is argued that conditioning on all marginal totals is appropriate by examining the likelihood based on these margins and secondly by comparing conditional and unconditional evaluations of certain specific outcomes. Several foundational issues which naturally arise are also discussed.     

A modified Mack–Wolfe test for the umbrella alternative problem

ABSTRACT

The Mack–Wolfe test is the most frequently used non parametric procedure for the umbrella alternative problem. In this paper, modifications of the Mack–Wolfe test are proposed for both known peak and unknown peak umbrellas. The exact mean and variance of the proposed tests in the null hypothesis are also derived. We compare these tests with some of the existing tests in terms of the type I error rate and power. In addition, a real data example is presented.     

Parametric bootstrap goodness-of-fit testing for Wehrly–Johnson bivariate circular distributions

The Wehrly–Johnson family of bivariate circular distributions is by far the most general one currently available for modelling data on the torus. It allows complete freedom in the specification of the marginal circular densities as well as the binding circular density which regulates any dependence that might exist between them. We propose a parametric bootstrap approach for testing the goodness-of-fit of Wehrly–Johnson distributions when the forms of their marginal and binding densities are assumed known. The approach admits the use of any test for toroidal uniformity, and we consider versions of it incorporating three such tests. Simulation is used to illustrate the operating characteristics of the approach when the underlying distribution is assumed to be bivariate wrapped Cauchy. An analysis of wind direction data recorded at a Texan weather station illustrates the use of the proposed goodness-of-fit testing procedure.     

The performance of tests when observations have different variances

It is common to test if there is an effect due to a treatment. The commonly used tests have the assumption that the observations differ in location, and that their variances are the same over the groups. Different variances can arise if the observations being analyzed are means of different numbers of observations on individuals or slopes of growth curves with missing data. This study is concerned with cases in which the unequal variances are known, or known to a constant of proportionality. It examines the performance of the ttest, the Mann–Whitney–Wilcoxon Rank Sum test, the Median test, and the Van der Waerden test under these conditions. The t-test based on the weighted means is the likelihood ratio test under normality and has the usual optimality properties. The other tests are compared to it. One may align and scale the observations by subtracting the mean and dividing by the standard deviation of each point. This leads to other, analogous test statistics based on these adjusted observations. These statistics are also compared. Finally, the regression scores tests are compared to the other procedures.     

A Bayesian benchmarking of the Scott–Smith model for small areas

When the finite population ‘totals’ are estimated for individual areas, they do not necessarily add up to the known ‘total’ for all areas. Benchmarking (BM) is a technique used to ensure that the totals for all areas match the grand total, which can be obtained from an independent source. BM is desirable to practitioners of survey sampling. BM shifts the small-area estimators to accommodate the constraint. In doing so, it can provide increased precision to the small-area estimators of the finite population means or totals. The Scott–Smith model is used to benchmark the finite population means of small areas. This is a one-way random effects model for a superpopulation, and it is computationally convenient to use a Bayesian approach. We illustrate our method by estimating body mass index using data in the third National Health and Nutrition Examination Survey. Several properties of the benchmarked small-area estimators are obtained using a simulation study.     

Rank Tests for Clustered Survival Data

In a clinical trial, we may randomize subjects (called clusters) to different treatments (called groups), and make observations from multiple sites (called units) of each subject. In this case, the observations within each subject could be dependent, whereas those from different subjects are independent. If the outcome of interest is the time to an event, we may use the standard rank tests proposed for independent survival data, such as the logrank and Wilcoxon tests, to test the equality of marginal survival distributions, but their standard error should be modified to accommodate the possible intracluster correlation. In this paper we propose a method of calculating the standard error of the rank tests for two-sample clustered survival data. The method is naturally extended to that for K-sample tests under dependence.     

Pseudo‐likelihood ratio tests for semiparametric multivariate copula model selection

The authors propose pseudo‐likelihood ratio tests for selecting semiparametric multivariate copula models in which the marginal distributions are unspecified, but the copula function is parameterized and can be misspecified. For the comparison of two models, the tests differ depending on whether the two copulas are generalized nonnested or generalized nested. For more than two models, the procedure is built on the reality check test of White (2000). Unlike White (2000), however, the test statistic is automatically standardized for generalized nonnested models (with the benchmark) and ignores generalized nested models asymptotically. The authors illustrate their approach with American insurance claim data.     

Minimal Basis for a Connected Markov Chain over 3 × 3 ×K Contingency Tables with Fixed Two-Dimensional Marginals

This paper considers a connected Markov chain for sampling 3 × 3 ×K contingency tables having fixed two‐dimensional marginal totals. Such sampling arises in performing various tests of the hypothesis of no three‐factor interactions. A Markov chain algorithm is a valuable tool for evaluating P‐values, especially for sparse datasets where large‐sample theory does not work well. To construct a connected Markov chain over high‐dimensional contingency tables with fixed marginals, algebraic algorithms have been proposed. These algorithms involve computations in polynomial rings using Gröbner bases. However, algorithms based on Gröbner bases do not incorporate symmetry among variables and are very time‐consuming when the contingency tables are large. We construct a minimal basis for a connected Markov chain over 3 × 3 ×K contingency tables. The minimal basis is unique. Some numerical examples illustrate the practicality of our algorithms.     

Nonparametric Two‐Sample Tests of the Marginal Mark Distribution with Censored Marks

Occasionally, investigators collect auxiliary marks at the time of failure in a clinical study. Because the failure event may be censored at the end of the follow‐up period, these marked endpoints are subject to induced censoring. We propose two new families of two‐sample tests for the null hypothesis of no difference in mark‐scale distribution that allows for arbitrary associations between mark and time. One family of proposed tests is a nonparametric extension of an existing semi‐parametric linear test of the same null hypothesis while a second family of tests is based on novel marked rank processes. Simulation studies indicate that the proposed tests have the desired size and possess adequate statistical power to reject the null hypothesis under a simple change of location in the marginal mark distribution. When the marginal mark distribution has heavy tails, the proposed rank‐based tests can be nearly twice as powerful as linear tests.     

More powerful modifications of unit root tests allowing structural change

It is well known that more powerful variants of Dickey–Fuller unit root tests are available. We apply two of these modifications, on the basis of simple maximum statistics and weighted symmetric estimation, to Perron tests allowing for structural change in trend of the additive outlier type. Local alternative asymptotic distributions of the modified test statistics are derived, and it is shown that their implementation can lead to appreciable finite sample and asymptotic gains in power over the standard tests. Also, these gains are largely comparable with those from GLS-based modifications to Perron tests, though some interesting differences do arise. This is the case for both exogenously and endogenously chosen break dates. For the latter choice, the new tests are applied to the Nelson–Plosser data.     

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.