An exact test for trend among binomial proportions based on a modified Baumgartner-Weiß-Schindler statistic

The Cochran-Armitage test is the most frequently used test for trend among binomial proportions. This test can be performed based on the asymptotic normality of its test statistic or based on an exact null distribution. As an alternative, a recently introduced modification of the Baumgartner-Weiß-Schindler statistic, a novel nonparametric statistic, can be used. Simulation results indicate that the exact test based on this modification is preferable to the Cochran-Armitage test. This exact test is less conservative and more powerful than the exact Cochran-Armitage test. The power comparison to the asymptotic Cochran-Armitage test does not show a clear winner, but the difference in power is usually small. The exact test based on the modification is recommended here because, in contrast to the asymptotic Cochran-Armitage test, it guarantees a type I error rate less than or equal to the significance level. Moreover, an exact test is often more appropriate than an asymptotic test because randomization rather than random sampling is the norm, for example in biomedical research. The methods are illustrated with an example data set.     

Rank invariant tests with left truncated and interval censored data

Peto and Peto (1972) have studied rank invariant tests to compare two survival curves for right censored data. We apply their tests, including the logrank test and the generalized Wilcoxon test, to left truncated and interval censored data. The significance levels of the tests are approximated by Monte Carlo permutation tests. Simulation studies are conducted to show their size and power under different distributional differences. In particular, the logrank test works well under the Cox proportional hazards alternatives, as for the usual right censored data. The methods are illustrated by the analysis of the Massachusetts Health Care Panel Study dataset.     

PERMUTATION TESTS AND OTHER TEST STATISTICS FOR ILL-BEHAVED DATA: EXPERIENCE OF THE NINDS t-PA STROKE TRIAL

Permutation tests are often used to analyze data since they may not require one to make assumptions regarding the form of the distribution to have a random and independent sample selection. We initially considered a permutation test to assess the treatment effect on computed tomography lesion volume in the National Institute of Neurological Disorders and Stroke (NINDS) t-PA Stroke Trial, which has highly skewed data. However, we encountered difficulties in summarizing the permutation test results on the lesion volume. In this paper, we discuss some aspects of permutation tests and illustrate our findings. This experience with the NINDS t-PA Stroke Trial data emphasizes that permutation tests are useful for clinical trials and can be used to validate assumptions of an observed test statistic. The permutation test places fewer restrictions on the underlying distribution but is not always distribution-free or an exact test, especially for ill-behaved data. Quasi-likelihood estimation using the generalized estimating equation (GEE) approach on transformed data seems to be a good choice for analyzing CT lesion data, based on both its corresponding permutation test and its clinical interpretation.     

Impact of surviving time on tests for carcinogenicity

In tumorigenicity experiments, each animal begins in a tumor-free state and then either develops a tumor or dies before developing a tumor. Animals that develop a tumor either die from the tumor or from other competing causes. All surviving animals are sacrificed at the end of the experiment, normally two years. The two most commonly used statistical tests are the logrank test for comparing hazards of death from rapidly lethal tumors and the Hoel-Walburg test for comparing prevalences of nonlethal tumors. However, the data obtained from a carcinogenicity experiment generally contains a mixture of fatal and incidental tumors. Peto et al.(1980)suggested combining the fatal and incidental tests for a comparison of tumor onset distributions.

Extensive simulations show that the trend test for tumor onset using the Peto procedure has the proper size, under the simulation constraints, when each group has identical mortality patterns, and the test with continuity correction tends to be conservative. When the animals n the dosed groups have reduced survival rates, the type I error rate is likely to exceed the nominal level. The continuity correction is recommended for a small reduction in survival time among the dosed groups to ensure the proper size. However, when there is a large reduction in survival times in the dosed groups, the onset test does not have the proper size.     

An exact two-sample test based on the baumgartner-weiss-schindler statistic and a modification of lepage's test

It is shown that the nonparametric two-saniDle test recently proposed by Baumgartner, WeiB, Schindler (1998, Biometrics, 54, 1129-1135) does not control the type I error rate in case of small sample sizes. We investigate the exact permutation test based on their statistic and demonstrate that this test is almost not conservative. Comparing exact tests, the procedure based on the new statistic has a less conservative size and is, according to simulation results, more powerful than the often employed Wilcoxon test. Furthermore, the new test is also powerful with regard to less restrictive settings than the location-shift model. For example, the test can detect location-scale alternatives. Therefore, we use the test to create a powerful modification of the nonparametric location-scale test according to Lepage (1971, Biometrika, 58, 213-217). Selected critical values for the proposed tests are given.     

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.     

The split sample permutation t-tests

Without the exchangeability assumption, permutation tests for comparing two population means do not provide exact control of the probability of making a Type I error. Another drawback of permutation tests is that it cannot be used to test hypothesis about one population. In this paper, we propose a new type of permutation tests for testing the difference between two population means: the split sample permutation t-tests. We show that the split sample permutation t-tests do not require the exchangeability assumption, are asymptotically exact and can be easily extended to testing hypothesis about one population. Extensive simulations were carried out to evaluate the performance of two specific split sample permutation t-tests: the split in the middle permutation t-test and the split in the end permutation t-test. The simulation results show that the split in the middle permutation t-test has comparable performance to the permutation test if the population distributions are symmetric and satisfy the exchangeability assumption. Otherwise, the split in the end permutation t-test has significantly more accurate control of level of significance than the split in the middle permutation t-test and other existing permutation tests.     

Permutation tests for homogeneity based on some characterizations

In this work, non parametric tests are proposed for testing the homogeneity of two or more populations. The tests are based on recently obtained characterizations. The test procedure is based on the permutation bootstrap technique. For the two-sample case the new tests are compared with permutation tests based on the empirical characteristic function and some other tests. The comparison is fulfilled via a Monte Carlo simulation.     

On the theory of modified randomization tests for nonparametric hypotheses

A general randomization test for nonparametric hypotheses which is a modification of permutation tests in proposed. The exact level of the test is derived and under mild gegularity conditions, a general result on the consistency of the power function is obtained. Applications to several testing problems are considered. Asymptotic expansions of the power of this test are derived with respect to contiguous alternatives thus test are derived with respect to contiguous alternatives thus enabling us to make deficiency comparisons with permutation tests. The paper concludes with some Monte Carlo simulations verifying the theoretical results derived.     

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.     

Significance levels and confidence intervals for permutation tests

A computational algorithm is given which calculates exact significance levels of a wide class of permutation tests in the one and two sample problems. This class includes the permutation test based on the means, locally most powerful permutation tests and linear rank tests. When a shift model is assumed confidence intervals can also be obtained. Approximate methods, based on asymptotic expansions, are also presented.     

Exact tests based on the Baumgartner-Weiß-Schindler statistic—A survey

It is the purpose of this paper to review recently-proposed exact tests based on the Baumgartner-Weiß-Schindler statistic and its modification. Except for the generalized Behrens-Fisher problem, these tests are broadly applicable, and they can be used to compare two groups irrespective of whether or not ties occur. In addition, a nonparametric trend test and a trend test for binomial proportions are possible. These exact tests are preferable to commonly-applied tests, such as the Wilcoxon rank sum test, in terms of both type I error rate and power.     

Multivariate tests comparing binomial probabilities, with application to safety studies for drugs

Summary.  In magazine advertisements for new drugs, it is common to see summary tables that compare the relative frequency of several side-effects for the drug and for a placebo, based on results from placebo-controlled clinical trials. The paper summarizes ways to conduct a global test of equality of the population proportions for the drug and the vector of population proportions for the placebo. For multivariate normal responses, the Hotelling T ²-test is a well-known method for testing equality of a vector of means for two independent samples. The tests in the paper are analogues of this test for vectors of binary responses. The likelihood ratio tests can be computationally intensive or have poor asymptotic performance. Simple quadratic forms comparing the two vectors provide alternative tests. Much better performance results from using a score-type version with a null-estimated covariance matrix than from the sample covariance matrix that applies with an ordinary Wald test. For either type of statistic, asymptotic inference is often inadequate, so we also present alternative, exact permutation tests. Follow-up inferences are also discussed, and our methods are applied to safety data from a phase II clinical trial.     

A non-parametric maximum test for the Behrens–Fisher problem

Non-normality and heteroscedasticity are common in applications. For the comparison of two samples in the non-parametric Behrens–Fisher problem, different tests have been proposed, but no single test can be recommended for all situations. Here, we propose combining two tests, the Welch t test based on ranks and the Brunner–Munzel test, within a maximum test. Simulation studies indicate that this maximum test, performed as a permutation test, controls the type I error rate and stabilizes the power. That is, it has good power characteristics for a variety of distributions, and also for unbalanced sample sizes. Compared to the single tests, the maximum test shows acceptable type I error control.     

Log‐rank permutation tests for trend: saddlepoint p‐values and survival rate confidence intervals

Suppose p + 1 experimental groups correspond to increasing dose levels of a treatment and all groups are subject to right censoring. In such instances, permutation tests for trend can be performed based on statistics derived from the weighted log‐rank class. This article uses saddlepoint methods to determine the mid‐P‐values for such permutation tests for any test statistic in the weighted log‐rank class. Permutation simulations are replaced by analytical saddlepoint computations which provide extremely accurate mid‐P‐values that are exact for most practical purposes and almost always more accurate than normal approximations. The speed of mid‐P‐value computation allows for the inversion of such tests to determine confidence intervals for the percentage increase in mean (or median) survival time per unit increase in dosage. The Canadian Journal of Statistics 37: 5‐16; 2009 © 2009 Statistical Society of Canada     

Permutation Tests for Linear Models

Several approximate permutation tests have been proposed for tests of partial regression coefficients in a linear model based on sample partial correlations. This paper begins with an explanation and notation for an exact test. It then compares the distributions of the test statistics under the various permutation methods proposed, and shows that the partial correlations under permutation are asymptotically jointly normal with means 0 and variances 1. The method of Freedman & Lane (1983) is found to have asymptotic correlation 1 with the exact test, and the other methods are found to have smaller correlations with this test. Under local alternatives the critical values of all the approximate permutation tests converge to the same constant, so they all have the same asymptotic power. Simulations demonstrate these theoretical results.     

Row similarity in binary-valued matrices

An algorithm is developed for calculating the probability distribution of the number of matches between two specified rows of a matrix of zeroes and ones. Cases covered include row totals fixed, column totals fixed, and column totals and the two specified rows' totals fixed. The results are applied to presence-absence data on six species of ground finches on 23 Galàpagos islands and two constructed examples.     

The power of the fisher permutation test in 2 × k tables

The power of the Fisher permutation test extended to 2 × k tables is evaluated unconditionally as a function of the under-lying cell probabilities in the table. These results are then applied in assessing the sensitivity of two-generation cancer bioassays in which a fixed number of pups from each litter born in the first generation are selected to continue on test in the second generation. In this case, the two rows of the table correspond to two treatment groups and the k columns correspond to the number of animals responding in a litter. The cell probabilities in this application are based on a suitable beta-binomial superpopulation model.     

Distribution-free tests for cluster samples of ordinal responses

A simulation comparison is done of Mann–Whitney U test extensions recently proposed for simple cluster samples or for repeated ordinal responses. These are based on two approaches: the permutation approach of Fay and Gennings (four tests, two exact), and Edwardes’ approach (two asymptotic tests, one new). Edwardes’ approach permits confidence interval estimation, unlike the permutation approach. An appropriate parameter for estimation is P(X<Y)−P(X>Y), where X is the rank of a response from group 1 and Y is from group 2. The permutation tests are shown to be unsuitable for some survey data, since they are sensitive to a difference in cluster intra-correlations when there is no distribution difference between groups at the individual level. The exact permutation tests are of little use for less than seven clusters, precisely where they are most needed. Otherwise, the permutation tests perform well.     

Robust permutation tests for one sample

We consider robust permutation tests based on an estimating equation comparing the test statistics based on the score function with those based on the M-estimate. First we obtain a form for the tests so that the exact tests may be carried out using the same algorithms as used for permutation tests based on the mean. Then we compare the efficiencies of the tests in two cases, equivalent to the sign test and a test based on Huber scores, showing that they are equivalent in the Pitman sense but that they have different Bahadur slopes with neither exceeding the other over the whole range.