Two-stage k-sample designs for the ordered alternative problem

In preclinical studies and clinical dose-ranging trials, the Jonckheere-Terpstra test is widely used in the assessment of dose-response relationships. Hewett and Spurrier (1979) presented a two-stage analog of the test in the context of large sample sizes. In this paper, we propose an exact test based on Simon's minimax and optimal design criteria originally used in one-arm phase II designs based on binary endpoints. The convergence rate of the joint distribution of the first and second stage test statistics to the limiting distribution is studied, and design parameters are provided for a variety of assumed alternatives. The behavior of the test is also examined in the presence of ties, and the proposed designs are illustrated through application in the planning of a hypercholesterolemia clinical trial. The minimax and optimal two-stage procedures are shown to be preferable as compared with the one-stage procedure because of the associated reduction in expected sample size for given error constraints.     

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.     

A continuously adaptive nonparametric two–sample test

A two–sample test statistic for detecting shifts in location is developed for a broad range of underlying distributions using adaptive techniques. The test statistic is a linear rank statistics which uses a simple modification of the Wilcoxon test; the scores are Winsorized ranks where the upper and lower Winsorinzing proportions are estimated in the first stage of the adaptive procedure using sample the first stage of the adaptive procedure using sample measures of the distribution's skewness and tailweight. An empirical relationship between the Winsorizing proportions and the sample skewness and tailweight allows for a ‘continuous’ adaptation of the test statistic to the data. The test has good asymptotic properties, and the small sample results are compared with other populatr parametric, nonparametric, and two–stage tests using Monte Carlo methods. Based on these results, this proposed test procedure is recommended for moderate and larger sample sizes.     

Analyzing multiple end points with a two-stage design in clinical trials

In many clinical trials, the assessment of the response to interventions can include a large variety of outcome variables which are generally correlated. The use of multiple significance tests is likely to increase the chance of detecting a difference in at least one of the outcomes between two treatments. Furthermore, univariate tests do not take into account the correlation structure. A new test is proposed that uses information from the interim analysis in a two-stage design to form the rejection region boundaries at the second stage. Initially, the test uses Hotelling’s T² at the end of the first stage allowing only, for early acceptance of the null hypothesis and an O’Brien ‘type’ procedure at the end of the second stage. This test allows one to ‘cheat’ and look at the data at the interim analysis to form rejection regions at the second stage, provided one uses the correct distribution of the final test statistic. This distribution is derived and the power of the new test is compared to the power of three common procedures for testing multiple outcomes: Bonferroni’s inequality, Hotelling’s T²and O’Brien’s test. O’Brien’s test has the best power to detect a difference when the outcomes are thought to be affected in exactly the same direction and the same magnitude or in exactly the same relative effects as those proposed prior to data collection. However, the statistic is not robust to deviations in the alternative parameters proposed a priori, especially for correlated outcomes. The proposed new statistic and the derivation of its distribution allows investigators to consider information from the first stage of a two-stage design and consequently base the final test on the direction observed at the first stage or modify the statistic if the direction differs significantly from what was expected a prior.     

A new estimator of Kullback–Leibler information and its application in goodness of fit tests*

We propose here a general statistic for the goodness of fit test of statistical distributions. The proposed statistic is constructed based on an estimate of Kullback–Leibler information. The proposed test is consistent and the limiting distribution of the test statistic is derived. Then, the established results are used to introduce goodness of fit tests for the normal, exponential, Laplace and Weibull distributions. A simulation study is carried out for examining the power of the proposed test and to compare it with those of some existing procedures. Finally, some illustrative examples are presented and analysed, and concluding comments are made.     

A simple algorithm for the adaptation of scores and power behavior of the corresponding rank test

The purpose of this paper is twofold:On one hand we want to give a very simple algorithm for evaluating a special rank estimator of the type given in Behnen, Neuhaus, and Ruymgaart (1983) for the approximate optimal choice of the scores-generating function of a two-sample linear rank test for the general testing problem H_o:F=G versus H₁:F ≤ G, F ≠ G, in order to demonstrate that the corresponding adaptive rank statistic is simple enough for practical applications. On the other hand we prove the asymptotic normality of the adaptive rank statistic under H (leading to approximate critical values) and demonstrate the adaptive behavior of the corresponding rank test by a Monte Carlo power simulation for sample sizes as low as m=10, n=10.     

A Multiple Three-Decision Procedures for Comparing Several Treatments with a Control Under Heteroscedasticity

In this article, the design-oriented two-stage multiple three-decision procedure is proposed to classify a set of normal populations with respect to a control under heteroscedasticity. The statistical tables of percentage points and the power-related design constants, to implement our new two-stage procedure, are given. Sometimes when the sample for the second stage is not available, the one-stage data analysis procedure is proposed. Classifying a treatment better than control when it is actually worse (and vice versa) is known as type III error. Both the two-stage and one-stage procedures control the type III error rate at a specified level. The relationship between the two-stage and one-stage procedures is discussed. Finally, the application of the proposed procedures is illustrated with an example.     

Adaptively combining independent jonckheere statistics in a randomized block design with unequal scales

Jonckheere (1954) proposed a test statistic which is commonly used in testing for ordered alternatives in block designs.- We consider the application of Jonckheere's test statistic in block designs which have unequal scale parameters for the blocks. Estimates of the unknown scale parameters ar-fcrmed and are used to construct a modification of Jonckheere's test statistic using adaptive ideas. A Monte Carlo study shows that the modified Jonckheere is significantly more powerful than the original Jonckheere in many unequal scale situations.     

Nonparametric analysis of treatment effects with missing observations

This paper develops a test for comparing treatment effects when observations are missing at random for repeated measures data on independent subjects. It is assumed that missingness at any occasion follows a Bernoulli distribution. It is shown that the distribution of the vector of linear rank statistics depends on the unknown parameters of the probability law that governs missingness, which is absent in the existing conditional methods employing rank statistics. This dependence is through the variance–covariance matrix of the vector of linear ranks. The test statistic is a quadratic form in the linear rank statistics when the variance–covariance matrix is estimated. The limiting distribution of the test statistic is derived under the null hypothesis. Several methods of estimating the unknown components of the variance–covariance matrix are considered. The estimate that produces stable empirical Type I error rate while maintaining the highest power among the competing tests is recommended for implementation in practice. Simulation studies are also presented to show the advantage of the proposed test over other rank-based tests that do not account for the randomness in the missing data pattern. Our method is shown to have the highest power while also maintaining near-nominal Type I error rates. Our results clearly illustrate that even for an ignorable missingness mechanism, the randomness in the pattern of missingness cannot be ignored. A real data example is presented to highlight the effectiveness of the proposed method.     

A two sample ordered alternative test for means and variances

A two sample test of likelihood ratio type is proposed, assuming normal distribution theory, for testing the hypothesis that two samples come from identical normal populations versus the alternative that the populations are normal but vary in mean value and variance with one population having a smaller mean and smaller variance than the other. The small sample and large sample distribution of the proposed statistic are derived assuming normality. Some computations are presented which show the speed of convergence of small sample critical values to their asymptotic counterparts. Comparisons of local power of the proposed test are made with several potential competing tests. Asymptotics for the test statistic are derived when underlying distributions are not necessarily normal.     

On the power of the t-test and some rank tests when outliers may be present

The power of some rank tests, used for testing the hypothesis of shift, is found when the underlying distributions contain outliers. The outliers are assumed to occur as the result of mixing two normal distributions with common variance. A small sample case shows how the scores for the rank tests are found and the exact power is computed for each of these rank tests. A Monte Carlo study provides an estimate of the power of the usual two sample t-test.     

A minimum variance adaptive technique for parameter estimation and hypothesis testing

This report presents numerical results of an approach for parameter estimation and hypothesis testing that does not rely on specific assumptions about the underlying distribution of errors in the measured data. This approach combines robust estimation procedures, the bootstrap method for estimation of parameter uncertainties, permutation techniques for hypothesis testing, and adaptive approaches to estimation in order to obtain the minimum variance estimator or test statistic (within a predefined class) for the data under consideration. The technique produces efficient estimators of central tendency and powerful test statistics, even for small sample sizes. (Portions of this work have been presented in preliminary form (Turkheimer et al., 1996)).     

Comparing mean ranks for repeated measures data

Rank tests are considered that compare t treatments in repeated measures designs. A statistic is given that contains as special cases several that have been proposed for this problem, including one that corresponds to the randomized block ANOVA statistic applied to the rank transformed data. Another statistic is proposed, having a null distribution holding under more general conditions, that is the rank transform of the Hotelling statistic for repeated measures. A statistic of this type is also given for data that are ordered categorical rather than fully rankedo Unlike the Friedman statistic, the statistics discussed in this article utilize a single ranking of the entire sample. Power calculations for an underlying normal distribution indicate that the rank transformed ANOVA test can be substantially more powerful than the Friedman test.     

Letters to the Editor

Although several authors have indicated that the median test has low power in small samples, it continues to be presented in many statistical textbooks, included in a number of popular statistical software packages, and used in a variety of application areas. We present results of a power simulation study that shows that the median test has noticeably lower power, even for the double exponential distribution for which it is asymptotically most powerful, than other readily available rank tests. We suggest that the median test be “retired” from routine use and recommend alternative rank tests that have superior power over a relatively large family of symmetric distributions.     

A note on a parametric extension of rank tests to the analysis of multifactor experiments within blocks

A general rank test procedure based on an underlying multinomial distribution is suggested for randomized block experiments with multifactor treatment combinations within each block. The Wald statistic for the multinomial is used to test hypotheses about the within–block rankings. This statistic is shown to be related to the one–sample Hotellingt's T² statistic, suggesting a method for computing the test statistic using the standard statistical computer packages.     

A rank test for constancy of a location oil scale parameter

X nonlinear rank statistic is proposed to test whether a location or scale parameter of a continuous distribution has remained constant over time. The test is based on frequency domain characteristics of the data and uses the periodogram of appropriate rank scores. The test has good power against alternatives involving multiple abrupt up and down changes     

A test for independence in a multivariate exponential distribution with equal correlation coefficients

A rank statistic is considered which may be used for testing for total independence in a p-variate exponential distribution with equal correlation coefficients. Critical values for the statistic are provided for p = 3.4 and sample sizes less than or equal to 20. Finally, the small sample power performance of the rank test relative to that of the locally most powerful similar lest under the exponential alternative is evaluated.     

Tests of hypotheses based on ranks in the general linear model

A unified approach is developed for testing hypotheses in the general linear model based on the ranks of the residuals. It complements the nonparametric estimation procedures recently reported in the literature. The testing and estimation procedures together provide a robust alternative to least squares. The methods are similar in spirit to least squares so that results are simple to interpret. Hypotheses concerning a subset of specified parameters can be tested, while the remaining parameters are treated as nuisance parameters. Asymptotically, the test statistic is shown to have a chi-square distribution under the null hypothesis. This result is then extended to cover a sequence of contiguous alternatives from which the Pitman efficacy is derived. The general application of the test requires the consistent estimation of a functional of the underlying distribution and one such estimate is furnished.     

A test for bivariate two sample location problem

A consistent test for difference in locations between two bivariate populations is proposed, The test is similar as the Mann-Whitney test and depends on the exceedances of slopes of the two samples where slope for each sample observation is computed by taking the ratios of the observed values. In terms of the slopes, it reduces to a univariate problem, The power of the test has been compared with those of various existing tests by simulation. The proposed test statistic is compared with Mardia's(1967) test statistics, Peters-Randies(1991) test statistic, Wilcoxon's rank sum test. statistic and Hotelling' T² test statistic using Monte Carlo technique. It performs better than other statistics compared for small differences in locations between two populations when underlying population is population 7(light tailed population) and sample size 15 and 18 respectively. When underlying population is population 6(heavy tailed population) and sample sizes are 15 and 18 it performas better than other statistic compared except Wilcoxon's rank sum test statistics for small differences in location between two populations. It performs better than Mardia's(1967) test statistic for large differences in location between two population when underlying population is bivariate normal mixture with probability p=0.5, population 6, Pearson type II population and Pearson type VII population for sample size 15 and 18 .Under bivariate normal population it performs as good as Mardia' (1967) test statistic for small differences in locations between two populations and sample sizes 15 and 18. For sample sizes 25 and 28 respectively it performs better than Mardia's (1967) test statistic when underlying population is population 6, Pearson type II population and Pearson type VII population     

An adaptive two-sample location-scale test of lepage type for symmetric distributions

For the two-sample location and scale problem we propose an adaptive test which is based on so called Lepage type tests. The well known test of Lepage (1971) is a combination of the Wilcoxon test for location alternatives and the Ansari-Bradley test for scale alternatives and it behaves well for symmetric and medium-tailed distributions. For the cae of short-, medium- and long-tailed distributions we replace the Wilcoxon test and the .Ansari-Bradley test by suitable other two-sample tests for location and scale, respectively, in oder to get higher power than the classical Lepage test for such distribotions. These tests here are called Lepage type tests. in practice, however, we generally have no clear idea about the distribution having generated our data. Thus, an adaptive test should be applied which takes the the given data set inio consideration. The proposed adaptive test is based on the concept of Hogg (1974), i.e., first, to classify the unknown symmetric distribution function with respect to a measure for tailweight and second, to apply an appropriate Lepage type test for this classified type of distribution. We compare the adaptive test with the three Lepage type tests in the adaptive scheme and with the classical Lepage test as well as with other parametric and nonparametric tests. The power comparison is carried out via Monte Carlo simulation. It is shown that the adaptive test is the best one for the broad class of distributions considered.