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.     

Ordered Tests for Right–Censored Survival Data

In comparative clinical trials or animal carcinogenesis studies, the effect of increasing dose levels of an agent or an increasing number of additional modalities are frequently evaluated on the prolonged survival time of patients with a particular disease. It is of particular interest to test the ordered alternative that a treatment level increase leads to better survival. This paper considers an ordered test based on the two–sample weighted Kaplan–Meier statistics (Pepe & Fleming, 1989, 1991). It evaluates asymptotic relative efficiencies of the proposed ordered weighted Kaplan–Meier test, the competing ordered weighted logrank test (Liu et al., 1993) and modified ordered logrank test (Liu & Tsai, 1999) under Lehmann alternatives, for various piecewise exponential survival distributions. Finally, it demonstrates the proposed test on an appropriate dataset.     

Exact sample-size determination in testing non-inferiority under a simple crossover trial

For testing the non-inferiority (or equivalence) of an experimental treatment to a standard treatment, the odds ratio (OR) of patient response rates has been recommended to measure the relative treatment efficacy. On the basis of an exact test procedure proposed elsewhere for a simple crossover design, we develop an exact sample-size calculation procedure with respect to the OR of patient response rates for a desired power of detecting non-inferiority at a given nominal type I error. We note that the sample size calculated for a desired power based on an asymptotic test procedure can be much smaller than that based on the exact test procedure under a given situation. We further discuss the advantage and disadvantage of sample-size calculation using the exact test and the asymptotic test procedures. We employ an example by studying two inhalation devices for asthmatics to illustrate the use of sample-size calculation procedure developed here.     

Asymptotic hypothesis test to compare likelihood ratios of multiple diagnostic tests in unpaired designs

The accuracy of a binary diagnostic test is usually measured in terms of its sensitivity and its specificity. Other measures of the performance of a diagnostic test are the positive and negative likelihood ratios, which quantify the increase in knowledge about the presence of the disease through the application of a diagnostic test, and which depend on the sensitivity and specificity of the diagnostic test. In this article, we construct an asymptotic hypothesis test to simultaneously compare the positive and negative likelihood ratios of two or more diagnostic tests in unpaired designs. The hypothesis test is based on the logarithmic transformation of the likelihood ratios and on the chi-square distribution. Simulation experiments have been carried out to study the type I error and the power of the constructed hypothesis test when comparing two and three binary diagnostic tests. The method has been extended to the case of multiple multi-level diagnostic tests.     

Asymptotic test statistics for coefficients of variation

A one-sample asymptotically normal test statistic Is derived for testing the hypothesis that the coefficient of variation of a normal population is equal to a specified value. Based on this derivation, an asymptotically noraml two-sample test statistic and an asymptotically chi-square k-sample test statistic are derived for testing the hypothesis that the coefficients of variation of k ≥2 normal populations are equal. The two and k-sample test statistics allow for unequal sample sizes. Results of a simulation study which evaluate the size and power of the test statistics and compare the test statistics to earlier ones developed by McKay (1932) and Bennett (1976) are presented.     

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.     

Testing for error cross-sectional uncorrelatedness in a two-way error components panel data model

This paper proposes a new test for the error cross-sectional uncorrelatedness in a two-way error components panel data model based on large panel data sets. By virtue of an existing statistic under the raw data circumstance, an analogous test statistic using the within residuals of the model is constructed. We show that the resulting statistic needs bias correction to make valid inference, and then propose a method to implement feasible correction. Simulation shows that the test based on the feasible bias-corrected statistic performs well. Additionally, we employ a real data set to illustrate the use of the new test.     

Sample size determination for testing equality in Poisson frequency data under an AB/BA crossover trial

Assuming that the frequency of occurrence follows the Poisson distribution, we develop sample size calculation procedures for testing equality based on an exact test procedure and an asymptotic test procedure under an AB/BA crossover design. We employ Monte Carlo simulation to demonstrate the use of these sample size formulae and evaluate the accuracy of sample size calculation formula derived from the asymptotic test procedure with respect to power in a variety of situations. We note that when both the relative treatment effect of interest and the underlying intraclass correlation between frequencies within patients are large, the sample size calculation based on the asymptotic test procedure can lose accuracy. In this case, the sample size calculation procedure based on the exact test is recommended. On the other hand, if the relative treatment effect of interest is small, the minimum required number of patients per group will be large, and the asymptotic test procedure will be valid for use. In this case, we may consider use of the sample size calculation formula derived from the asymptotic test procedure to reduce the number of patients needed for the exact test procedure. We include an example regarding a double‐blind randomized crossover trial comparing salmeterol with a placebo in exacerbations of asthma to illustrate the practical use of these sample size formulae. Copyright © 2013 John Wiley & Sons, Ltd.     

New innovational outlier unit root test with a break at an unknown time

The Perron test which is based on a Dickey–Fuller test regression is a commonly employed approach to test for a unit root in the presence of a structural break of unknown timing. In the case of an innovational outlier (IO), the Perron test tends to exhibit spurious rejections in finite samples when the break occurs under the null hypothesis. In the present paper, a new Perron-type IO unit root test is developed. It is shown in Monte Carlo experiments that the new test does not over-reject the null hypothesis. Even for the case of a level and slope break for trending data, the empirical size is near its nominal level. The test distribution equals the case of a known break date. Furthermore, the test is able to identify the true break date very accurately even for small breaks. As an application serves the Nelson–Plosser data set.     

An empirical likelihood ratio based goodness-of-fit test for Inverse Gaussian distributions

The Inverse Gaussian (IG) distribution is commonly introduced to model and examine right skewed data having positive support. When applying the IG model, it is critical to develop efficient goodness-of-fit tests. In this article, we propose a new test statistic for examining the IG goodness-of-fit based on approximating parametric likelihood ratios. The parametric likelihood ratio methodology is well-known to provide powerful likelihood ratio tests. In the nonparametric context, the classical empirical likelihood (EL) ratio method is often applied in order to efficiently approximate properties of parametric likelihoods, using an approach based on substituting empirical distribution functions for their population counterparts. The optimal parametric likelihood ratio approach is however based on density functions. We develop and analyze the EL ratio approach based on densities in order to test the IG model fit. We show that the proposed test is an improvement over the entropy-based goodness-of-fit test for IG presented by Mudholkar and Tian (2002). Theoretical support is obtained by proving consistency of the new test and an asymptotic proposition regarding the null distribution of the proposed test statistic. Monte Carlo simulations confirm the powerful properties of the proposed method. Real data examples demonstrate the applicability of the density-based EL ratio goodness-of-fit test for an IG assumption in practice.     

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.     

Mantel test for spatial functional data

Statistics for spatial functional data is an emerging field in statistics which combines methods of spatial statistics and functional data analysis to model spatially correlated functional data. Checking for spatial autocorrelation is an important step in the statistical analysis of spatial data. Several statistics to achieve this goal have been proposed. The test based on the Mantel statistic is widely known and used in this context. This paper proposes an application of this test to the case of spatial functional data. Although we focus particularly on geostatistical functional data, that is functional data observed in a region with spatial continuity, the test proposed can also be applied with functional data which can be measured on a discrete set of areas of a region (areal functional data) by defining properly the distance between the areas. Based on two simulation studies, we show that the proposed test has a good performance. We illustrate the methodology by applying it to an agronomic data set.     

A New Discordancy Test in Circular Data Using Spacings Theory

In this article, we propose a new test of discordancy based on spacing theory in circular data. The test should provide a good alternative to existing tests of discordancy for detecting single or well-separated multiple outliers. On top of that, the new method can be generalized to identify a patch of outliers in data. The percentage points are calculated and the performance is examined. We first investigate the performance of the test for detecting a single outlier and show that the new test performs well compared to other known tests. We then show that the generalized test works well in detecting a patch of outliers in the data. As an illustration, a practical example based on an eye dataset obtained from a glaucoma clinic at the University of Malaya Medical Center, Malaysia is presented.     

A sequential testing procedure for outliers and structural change

In this paper a test for outliers based on externally studentized residuals is shown to be related to a test for predictive failure. The relationships between a test for outliers, a test for a correlated mean shift and a test for an intercept shift are developed. A sequential testing procedure for outliers and structural change is shown to be independent, so that the overall size of the joint test can be determined exactly. It is established that a joint test for outliers and constancy of variances cannot be performed.     

Testing for programme effects in a regression discontinuity design with imperfect compliance

Summary. The administrators of an office automation training programme in Italy enrolled applicants on the basis of their score in an attitudinal test, with low scoring subjects mandated out of the programme. Some of the applicants who were mandated out resorted to an alternative programme. To identify the effect of the programme by comparing participants with non-participants we need to account properly for both the selection by the score and the contamination of the comparison group by a number of non-complying subjects. The estimand resulting from using the mandated status as an instrumental variable for the actual status identifies the effect of the programme on complying subjects exhibiting a score in the attitudinal test in the neighbourhood of the threshold for selection. Simple nonparametric instrumental variable estimators based on the work of Robinson and Hahn and co-workers reveal that the programme had no effect on the probability of being in work several months after its completion. Simulation results show that in spite of the small sample size the test for the no-impact hypothesis has non-negligible power even at small departures from the null hypothesis. As a side-result Robinson's test turns out to be appreciably more powerful than the other test.     

A Weighted Least Squares Approach to Levene's Test of Homogeneity of Variance

In 1960 Levene suggested a potentially robust test of homogeneity of variance based on an ordinary least squares analysis of variance of the absolute values of mean-based residuals. Levene's test has since been shown to have inflated levels of significance when based on the F-distribution, and tests a hypothesis other than homogeneity of variance when treatments are unequally replicated, but the incorrect formulation is now standard output in several statistical packages. This paper develops a weighted least squares analysis of variance of the absolute values of both mean-based and median-based residuals. It shows how to adjust the residuals so that tests using the F -statistic focus on homogeneity of variance for both balanced and unbalanced designs. It shows how to modify the F -statistics currently produced by statistical packages so that the distribution of the resultant test statistic is closer to an F-distribution than is currently the case. The weighted least squares approach also produces component mean squares that are unbiased irrespective of which variable is used in Levene's test. To complete this aspect of the investigation the paper derives exact second-order moments of the component sums of squares used in the calculation of the mean-based test statistic. It shows that, for large samples, both ordinary and weighted least squares test statistics are equivalent; however they are over-dispersed compared to an F variable.     

Distribution-free test for symmetry based on Bonferroni's measure

We propose a test based on Bonferroni's measure of skewness. The test detects the asymmetry of a distribution function about an unknown median. We study the asymptotic distribution of the given test statistic and provide a consistent estimate of its variance. The asymptotic relative efficiency of the proposed test is computed along with Monte Carlo estimates of its power. This allows us to perform a comparison of the test based on Bonferroni's measure with other tests for symmetry.     

Seme distributions and their implications for an internal pilot study with a univariate linear model

In planning a study, the choice of sample size may depend on a variance value based on speculation or obtained from an earlier study. Scientists may wish to use an internal pilot design to protect themselves against an incorrect choice of variance. Such a design involves collecting a portion of the originally planned sample and using it to produce a new variance estimate. This leads to a new power analysis and increasing or decreasing sample size. For any general linear univariate model, with fixed predictors and Gaussian errors, we prove that the uncorrected fixed sample F-statistic is the likelihood ratio test statistic. However, the statistic does not follow an F distribution. Ignoring the discrepancy may inflate test size. We derive and evaluate properties of the components of the likelihood ratio test statistic in order to characterize and quantify the bias. Most notably, the fixed sample size variance estimate becomes biased downward. The bias may inflate test size for any hypothesis test, even if the parameter being tested was not involved in the sample size re-estimation. Furthermore, using fixed sample size methods may create biased confidence intervals for secondary parameters and the variance estimate.     

The performance of univariate goodness-of-fit tests for normality based on the empirical characteristic function in large samples

A test based on the studentized empirical characteristic function calculated in a single point is derived. An empirical power comparison is made between this test and tests like the Epps–Pulley, Shapiro–Wilks, Anderson–Darling and other tests for normality. It is shown to outperform the more complicated Epps-Pulley test based on the empirical characteristic function and a Cramér-von Mises type expression in a simulation study. The test performs especially good in large samples and the derived test statistic has an asymptotic normal distribution which is easy to apply.     

Hypothesis Testing Problems in an Unbalanced Longitudinal Ophthalmology Study

ABSTRACT

The analysis of clustered data in a longitudinal ophthalmology study is complicated by correlations between repeatedly measured visual outcomes of paired eyes in a participant and missing observations due to the loss of follow-up. In the present article we consider hypothesis testing problems in an ophthalmology study, where eligible eyes are randomized to two treatments (when two eyes of a participant are eligible, the paired eyes are assigned to different treatments), and vision function outcomes are repeatedly measured over time. A large sample-based nonparametric test statistic and a nonparametric Bootstrap test analog are proposed for testing an interaction effect of two factors and testing an effect of a eye-specific factor within a level of the other person-specific factor on visual function outcomes. Both test statistics allow for missing observations, correlations between repeatedly measured outcomes on individual eyes, and correlations between repeatedly measured outcomes on both eyes of each participant. A simulation study shows that these proposed test statistics maintain nominal significance levels approximately and comparable powers to each other, as well as higher powers than the naive test statistic ignoring correlations between repeated bilateral measurements of both eyes in the same person. For illustration, we apply the proposed test statistics to the changes of visual field defect score in the Advanced Glaucoma Intervention Study.