A random-sum Wilcoxon statistic and its application to analysis of ROC and LROC data

The Wilcoxon-Mann-Whitney statistic is commonly used for a distribution-free comparison of two groups. One requirement for its use is that the sample sizes of the two groups are fixed. This is violated in some of the applications such as medical imaging studies and diagnostic marker studies; in the former, the violation occurs since the number of correctly localized abnormal images is random, while in the latter the violation is due to some subjects not having observable measurements. For this reason, we propose here a random-sum Wilcoxon statistic for comparing two groups in the presence of ties, and derive its variance as well as its asymptotic distribution for large sample sizes. The proposed statistic includes the regular Wilcoxon rank-sum statistic. Finally, we apply the proposed statistic for summarizing location response operating characteristic data from a liver computed tomography study, and also for summarizing diagnostic accuracy of biomarker data.     

Designing historical control studies with survival endpoints using exact statistical inference

Historical control trials compare an experimental treatment with a previously conducted control treatment. By assigning all recruited samples to the experimental arm, historical control trials can better identify promising treatments in early phase trials compared with randomized control trials. Existing designs of historical control trials with survival endpoints are based on asymptotic normal distribution. However, it remains unclear whether the asymptotic distribution of the test statistic is close enough to the true distribution given relatively small sample sizes in early phase trials. In this article, we address this question by introducing an exact design approach for exponentially distributed survival endpoints, and compare it with an asymptotic design in both real examples and simulation examples. Simulation results show that the asymptotic test could lead to bias in the sample size estimation. We conclude the proposed exact design should be used in the design of historical control trials.     

Testing homogeneity against trend based on rank in one-way layout

In this paper, we are concerned with testing homogeneity against trend. Parsons (1979) considered the exact distribution of the test statistic based on the Wilcoxon type scores. We extend his result to the case of the general scores. Then we give a table of significance probabilities for the Fisher-Yates normal scores. We also study the asymptotic distribution of the test statis-tic based on the general scores under the null hypothesis, and the asymptotic relative efficiency against Bartholomew's likelihood ratio test assuming normality     

A test for bivariate symmetry based on the empirical distribution function

Hollander (1970) proposed a conditionally distribution-free test of bivariate symmetry based on the empirical distribution function. In this paper Hollander’s test statistic is examined In greater detail: in particular; its conditional asymptotic distribution is derived under the null hypothesis as well as under a sequence of local alternatives. Percentage points of the asymptotic distribution are presented; a power comparison between Hollander’s statistic and the likelihood ratio criterion in testing a variant of the sphericity hypothesis in multivariate analysis is made.     

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.     

A New Test Procedure for Decreasing Mean Residual Life

A new test statistic for testing the strict DMRL property of life distribution is developed. The asymptotic normality is established and the comparison between the test proposed and some other related ones in literature is conducted through evaluating the Pitman's asymptotic relative efficiency. Edge-worth expansion is also employed to improve the accuracy of the convergence rate of the test statistic. Some numerical results are presented as well to demonstrate the performance and the asymptotic normality of the new testing procedure.     

Penalized likelihood ratio test for a biomarker threshold effect in clinical trials based on generalized linear models

In a clinical trial, the responses to the new treatment may vary among patient subsets with different characteristics in a biomarker. It is often necessary to examine whether there is a cutpoint for the biomarker that divides the patients into two subsets of those with more favourable and less favourable responses. More generally, we approach this problem as a test of homogeneity in the effects of a set of covariates in generalized linear regression models. The unknown cutpoint results in a model with nonidentifiability and a nonsmooth likelihood function to which the ordinary likelihood methods do not apply. We first use a smooth continuous function to approximate the indicator function defining the patient subsets. We then propose a penalized likelihood ratio test to overcome the model irregularities. Under the null hypothesis, we prove that the asymptotic distribution of the proposed test statistic is a mixture of chi-squared distributions. Our method is based on established asymptotic theory, is simple to use, and works in a general framework that includes logistic, Poisson, and linear regression models. In extensive simulation studies, we find that the proposed test works well in terms of size and power. We further demonstrate the use of the proposed method by applying it to clinical trial data from the Digitalis Investigation Group (DIG) on heart failure.     

Gini index based goodness-of-fit test for the logistic distribution

To model growth curves in survival analysis and biological studies the logistic distribution has been widely used. In this article, we propose a goodness-of-fit test for the logistic distribution based on an estimate of the Gini index. The exact distribution of the proposed test statistic and also its asymptotic distribution are presented. In order to compute the proposed test statistic, parameters of the logistic distribution are estimated by approximate maximum likelihood estimators (AMLEs), which are simple explicit estimators. Through Monte Carlo simulations, power comparisons of the proposed test with some known competing tests are carried. Finally, an illustrative example is presented and analyzed.     

A signed rank test for censored paired lifetimes

A locally most powerful signed rank test is proposed for the comparison of two independent lifetimes under the accelerated failure time model.

The test is based on N independent pairs(X_i, Y_i), i = 1, …, N: it is supposed that the shortest lifetime in each pair is observed and the experiment is stopped after r(r≤N and fixed) such lifetimes are available (type II censoring).

Actual scores of the test statistic are computed for some specific source distributions of the observations. The asymptotic distribution of the test statistic, as well as the asymptotic power and efficiency are given. The values of these efficiencies are computed for the case where the X_i follow and exponential, Weibull Gamma or Rayleigh distribution.     

Small-sample comparisons for powerdivergence goodness-of-fit statistics for symmetric and skewed simple null hypotheses

Power-divergence goodness-of-fit statistics have asymptotically a chi-squared distribution. Asymptotic results may not apply in small-sample situations, and the exact significance of a goodness-of-fit statistic may potentially be over- or under-stated by the asymptotic distribution. Several correction terms have been proposed to improve the accuracy of the asymptotic distribution, but their performance has only been studied for the equiprobable case. We extend that research to skewed hypotheses. Results are presented for one-way multinomials involving k = 2 to 6 cells with sample sizes N = 20, 40, 60, 80 and 100 and nominal test sizes f = 0.1, 0.05, 0.01 and 0.001. Six power-divergence goodness-of-fit statistics were investigated, and five correction terms were included in the study. Our results show that skewness itself does not affect the accuracy of the asymptotic approximation, which depends only on the magnitude of the smallest expected frequency (whether this comes from a small sample with the equiprobable hypothesis or a large sample with a skewed hypothesis). Throughout the conditions of the study, the accuracy of the asymptotic distribution seems to be optimal for Pearson's X² statistic (the power-divergence statistic of index u = 1) when k > 3 and the smallest expected frequency is as low as between 0.1 and 1.5 (depending on the particular k, N and nominal test size), but a computationally inexpensive improvement can be obtained in these cases by using a moment-corrected h² distribution. If the smallest expected frequency is even smaller, a normal correction yields accurate tests through the log-likelihood-ratio statistic G² (the power-divergence statistic of index u = 0).     

An Affine-Invariant Generalization of the Wilcoxon Signed-Rank Test for the Bivariate Location Problem

This paper proposes an affine‐invariant test extending the univariate Wilcoxon signed‐rank test to the bivariate location problem. It gives two versions of the null distribution of the test statistic. The first version leads to a conditionally distribution‐free test which can be used with any sample size. The second version can be used for larger sample sizes and has a limiting χ₂² distribution under the null hypothesis. The paper investigates the relationship with a test proposed by Jan & Randles (1994). It shows that the Pitman efficiency of this test relative to the new test is equal to 1 for elliptical distributions but that the two tests are not necessarily equivalent for non‐elliptical distributions. These facts are also demonstrated empirically in a simulation study. The new test has the advantage of not requiring the assumption of elliptical symmetry which is needed to perform the asymptotic version of the Jan and Randles test.     

A test for equality of variances with censored samples

A variance homogeneity test for type II right-censored samples is proposed. The test is based on Bartlett's statistic. The asymptotic distribution of the statistic is investigated. The limiting distribution is that of a linear combination of i.i.d. chi-square variables with 1 degree of freedom. By using simulation, the critical values of the null distribution of the modified Bartlett's statistic for testing the homogeneity of variances of two normal populations are obtained when the sample sizes and censoring levels are not equal. Also, we investigate the properties of the proposed test (size, power and robustness). Results show that the distribution of the test statistic depends on the censoring level. An example of the use of the new methodology in animal science involving reproduction in ewes is provided.     

Mood's test for dispersion as a counting of triplets

The well-known equivalence of Wilcoxon and Mann-Whitney location statistics is herein extended to dispersion tests. Mood (1954) statistic is related to a statistic based on “triplets”. The triplet version of Mood statistic is useful for proving the asymptotic normality (under alternatives) of the test.     

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.     

Cressie–Read Power-Divergence Statistics for Non-Gaussian Vector Stationary Processes

Abstract.  For a class of vector-valued non-Gaussian stationary processes, we develop the Cressie–Read power-divergence (CR) statistic approach which has been proposed for the i.i.d. case. The CR statistic includes empirical likelihood as a special case. Therefore, by adopting this CR statistic approach, the theory of estimation and testing based on empirical likelihood is greatly extended. We use an extended Whittle likelihood as score function and derive the asymptotic distribution of the CR statistic. We apply this result to estimation of autocorrelation and the AR coefficient, and get narrower confidence intervals than those obtained by existing methods. We also consider the power properties of the test based on asymptotic theory. Under a sequence of contiguous local alternatives, we derive the asymptotic distribution of the CR statistic. The problem of testing autocorrelation is discussed and we introduce some interesting properties of the local power.     

Spectral Density Based Goodness-of-Fit Tests for Time Series Models

A new goodness-of-fit test for time series models is proposed. The test statistic is based on the distance between a kernel estimator of the ratio between the true and the hypothesized spectral density and the expected value of the estimator under the null. It provides a quantification of how well a parametric spectral density model fits the sample spectral density (periodogram). The asymptotic distribution of the statistic proposed is derived and its power properties are discussed. To improve upon the large sample (Gaussian) approximation of the distribution of the test statistic under the null, a bootstrap procedure is presented and justified theoretically. The finite sample performance of the test is investigated through a simulation experiment and applications to real data sets are given.     

Optimal two-stage designs for phase ii clinical trials with differentiation of complete and partial responses

In the usual design and analysis of a phase II trial, there is no differentiation between complete response and partial response. Since complete response is considered more desirable this paper proposes a weighted score method which extends Simon's (1989) two-stage design to the situation where the complete and partial responses are differentiated. The weight assigned to the complete response is suggested by examining the likelihood ratio (LR) statistic for testing a simple hypothesis of a trinomial distribution. Both optimal and minimax designs are tabulated for a wide range of design parameters. The weighted score approach is shown to give more efficient designs, especially when the response probability is moderate to large.     

Comparison of Confidence Intervals on Between Group Variance in Unbalanced Heteroscedastic One-Way Random Models

A computer algorithm for computing the alternative distributions of the Wilcoxon signed rank statistic under shift alternatives is discussed. An explicit error bound is derived for the numeric integration approximation to these distributions.

A nonparametric process control procedure in which the standard CUSUM procedure is applied to the Wilcoxon signed rank statistic is discussed. In order to implement this procedure, the distribution of the Wilcoxon statistic under shift of the underlying distribution from its point of symmetry needs to be computed. The average run length of the nonparametric and parametric CUSUM are compared.     

Testing transition probability matrix of a multi-state model with censored data

In this paper, we develop procedures to test hypotheses concerning transition probability matrices arising from certain nonhomogeneous Markov processes. It is assumed that the data consist of sample paths, some of which are observed until a certain terminal state, and the other paths are censored. Problems of this type arise in the context of multi-state models relevant to Health Related Quality of Life (HRQoL) and Competing Risks. The test statistic is based on the estimator for the associated intensity matrix. We show that the asymptotic null distribution of the proposed statistic is Gaussian, and demonstrate how the procedure can be adopted for HRQoL studies and competing risks model using real data sets. Finally, we establish that the test statistic for the HRQoL has greatest local asymptotic power against a sequence of proportional hazards alternatives converging to the null hypothesis.     

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.