An improved ranked set two‐sample mann‐whitney‐wilcoxon test

The authors present an improved ranked set two‐sample Mann‐Whitney‐Wilcoxon test for a location shift between samples from two distributions F and G. They define a function that measures the amount of information provided by each observation from the two samples, given the actual joint ranking of all the units in a set. This information function is used as a guide for improving the Pitman efficacy of the Mann‐Whitney‐Wilcoxon test. When the underlying distributions are symmetric, observations at their mode(s) must be quantified in order to gain efficiency. Analogous results are provided for asymmetric distributions.     

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.     

Distribution of the two‐sample t‐test statistic following blinded sample size re‐estimation

We consider the blinded sample size re‐estimation based on the simple one‐sample variance estimator at an interim analysis. We characterize the exact distribution of the standard two‐sample t‐test statistic at the final analysis. We describe a simulation algorithm for the evaluation of the probability of rejecting the null hypothesis at given treatment effect. We compare the blinded sample size re‐estimation method with two unblinded methods with respect to the empirical type I error, the empirical power, and the empirical distribution of the standard deviation estimator and final sample size. We characterize the type I error inflation across the range of standardized non‐inferiority margin for non‐inferiority trials, and derive the adjusted significance level to ensure type I error control for given sample size of the internal pilot study. We show that the adjusted significance level increases as the sample size of the internal pilot study increases. Copyright © 2016 John Wiley & Sons, Ltd.     

Sample size calculation for the one‐sample log‐rank test

In this paper, an exact variance of the one‐sample log‐rank test statistic is derived under the alternative hypothesis, and a sample size formula is proposed based on the derived exact variance. Simulation results showed that the proposed sample size formula provides adequate power to design a study to compare the survival of a single sample with that of a standard population. Copyright © 2014 John Wiley & Sons, Ltd.     

Increasing the sample size at interim for a two‐sample experiment without Type I error inflation

For the case of a one‐sample experiment with known variance σ²=1, it has been shown that at interim analysis the sample size (SS) may be increased by any arbitrary amount provided: (1) The conditional power (CP) at interim is ?50% and (2) there can be no decision to decrease the SS (stop the trial early). In this paper we verify this result for the case of a two‐sample experiment with proportional SS in the treatment groups and an arbitrary common variance. Numerous authors have presented the formula for the CP at interim for a two‐sample test with equal SS in the treatment groups and an arbitrary common variance, for both the one‐ and two‐sided hypothesis tests. In this paper we derive the corresponding formula for the case of unequal, but proportional SS in the treatment groups for both one‐sided superiority and two‐sided hypothesis tests. Finally, we present an SAS macro for doing this calculation and provide a worked out hypothetical example. In discussion we note that this type of trial design trades the ability to stop early (for lack of efficacy) for the elimination of the Type I error penalty. The loss of early stopping requires that such a design employs a data monitoring committee, blinding of the sponsor to the interim calculations, and pre‐planning of how much and under what conditions to increase the SS and that this all be formally written into an interim analysis plan before the start of the study. Copyright © 2009 John Wiley & Sons, Ltd.     

A nonparametric test for interaction in two‐way layouts

The authors present a new nonparametric approach to test for interaction in two‐way layouts. Based on the concept of composite linear rank statistics, they combine the correlated row and column ranking information to construct the test statistic. They determine the limiting distributions of the proposed test statistic under the null hypothesis and Pitman alternatives. They also propose consistent estimators for the limiting covariance matrices associated with the test. They illustrate the application of their test in practical settings using a microarray data set.     

Empirical likelihood tests for two‐sample problems via nonparametric density estimation

The authors study the problem of testing whether two populations have the same law by comparing kernel estimators of the two density functions. The proposed test statistic is based on a local empirical likelihood approach. They obtain the asymptotic distribution of the test statistic and propose a bootstrap approximation to calibrate the test. A simulation study is carried out in which the proposed method is compared with two competitors, and a procedure to select the bandwidth parameter is studied. The proposed test can be extended to more than two samples and to multivariate distributions.     

Graphical displays for clarifying how allocation ratio affects total sample size for the two sample logrank test

For time‐to‐event data, the power of the two sample logrank test for the comparison of two treatment groups can be greatly influenced by the ratio of the number of patients in each of the treatment groups. Despite the possible loss of power, unequal allocations may be of interest due to a need to collect more data on one of the groups or to considerations related to the acceptability of the treatments to patients. Investigators pursuing such designs may be interested in the cost of the unbalanced design relative to a balanced design with respect to the total number of patients required for the study. We present graphical displays to illustrate the sample size adjustment factor, or ratio of the sample size required by an unequal allocation compared to the sample size required by a balanced allocation, for various survival rates, treatment hazards ratios, and sample size allocation ratios. These graphical displays conveniently summarize information in the literature and provide a useful tool for planning sample sizes for the two sample logrank test. Copyright © 2010 John Wiley & Sons, Ltd.     

A review and re‐interpretation of a group‐sequential approach to sample size re‐estimation in two‐stage trials

In this paper, we review the adaptive design methodology of Li et al. (Biostatistics 3 :277–287) for two‐stage trials with mid‐trial sample size adjustment. We argue that it is closer in principle to a group sequential design, in spite of its obvious adaptive element. Several extensions are proposed that aim to make it even more attractive and transparent alternative to a standard (fixed sample size) trial for funding bodies to consider. These enable a cap to be put on the maximum sample size and for the trial data to be analysed using standard methods at its conclusion. The regulatory view of trials incorporating unblinded sample size re‐estimation is also discussed. © 2014 The Authors. Pharmaceutical Statistics published by John Wiley & Sons, Ltd.     

Partially replicated two‐level fractional factorial designs

In a two‐level factorial experiment, the authors consider designs with partial duplication which permit estimation of the constant term, all main effects and some specified two‐factor interactions, assuming that the other effects are negligible. They construct parallel‐flats designs with two identical parallel flats that meet prior specifications; they also consider classes of 3‐flat and 4‐flat designs. They show that the designs obtained can have a very simple covariance structure and high D‐efficiency. They give an algorithm from which they generate a series of practical designs with run sizes 12, 16, 24, and 32.     

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.     

One‐Way anova for Functional Data via Globalizing the Pointwise F‐test

In this paper, we propose and study a new global test, namely, GPF test, for the one‐way anova problem for functional data, obtained via globalizing the usual pointwise F‐test. The asymptotic random expressions of the test statistic are derived, and its asymptotic power is investigated. The GPF test is shown to be root‐n consistent. It is much less computationally intensive than a parametric bootstrap test proposed in the literature for the one‐way anova for functional data. Via some simulation studies, it is found that in terms of size‐controlling and power, the GPF test is comparable with two existing tests adopted for the one‐way anova problem for functional data. A real data example illustrates the GPF test.     

On the robustness of the power function of the one-sample test for the negative exponential distribution

The robustness of the power function of the standard one-sample parametric test for the mean of the negative exponential distribution is examined. The main form of departure from the exponential assumption is a mixture of negative exponential components although an alternative Gamma distribution is also examined. It is found that the test is sensitive to these departures although the effect of mixtures with short tails is less dramatic than those with long tails.     

Computation of the percentage points and the power for the two-sided Kolmogorov-Smirnov one sample test

Two recursive schemes are presented for the calculation of the probabilityP(g(x)≤S _n (x)≤h(x) for allx∈®), whereS _n is the empirical distribution function of a sample from a continuous distribution andh, g are continuous and isotone functions. The results are specialized for the calculation of the distribution and the corresponding percentage points of the test statistic of the two-sided Kolmogorov-Smirnov one sample test. The schemes allow the calculation of the power of the test too. Finally an extensive tabulation of percentage points for the Kolmogorov-Smirnov test is given.     

A resampling‐based test for two crossing survival curves

The area between two survival curves is an intuitive test statistic for the classical two‐sample testing problem. We propose a bootstrap version of it for assessing the overall homogeneity of these curves. Our approach allows ties in the data as well as independent right censoring, which may differ between the groups. The asymptotic distribution of the test statistic as well as of its bootstrap counterpart are derived under the null hypothesis, and their consistency is proven for general alternatives. We demonstrate the finite sample superiority of the proposed test over some existing methods in a simulation study and illustrate its application by a real‐data example.     

Sample‐size calculations for multi‐group comparison in population pharmacokinetic experiments

This paper describes an approach for calculating sample size for population pharmacokinetic experiments that involve hypothesis testing based on multi‐group comparison detecting the difference in parameters between groups under mixed‐effects modelling. This approach extends what has been described for generalized linear models and nonlinear population pharmacokinetic models that involve only binary covariates to more complex nonlinear population pharmacokinetic models. The structural nonlinear model is linearized around the random effects to obtain the marginal model and the hypothesis testing involving model parameters is based on Wald's test. This approach provides an efficient and fast method for calculating sample size for hypothesis testing in population pharmacokinetic models. The approach can also handle different design problems such as unequal allocation of subjects to groups and unbalanced sampling times between and within groups. The results obtained following application to a one compartment intravenous bolus dose model that involved three different hypotheses under different scenarios showed good agreement between the power obtained from NONMEM simulations and nominal power. Copyright © 2009 John Wiley & Sons, Ltd.     

Two‐Sample Bootstrap Hypothesis Tests for Three‐Dimensional Labelled Landmark Data

Abstract. We investigate resampling methodologies for testing the null hypothesis that two samples of labelled landmark data in three dimensions come from populations with a common mean reflection shape or mean reflection size‐and‐shape. The investigation includes comparisons between (i) two different test statistics that are functions of the projection onto tangent space of the data, namely the James statistic and an empirical likelihood statistic; (ii) bootstrap and permutation procedures; and (iii) three methods for resampling under the null hypothesis, namely translating in tangent space, resampling using weights determined by empirical likelihood and using a novel method to transform the original sample entirely within refection shape space. We present results of extensive numerical simulations, on which basis we recommend a bootstrap test procedure that we expect will work well in practise. We demonstrate the procedure using a data set of human faces, to test whether humans in different age groups have a common mean face shape.     

Nonparametric test for checking lack of fit of the quantité regression model under random censoring

The author proposes a nonparametric test for checking the lack of fit of the quantile function of survival time given the covariates; she assumes that survival time is subjected to random right censoring. Her test statistic is a kemel‐based smoothing estimator of a moment condition. The test statistic is asymptotically Gaussian under the null hypothesis. The author investigates its behavior under local alternative sequences. She assesses its finite‐sample power through simulations and illustrates its use with the Stanford heart transplant data.