Goodness‐of‐fit Test for Directional Data

In this paper, we study the problem of testing the hypothesis on whether the density f of a random variable on a sphere belongs to a given parametric class of densities. We propose two test statistics based on the L² and L¹ distances between a non‐parametric density estimator adapted to circular data and a smoothed version of the specified density. The asymptotic distribution of the L² test statistic is provided under the null hypothesis and contiguous alternatives. We also consider a bootstrap method to approximate the distribution of both test statistics. Through a simulation study, we explore the moderate sample performance of the proposed tests under the null hypothesis and under different alternatives. Finally, the procedure is illustrated by analysing a real data set based on wind direction measurements.     

Simultaneous confidence interval for assessing non‐inferiority with assay sensitivity in a three‐arm trial with binary endpoints

A three‐arm trial including an experimental treatment, an active reference treatment and a placebo is often used to assess the non‐inferiority (NI) with assay sensitivity of an experimental treatment. Various hypothesis‐test‐based approaches via a fraction or pre‐specified margin have been proposed to assess the NI with assay sensitivity in a three‐arm trial. There is little work done on confidence interval in a three‐arm trial. This paper develops a hybrid approach to construct simultaneous confidence interval for assessing NI and assay sensitivity in a three‐arm trial. For comparison, we present normal‐approximation‐based and bootstrap‐resampling‐based simultaneous confidence intervals. Simulation studies evidence that the hybrid approach with the Wilson score statistic performs better than other approaches in terms of empirical coverage probability and mesial‐non‐coverage probability. An example is used to illustrate the proposed approaches.     

Homogeneity test, sample size determination and interval construction of difference of two proportions in stratified bilateral-sample designs

In stratified otolaryngologic (or ophthalmologic) studies, the misleading results may be obtained when ignoring the confounding effect and the correlation between responses from two ears. Score statistic and Wald-type statistic are presented to test equality in a stratified bilateral-sample design, and their corresponding sample size formulae are given. Score statistic for testing homogeneity of difference between two proportions and score confidence interval of the common difference of two proportions in a stratified bilateral-sample design are derived. Empirical results show that (1) score statistic and Wald-type statistic based on dependence model assumption outperform other statistics in terms of the type I error rates; (2) score confidence interval demonstrates reasonably good coverage property; (3) sample size formula via Wald-type statistic under dependence model assumption is rather accurate. A real example is used to illustrate the proposed methodologies.     

Comparing Conditional Quantile Curves

Abstract. We consider the problem of testing the equality of J quantile curves from independent samples. A test statistic based on an L²‐distance between non‐crossing non‐parametric estimates of the quantile curves from the individual samples is proposed. Asymptotic normality of this statistic is established under the null hypothesis, local and fixed alternatives, and the finite sample properties of a bootstrap‐based version of this test statistic are investigated by means of a simulation study.     

Which Wald statistic? Choosing a parameterization of the Wald statistic to maximize power in k-sample generalized estimating equations

The Wald statistic is known to vary under reparameterization. This raises the question: which parameterization should be chosen, in order to optimize power of the Wald statistic? We specifically consider k-sample tests of generalized linear models (GLMs) and generalized estimating equations (GEEs) in which the alternative hypothesis contains only two parameters. An example is presented in which such an alternative hypothesis is of interest. Amongst a general class of parameterizations, we find the parameterization that maximizes power via analysis of the non-centrality parameter, and show how the effect on power of reparameterization depends on sampling design and the differences in variance across samples. There is no single parameterization with optimal power across all alternatives. The Wald statistic commonly used under the canonical parameterization is optimal in some instances but it performs very poorly in others. We demonstrate results by example and by simulation, and describe their implications for likelihood ratio statistics and score statistics. We conclude that due to poor power properties, the routine use of score statistics and Wald statistics under the canonical parameterization for GEEs is a questionable practice.     

Confidence Intervals for the Current Status Model

We discuss a new way of constructing pointwise confidence intervals for the distribution function in the current status model. The confidence intervals are based on the smoothed maximum likelihood estimator, using local smooth functional theory and normal limit distributions. Bootstrap methods for constructing these intervals are considered. Other methods to construct confidence intervals, using the non‐standard limit distribution of the (restricted) maximum likelihood estimator, are compared with our approach via simulations and real data applications.     

Adjusted Supremum Score‐Type Statistics for Evaluating Non‐Standard Hypotheses

Supremum score test statistics are often used to evaluate hypotheses with unidentifiable nuisance parameters under the null hypothesis. Although these statistics provide an attractive framework to address non‐identifiability under the null hypothesis, little attention has been paid to their distributional properties in small to moderate sample size settings. In situations where there are identifiable nuisance parameters under the null hypothesis, these statistics may behave erratically in realistic samples as a result of a non‐negligible bias induced by substituting these nuisance parameters by their estimates under the null hypothesis. In this paper, we propose an adjustment to the supremum score statistics by subtracting the expected bias from the score processes and show that this adjustment does not alter the limiting null distribution of the supremum score statistics. Using a simple example from the class of zero‐inflated regression models for count data, we show empirically and theoretically that the adjusted tests are superior in terms of size and power. The practical utility of this methodology is illustrated using count data in HIV research.     

Inference from PseudoLikelihoods with Plug‐In Estimates

Effective implementation of likelihood inference in models for high‐dimensional data often requires a simplified treatment of nuisance parameters, with these having to be replaced by handy estimates. In addition, the likelihood function may have been simplified by means of a partial specification of the model, as is the case when composite likelihood is used. In such circumstances tests and confidence regions for the parameter of interest may be constructed using Wald type and score type statistics, defined so as to account for nuisance parameter estimation or partial specification of the likelihood. In this paper a general analytical expression for the required asymptotic covariance matrices is derived, and suggestions for obtaining Monte Carlo approximations are presented. The same matrices are involved in a rescaling adjustment of the log likelihood ratio type statistic that we propose. This adjustment restores the usual chi‐squared asymptotic distribution, which is generally invalid after the simplifications considered. The practical implication is that, for a wide variety of likelihoods and nuisance parameter estimates, confidence regions for the parameters of interest are readily computable from the rescaled log likelihood ratio type statistic as well as from the Wald type and score type statistics. Two examples, a measurement error model with full likelihood and a spatial correlation model with pairwise likelihood, illustrate and compare the procedures. Wald type and score type statistics may give rise to confidence regions with unsatisfactory shape in small and moderate samples. In addition to having satisfactory shape, regions based on the rescaled log likelihood ratio type statistic show empirical coverage in reasonable agreement with nominal confidence levels.     

Simultaneous tests for homogeneity of two zero-inflated (beta) populations

ABSTRACT

Motivated by an example in marine science, we use Fisher’s method to combine independent likelihood ratio tests (LRTs) and asymptotic independent score tests to assess the equivalence of two zero-inflated Beta populations (mixture distributions with three parameters). For each test, test statistics for the three individual parameters are combined into a single statistic to address the overall difference between the two populations. We also develop non parametric and semiparametric permutation-based tests for simultaneously comparing two or three features of unknown populations. Simulations show that the likelihood-based tests perform well for large sample sizes and that the statistics based on combining LRT statistics outperforms the ones based on combining score test statistics. The permutation-based tests have overall better performance in terms of both power and type I error rate. Our methods are easy to implement and computationally efficient, and can be expanded to more than two populations and to other multiple parameter families. The permutation tests are entirely generic and can be useful in various applications dealing with zero (or other) inflation.     

Strengthening the understanding of the relationship between survival designs and test statistics

In the planning of randomized survival trials, the role of follow‐up time of trial participants introduces a level of complexity not encountered in non‐survival trials. Of the two commonly used survival designs, one design fixes the follow‐up time whereas the other allows it to vary. When the follow‐up time is fixed the number of events varies. Conversely, when the number of events is fixed, the follow‐up time varies. These two designs influence test statistics in ways that have not been fully explored resulting in a misunderstanding of the design–test statistic relationship. We use examples from the literature to strengthen the understanding of this relationship. Group sequential trials are briefly discussed. When the number of events is fixed, we demonstrate why a two‐sample risk difference test statistic reduces to a one‐sample test statistic which is nearly equal to the risk ratio test statistic. Some aspects of fixed event designs that need further consideration are also discussed. Copyright © 2009 John Wiley & Sons, Ltd.     

Goodness-of-fit statistics for log-link regression models

The use of log binomial regression, regression on binary outcomes using a log link, is becoming increasingly popular because it provides estimates of relative risk. However, little work has been done on model evaluation. We used simulations to compare the performance of five goodness-of-fit statistics applied to different models in a log binomial setting, namely the Hosmer–Lemeshow, the normalized Pearson chi-square, the normalized unweighted sum of squares, Le Cessie and van Howelingen's statistic based on smoothed residuals and the Hjort–Hosmer test. The normalized Pearson chi-square was unsuitable as the rejection rate depended also on the range of predicted probabilities. The Le Cessie and van Howelingen's test statistic had poor sampling properties when evaluating a correct model and was also considered to be unsuitable in this context. The performance of the remaining three statistics was comparable in most simulations. However, using real data the Hjort–Hosmer outperformed the other two statistics.     

The score test for the two‐sample occupancy model

The score test statistic from the observed information is easy to compute numerically. Its large sample distribution under the null hypothesis is well known and is equivalent to that of the score test based on the expected information, the likelihood‐ratio test and the Wald test. However, several authors have noted that under the alternative hypothesis this no longer holds and in particular the score statistic from the observed information can take negative values. We extend the anthology on the score test to a problem of interest in ecology when studying species occurrence. This is the comparison of two zero‐inflated binomial random variables from two independent samples under imperfect detection. An analysis of eigenvalues associated with the score test in this setting assists in understanding why using the observed information matrix in the score test can be problematic. We demonstrate through a combination of simulations and theoretical analysis that the power of the score test calculated under the observed information decreases as the populations being compared become more dissimilar. In particular, the score test based on the observed information is inconsistent. Finally, we propose a modified rule that rejects the null hypothesis when the score statistic is computed using the observed information is negative or is larger than the usual chi‐square cut‐off. In simulations in our setting this has power that is comparable to the Wald and likelihood ratio tests and consistency is largely restored. Our new test is easy to use and inference is possible. Supplementary material for this article is available online as per journal instructions.     

A smoothed Q‐learning algorithm for estimating optimal dynamic treatment regimes

In this paper, we propose a smoothed Q‐learning algorithm for estimating optimal dynamic treatment regimes. In contrast to the Q‐learning algorithm in which nonregular inference is involved, we show that, under assumptions adopted in this paper, the proposed smoothed Q‐learning estimator is asymptotically normally distributed even when the Q‐learning estimator is not and its asymptotic variance can be consistently estimated. As a result, inference based on the smoothed Q‐learning estimator is standard. We derive the optimal smoothing parameter and propose a data‐driven method for estimating it. The finite sample properties of the smoothed Q‐learning estimator are studied and compared with several existing estimators including the Q‐learning estimator via an extensive simulation study. We illustrate the new method by analyzing data from the Clinical Antipsychotic Trials of Intervention Effectiveness–Alzheimer's Disease (CATIE‐AD) study.     

Mean shift and influence measures in linear measurement error models with stochastic linear restrictions

We present influence diagnostics for linear measurement error models with stochastic linear restrictions using the corrected likelihood of Nakamura in 1990. The case deletion and mean shift outlier models are developed to identify outlying and influential observations. We derive a corrected score test statistic for outlier detection based on mean shift outlier models. The analogs of Cook's distance and likelihood distance are proposed to determine influential observations based on case deletion models. A parametric bootstrap procedure is used to obtain empirical distributions of the test statistics and a simulation study has been used to evaluate the performance of the proposed estimators based on the mean squares error criterion and the score test statistic. Finally, a numerical example is given to illustrate the theoretical results.     

Two‐Sample Test Against One‐Sided Alternatives

Abstract. This paper proposes, implements and investigates a new non‐parametric two‐sample test for detecting stochastic dominance. We pose the question of detecting the stochastic dominance in a non‐standard way. This is motivated by existing evidence showing that standard formulations and pertaining procedures may lead to serious errors in inference. The procedure that we introduce matches testing and model selection. More precisely, we reparametrize the testing problem in terms of Fourier coefficients of well‐known comparison densities. Next, the estimated Fourier coefficients are used to form a kind of signed smooth rank statistic. In such a setting, the number of Fourier coefficients incorporated into the statistic is a smoothing parameter. We determine this parameter via some flexible selection rule. We establish the asymptotic properties of the new test under null and alternative hypotheses. The finite sample performance of the new solution is demonstrated through Monte Carlo studies and an application to a set of survival times.     

Robust inference from multiple test statistics via permutations: a better alternative to the single test statistic approach for randomized trials

Formal inference in randomized clinical trials is based on controlling the type I error rate associated with a single pre‐specified statistic. The deficiency of using just one method of analysis is that it depends on assumptions that may not be met. For robust inference, we propose pre‐specifying multiple test statistics and relying on the minimum p‐value for testing the null hypothesis of no treatment effect. The null hypothesis associated with the various test statistics is that the treatment groups are indistinguishable. The critical value for hypothesis testing comes from permutation distributions. Rejection of the null hypothesis when the smallest p‐value is less than the critical value controls the type I error rate at its designated value. Even if one of the candidate test statistics has low power, the adverse effect on the power of the minimum p‐value statistic is not much. Its use is illustrated with examples. We conclude that it is better to rely on the minimum p‐value rather than a single statistic particularly when that single statistic is the logrank test, because of the cost and complexity of many survival trials. Copyright © 2013 John Wiley & Sons, Ltd.     

Goodness-of-fit test under length-biased sampling

In this article, we study a goodness-of-fit (GOF) test in the presence of length-biased sampling. For this purpose, we introduce a smoothed estimator of distribution function (d.f.) and we investigate its asymptotic behaviors, such as uniform consistency and asymptotic normality. Based on this estimator, we define a one-sample Kolmogorov type of GOF test for length-biased data. We conduct Monte Carlo simulations to evaluate the performance of the proposed test statistic and compare it with the one-sample Kolmogorov type of GOF test obtained by the non smoothed estimator of d.f.     

Exact unconditional testing procedures for comparing two independent Poisson rates

We consider seven exact unconditional testing procedures for comparing adjusted incidence rates between two groups from a Poisson process. Exact tests are always preferable due to the guarantee of test size in small to medium sample settings. Han [Comparing two independent incidence rates using conditional and unconditional exact tests. Pharm Stat. 2008;7(3):195–201] compared the performance of partial maximization p-values based on the Wald test statistic, the likelihood ratio test statistic, the score test statistic, and the conditional p-value. These four testing procedures do not perform consistently, as the results depend on the choice of test statistics for general alternatives. We consider the approach based on estimation and partial maximization, and compare these to the ones studied by Han (2008) for testing superiority. The procedures are compared with regard to the actual type I error rate and power under various conditions. An example from a biomedical research study is provided to illustrate the testing procedures. The approach based on partial maximization using the score test is recommended due to the comparable performance and computational advantage in large sample settings. Additionally, the approach based on estimation and partial maximization performs consistently for all the three test statistics, and is also recommended for use in practice.     

Hypothesis Testing in Functional Comparative Calibration Models

The purpose of this article is to investigate hypothesis testing in functional comparative calibration models. Wald type statistics are considered which are asymptotically distributed according to the chi-square distribution. The statistics are based on maximum likelihood, corrected score approach, and method of moment estimators of the model parameters, which are shown to be consistent and asymptotically normally distributed. Results of analytical and simulation studies seem to indicate that the Wald statistics based on the method of moment estimators and the corrected score estimators are, as expected, less efficient than the Wald type statistic based on the maximum likelihood estimators for small n. Wald statistic based on moment estimators are simpler to compute than the other Wald statistics tests and their performance improves significantly as n increases. Comparisons with an alternative F statistics proposed in the literature are also reported.     

Multivariate association test for rare variants controlling for cryptic and family relatedness

In genetic studies of complex diseases, multiple measures of related phenotypes are often collected. Jointly analyzing these phenotypes may improve statistical power to detect sets of rare variants affecting multiple traits. In this work, we consider association testing between a set of rare variants and multiple phenotypes in family‐based designs. We use a mixed linear model to express the correlations among the phenotypes and between related individuals. Given the many sources of correlations in this situation, deriving an appropriate test statistic is not straightforward. We derive a vector of score statistics, whose joint distribution is approximated using a copula. This allows us to have closed‐form expressions for the p‐values of several test statistics. A comprehensive simulation study and an application to Genetic Analysis Workshop 18 data highlight the gains associated with joint testing over univariate approaches, especially in the presence of pleiotropy or highly correlated phenotypes. The Canadian Journal of Statistics 47: 90–107; 2019 © 2018 Statistical Society of Canada