Comparison of dichotomized and distributional approaches in rare event clinical trial design: a fixed Bayesian design

This research was motivated by our goal to design an efficient clinical trial to compare two doses of docosahexaenoic acid supplementation for reducing the rate of earliest preterm births (ePTB) and/or preterm births (PTB). Dichotomizing continuous gestational age (GA) data using a classic binomial distribution will result in a loss of information and reduced power. A distributional approach is an improved strategy to retain statistical power from the continuous distribution. However, appropriate distributions that fit the data properly, particularly in the tails, must be chosen, especially when the data are skewed. A recent study proposed a skew-normal method. We propose a three-component normal mixture model and introduce separate treatment effects at different components of GA. We evaluate operating characteristics of mixture model, beta-binomial model, and skew-normal model through simulation. We also apply these three methods to data from two completed clinical trials from the USA and Australia. Finite mixture models are shown to have favorable properties in PTB analysis but minimal benefit for ePTB analysis. Normal models on log-transformed data have the largest bias. Therefore we recommend finite mixture model for PTB study. Either finite mixture model or beta-binomial model is acceptable for ePTB study.     

Empirical Uncertain Bayes Methods in Area‐level Models

Random effects model can account for the lack of fitting a regression model and increase precision of estimating area‐level means. However, in case that the synthetic mean provides accurate estimates, the prior distribution may inflate an estimation error. Thus, it is desirable to consider the uncertain prior distribution, which is expressed as the mixture of a one‐point distribution and a proper prior distribution. In this paper, we develop an empirical Bayes approach for estimating area‐level means, using the uncertain prior distribution in the context of a natural exponential family, which we call the empirical uncertain Bayes (EUB) method. The regression model considered in this paper includes the Poisson‐gamma and the binomial‐beta, and the normal‐normal (Fay–Herriot) model, which are typically used in small area estimation. We obtain the estimators of hyperparameters based on the marginal likelihood by using a well‐known expectation‐maximization algorithm and propose the EUB estimators of area means. For risk evaluation of the EUB estimator, we derive a second‐order unbiased estimator of a conditional mean squared error by using some techniques of numerical calculation. Through simulation studies and real data applications, we evaluate a performance of the EUB estimator and compare it with the usual empirical Bayes estimator.     

Tests for Structural Changes in Time Series of Counts

We propose methods for detecting structural changes in time series with discrete‐valued observations. The detector statistics come in familiar L2‐type formulations incorporating the empirical probability generating function. Special emphasis is given to the popular models of integer autoregression and Poisson autoregression. For both models, we study mainly structural changes due to a change in distribution, but we also comment for the classical problem of parameter change. The asymptotic properties of the proposed test statistics are studied under the null hypothesis as well as under alternatives. A Monte Carlo power study on bootstrap versions of the new methods is also included along with a real data example.     

Score test for homogeneity of dispersion in generalized Poisson mixed models with excess zeros

In many applications, the clustered count data often contain excess zeros and the zero-inflated generalized Poisson mixed (ZIGPM) regression model may be suitable. However, dispersion in ZIGPM is often treated as fixed unknown parameter, and this assumption may be not appropriate in some situations. In this article, a score test for homogeneity of dispersion parameter in ZIGPM regression model is developed and corresponding test statistic is obtained. Sampling distribution and power of the score test statistic are investigated through Monte Carlo simulation. Finally, results from a biological example illustrate the usefulness of the diagnostic statistic.     

Robust modal estimation and variable selection for single-index varying-coefficient models

In this article, we present a new efficient iteration estimation approach based on local modal regression for single-index varying-coefficient models. The resulted estimators are shown to be robust with regardless of outliers and error distributions. The asymptotic properties of the estimators are established under some regularity conditions and a practical modified EM algorithm is proposed for the new method. Moreover, to achieve sparse estimator when there exists irrelevant variables in the index parameters, a variable selection procedure based on SCAD penalty is developed to select significant parametric covariates and the well-known oracle properties are also derived. Finally, some numerical examples with various distributed errors and a real data analysis are conducted to illustrate the validity and feasibility of our proposed method.     

A note on efficient simulation of multidimensional spatial autoregressive processes

In applications of spatial statistics, it is necessary to compute the product of some matrix W of spatial weights and a vector y of observations. The weighting matrix often needs to be adapted to the specific problems, such that the computation of Wy cannot necessarily be done with available R-packages. Hence, this article suggests one possibility treating such issues. The proposed technique avoids the computation of the matrix product by calculating each entry of Wy separately. Initially, a specific spatial autoregressive process is introduced. The performance of the proposed program is briefly compared to a basic program using the matrix multiplication.