Addressing potential prior‐data conflict when using informative priors in proof‐of‐concept studies

Bayesian methods are increasingly used in proof‐of‐concept studies. An important benefit of these methods is the potential to use informative priors, thereby reducing sample size. This is particularly relevant for treatment arms where there is a substantial amount of historical information such as placebo and active comparators. One issue with using an informative prior is the possibility of a mismatch between the informative prior and the observed data, referred to as prior‐data conflict. We focus on two methods for dealing with this: a testing approach and a mixture prior approach. The testing approach assesses prior‐data conflict by comparing the observed data to the prior predictive distribution and resorting to a non‐informative prior if prior‐data conflict is declared. The mixture prior approach uses a prior with a precise and diffuse component. We assess these approaches for the normal case via simulation and show they have some attractive features as compared with the standard one‐component informative prior. For example, when the discrepancy between the prior and the data is sufficiently marked, and intuitively, one feels less certain about the results, both the testing and mixture approaches typically yield wider posterior‐credible intervals than when there is no discrepancy. In contrast, when there is no discrepancy, the results of these approaches are typically similar to the standard approach. Whilst for any specific study, the operating characteristics of any selected approach should be assessed and agreed at the design stage; we believe these two approaches are each worthy of consideration. Copyright © 2015 John Wiley & Sons, Ltd.     

Bayesian approach to epsilon-skew-normal family

The estimation problem of epsilon-skew-normal (ESN) distribution parameters is considered within Bayesian approaches. This family of distributions contains the normal distribution, can be used for analyzing the asymmetric and near-normal data. Bayesian estimates under informative and non informative Jeffreys prior distributions are obtained and performances of ESN family and these estimates are shown via a simulation study. A real data set is also used to illustrate the ideas.     

Anova and minque type of estimators for the one-way random effects model

The one-way random effects model with unequal variances and unequal sample sizes is considered. Estimation of the variances, variance of a single observation (total variance), and the standard error of the unweighted mean are considered. Precision of the Analysis of Variance and Unweighted Sums of Squares type of estimators and the Minimum Norm Quadratic Unbiased Estimators with a priori weights are examined.     

Bayesian composite Tobit quantile regression

Composite quantile regression models have been shown to be effective techniques in improving the prediction accuracy [H. Zou and M. Yuan, Composite quantile regression and the oracle model selection theory, Ann. Statist. 36 (2008), pp. 1108–1126; J. Bradic, J. Fan, and W. Wang, Penalized composite quasi-likelihood for ultrahighdimensional variable selection, J. R. Stat. Soc. Ser. B 73 (2011), pp. 325–349; Z. Zhao and Z. Xiao, Efficient regressions via optimally combining quantile information, Econometric Theory 30(06) (2014), pp. 1272–1314]. This paper studies composite Tobit quantile regression (TQReg) from a Bayesian perspective. A simple and efficient MCMC-based computation method is derived for posterior inference using a mixture of an exponential and a scaled normal distribution of the skewed Laplace distribution. The approach is illustrated via simulation studies and a real data set. Results show that combine information across different quantiles can provide a useful method in efficient statistical estimation. This is the first work to discuss composite TQReg from a Bayesian perspective.     

Use of a historical control group in a noninferiority trial assessing a new antibacterial treatment: A case study and discussion of practical implementation aspects

When recruitment into a clinical trial is limited due to rarity of the disease of interest, or when recruitment to the control arm is limited due to ethical reasons (eg, pediatric studies or important unmet medical need), exploiting historical controls to augment the prospectively collected database can be an attractive option. Statistical methods for combining historical data with randomized data, while accounting for the incompatibility between the two, have been recently proposed and remain an active field of research. The current literature is lacking a rigorous comparison between methods but also guidelines about their use in practice. In this paper, we compare the existing methods based on a confirmatory phase III study design exercise done for a new antibacterial therapy with a binary endpoint and a single historical dataset. A procedure to assess the relative performance of the different methods for borrowing information from historical control data is proposed, and practical questions related to the selection and implementation of methods are discussed. Based on our examination, we found that the methods have a comparable performance, but we recommend the robust mixture prior for its ease of implementation.     

Reference priors for exponential families

Reference analysis, introduced by Bernardo (J. Roy. Statist. Soc. 41 (1979) 113) and further developed by Berger and Bernardo (On the development of reference priors (with discussion). In: J.M. Bernardo, J.O. Berger, A.P. Dawid, A.F.M. Smith (Eds.), Bayesian Statistics, Vol. 4, Clarendon Press, Oxford, pp. 35–60), has proved to be one of the most successful general methods to derive noninformative prior distributions. In practice, however, reference priors are typically difficult to obtain. In this paper we show how to find reference priors for a wide class of exponential family likelihoods.     

Bayesian methods for generalized linear models with covariates missing at random

The authors propose methods for Bayesian inference for generalized linear models with missing covariate data. They specify a parametric distribution for the covariates that is written as a sequence of one‐dimensional conditional distributions. They propose an informative class of joint prior distributions for the regression coefficients and the parameters arising from the covariate distributions. They examine the properties of the proposed prior and resulting posterior distributions. They also present a Bayesian criterion for comparing various models, and a calibration is derived for it. A detailed simulation is conducted and two real data sets are examined to demonstrate the methodology.     

Engaging Indigeneity in Development Policy

This article explores how development programming in rural poverty and the environment can work with indigenous peoples. It draws on research conducted in Asia and Latin America to suggest how indigeneity can be understood as specific kinds of marginalisation intersecting with self‐identification and recognition as indigenous. Current obstacles to effective engagement with indigenous peoples are outlined, and suggestions are offered for pro‐actively addressing their experience. Two critical areas where there are opportunities for donors to support indigenous peoples' priorities include ‘FPIC’ (Free Prior and Informed Consent) and a careful consideration of the implications of niche‐market engagement.     

A Bayesian method of sample size determination with practical applications

Summary.  The problem motivating the paper is the determination of sample size in clinical trials under normal likelihoods and at the substantive testing stage of a financial audit where normality is not an appropriate assumption. A combination of analytical and simulation-based techniques within the Bayesian framework is proposed. The framework accommodates two different prior distributions: one is the general purpose fitting prior distribution that is used in Bayesian analysis and the other is the expert subjective prior distribution, the sampling prior which is believed to generate the parameter values which in turn generate the data. We obtain many theoretical results and one key result is that typical non-informative prior distributions lead to very small sample sizes. In contrast, a very informative prior distribution may either lead to a very small or a very large sample size depending on the location of the centre of the prior distribution and the hypothesized value of the parameter. The methods that are developed are quite general and can be applied to other sample size determination problems. Some numerical illustrations which bring out many other aspects of the optimum sample size are given.     

Linear statistics in change-point estimation and their asymptotic behaviour

ABSTRACT The limiting behaviour of Bayes procedures in the asymptotic setting of the change-point estimation problem is studied. It is shown that the distribution of the difference between the Bayes estimator and the parameter converges to the distribution of a fairly complicated random variable. A class of linear statistics is introduced, and the form of the Bayes estimator within this class is deduced. The asymptotic properties of this linear estimator are investigated in two different settings for the prior distribution.