On Kronecker block designs for factorial experiments

In this paper the use of Kronecker designs for factorial experiments is considered. The two-factor Kronecker design is considered in some detail and the efficiency factors of the main effects and interaction in such a design are derived. It is shown that the efficiency factor of the interaction is at least as large as the product of the efficiency factors of the two main effects and when both the component designs are totally balanced then its efficiency factor will be higher than the efficiency factor of either of the two main effects. If the component designs are nearly balanced then its efficiency factor will be approximately at least as large as the efficiency factor of either of the two main effects. It is argued that these designs are particularly useful for factorial experiments.Extensions to the multi-factor design are given and it is proved that the two-factor Kronecker design will be connected if the component designs are connected.     

On some new search designs for 2m factorial experiments

In this paper, we obtain search designs with reasonably small number of treatments which permit the estimation of the general mean and main effects and search of one more unknown possible nonzero effect among two and three factor interactions in 2^m factorial experiments, 3 ? m ? 8.     

Using ranked set sampling with binary outcomes in cluster randomized designs

We study the use of ranked set sampling (RSS) with binary outcomes in cluster-randomized designs (CRDs), where a generalized linear mixed model (GLMM) is used to model the hierarchical data structure involved. Under the GLMM-based framework, we propose three different approaches to estimate the treatment effect, including the nonparametric (NP), maximum likelihood (ML) and pseudo likelihood (PL) estimators. We investigate their asymptotic properties and examine their finite-sample performance via simulation. Based on these three RSS estimators, we further develop procedures for testing the existence of the treatment effect. We examine the power and size of our proposed RSS tests and compare them with existing tests based on simple random sampling (SRS). All the proposed RSS estimation and test methods are illustrated with two data examples, one for rare events and the other for non-extreme events. Throughout our investigations, we also consider the possible effect of imperfect ranking. Among the proposed methods, we provide recommendations on whether to use RSS rather than SRS with binary outcomes in CRDs and, if yes, when to use which RSS method. The Canadian Journal of Statistics 48: 342–365; 2020 © 2019 Statistical Society of Canada     

Minimax designs for 2k factorial experiments for generalized linear models

ABSTRACT

Formulas for A- and C-optimal allocations for binary factorial experiments in the context of generalized linear models are derived. Since the optimal allocations depend on GLM weights, which often are unknown, a minimax strategy is considered. This is shown to be simple to apply to factorial experiments. Efficiency is used to evaluate the resulting design. In some cases, the minimax design equals the optimal design. For other cases no general conclusion can be drawn. An example of a two-factor logit model suggests that the minimax design performs well, and often better than a uniform allocation.     

On the equivalence of definitions for regular fractions of mixed-level factorial designs

The notion of regularity for fractional factorial designs was originally defined only for two-level factorial designs. Recently, rather different definitions for regular fractions of mixed-level factorial designs have been proposed by Collombier [1996. Plans d’Expérience Factoriels. Springer, Berlin], Wu and Hamada [2000. Experiments. Wiley, New York] and Pistone and Rogantin [2008. Indicator function and complex coding for mixed fractional factorial designs. J. Statist. Plann. Inference 138, 787–802]. In this paper we prove that, surprisingly, these definitions are equivalent. The proof of equivalence relies heavily on the character theory of finite Abelian groups. The group-theoretic framework provides a unified approach to deal with mixed-level factorial designs and treat symmetric factorial designs as a special case. We show how within this framework each regular fraction is uniquely characterized by a defining relation as for two-level factorial designs. The framework also allows us to extend the result that every regular fraction is an orthogonal array of a strength that is related to its resolution, as stated in Dey and Mukerjee [1999. Fractional Factorial Plans. Wiley, New York] to mixed-level factorial designs.     

A bound connected with 26 factorial search designs of resolution 3.1

In this paper, we give a lower bound for the number of treatments required

for a plan to be a main effect plus one plan for 2^m (m = 6) factorial experiments, The lower bound problem is important in the event of generating new designs with similar properties or when one wants to study the criteria of optimality for such designs.     

A series of single array 2m factorial search designs for even m

By means of a search design one is able to search for and estimate a small set of non‐zero elements from the set of higher order factorial interactions in addition to estimating the lower order factorial effects. One may be interested in estimating the general mean and main effects, in addition to searching for and estimating a non‐negligible effect in the set of 2‐ and 3‐factor interactions, assuming 4‐ and higher‐order interactions are all zero. Such a search design is called a ‘main effect plus one plan’ and is denoted by MEP.1. Construction of such a plan, for 2^m factorial experiments, has been considered and developed by several authors and leads to MEP.1 plans for an odd number m of factors. These designs are generally determined by two arrays, one specifying a main effect plan and the other specifying a follow‐up. In this paper we develop the construction of search designs for an even number of factors m, m≠6. The new series of MEP.1 plans is a set of single array designs with a well structured form. Such a structure allows for flexibility in arriving at an appropriate design with optimum properties for search and estimation.     

Model‐free causal inference of binary experimental data

For binary experimental data, we discuss randomization‐based inferential procedures that do not need to invoke any modeling assumptions. In addition to the classical method of moments, we also introduce model‐free likelihood and Bayesian methods based solely on the physical randomization without any hypothetical super population assumptions about the potential outcomes. These estimators have some properties superior to moment‐based ones such as only giving estimates in regions of feasible support. Due to the lack of identification of the causal model, we also propose a sensitivity analysis approach that allows for the characterization of the impact of the association between the potential outcomes on statistical inference.     

Experimentation order with good properties for 2 k factorial designs

Randomizing the order of experimentation in a factorial design does not always achieve the desired effect of neutralizing the influence of unknown factors. In fact, with some very reasonable assumptions, an important proportion of random orders achieve the same degree of protection as that obtained by experimenting in the design matrix standard order. In addition, randomization can induce a large number of changes in factor levels and thus make experimentation expensive and difficult. De Leon et al. [Experimentation order in factorial designs with 8 or 16 runs, J. Appl. Stat. 32 (2005), pp. 297–313] proposed experimentation orders for designs with eight or 16 runs that combine an excellent level of protection against the influence of unknown factors, with the minimum number of changes in factor levels. This article presents a new methodology to obtain experimentation orders with the desired properties for designs with any number of runs.     

On the use of partial confounding for the construction of alternative regular two-level blocked fractional factorial designs

In this paper, we consider experimental situations in which a regular fractional factorial design is to be used to study the effects of m two-level factors using n=2^m−k experimental units arranged in 2^p blocks of size 2^m−k−p. In such situations, two-factor interactions are often confounded with blocks and complete information is lost on these two-factor interactions. Here we consider the use of the foldover technique in conjunction with combining designs having different blocking schemes to produce alternative partially confounded blocked fractional factorial designs that have more estimable two-factor interactions or a higher estimation capacity or both than their traditional counterparts.     

Bayesian design and analysis of composite endpoints in clinical trials with multiple dependent binary outcomes

The author considers studies with multiple dependent primary endpoints. Testing hypotheses with multiple primary endpoints may require unmanageably large populations. Composite endpoints consisting of several binary events may be used to reduce a trial to a manageable size. The primary difficulties with composite endpoints are that different endpoints may have different clinical importance and that higher‐frequency variables may overwhelm effects of smaller, but equally important, primary outcomes. To compensate for these inconsistencies, we weight each type of event, and the total number of weighted events is counted. To reflect the mutual dependency of primary endpoints and to make the weighting method effective in small clinical trials, we use the Bayesian approach. We assume a multinomial distribution of multiple endpoints with Dirichlet priors and apply the Bayesian test of noninferiority to the calculation of weighting parameters. We use composite endpoints to test hypotheses of superiority in single‐arm and two‐arm clinical trials. The composite endpoints have a beta distribution. We illustrate this technique with an example. The results provide a statistical procedure for creating composite endpoints. Published 2013. This article is a U.S. Government work and is in the public domain in the USA.     

Bayesian latent variable model for mixed continuous and ordinal responses with possibility of missing responses

A general framework is proposed for joint modelling of mixed correlated ordinal and continuous responses with missing values for responses, where the missing mechanism for both kinds of responses is also considered. Considering the posterior distribution of unknowns given all available information, a Markov Chain Monte Carlo sampling algorithm via winBUGS is used for estimating the posterior distribution of the parameters. For sensitivity analysis to investigate the perturbation from missing at random to not missing at random, it is shown how one can use some elements of covariance structure. These elements associate responses and their missing mechanisms. Influence of small perturbation of these elements on posterior displacement and posterior estimates is also studied. The model is illustrated using data from a foreign language achievement study.     

Bayesian sample‐size determination methods considering both worthwhileness and unpromisingness for exploratory two‐arm randomized clinical trials with binary endpoints

A randomized exploratory clinical trial comparing an experimental treatment with a control treatment on a binary endpoint is often conducted to make a go or no‐go decision. Such an exploratory trial needs to have an adequate sample size such that it will provide convincing evidence that the experimental treatment is either worthwhile or unpromising relative to the control treatment. In this paper, we propose three new sample‐size determination methods for an exploratory trial, which utilize the posterior probabilities calculated from predefined efficacy and inefficacy criteria leading to a declaration of the worthwhileness or unpromisingness of the experimental treatment. Simulation studies, including numerical investigation, showed that all three methods could declare the experimental treatment as worthwhile or unpromising with a high probability when the true response probability of the experimental treatment group is higher or lower, respectively, than that of the control treatment group.     

Bayesian D-optimal Accelerated Life Test plans for series systems with competing exponential causes of failure

This paper provides methods of obtaining Bayesian D-optimal Accelerated Life Test (ALT) plans for series systems with independent exponential component lives under the Type-I censoring scheme. Two different Bayesian D-optimality design criteria are considered. For both the criteria, first optimal designs for a given number of experimental points are found by solving a finite-dimensional constrained optimization problem. Next, the global optimality of such an ALT plan is ensured by applying the General Equivalence Theorem. A detailed sensitivity analysis is also carried out to investigate the effect of different planning inputs on the resulting optimal ALT plans. Furthermore, these Bayesian optimal plans are also compared with the corresponding (frequentist) locally D-optimal ALT plans.     

A Bayesian approach for analysing longitudinal nominal outcomes using random coefficients transitional generalized logit model: an application to the labour force survey data

A random-effects transition model is proposed to model the economic activity status of household members. This model is introduced to take into account two kinds of correlations; one due to the longitudinal nature of the study, which will be considered using a transition parameter, and the other due to the existing correlation between responses of members of the same household which is taken into account by introducing random coefficients into the model. The results are presented based on the homogeneous (all parameters are not changed by time) and non-homogeneous Markov models with random coefficients. A Bayesian approach via the Gibbs sampling is used to perform parameter estimation. Results of using random-effects transition model are compared, using deviance information criterion, with those of three other models which exclude random effects and/or transition effects. It is shown that the full model gains more precision due to the consideration of all aspects of the process which generated the data. To illustrate the utility of the proposed model, a longitudinal data set which is extracted from the Iranian Labour Force Survey is analysed to explore the simultaneous effect of some covariates on the current economic activity as a nominal response. Also, some sensitivity analyses are performed to assess the robustness of the posterior estimation of the transition parameters to the perturbations of the prior parameters.     

Sensitivity analysis for the identifiability with application to latent random effect model for the mixed data

In this paper, we study the indentifiability of a latent random effect model for the mixed correlated continuous and ordinal longitudinal responses. We derive conditions for the identifiability of the covariance parameters of the responses. Also, we proposed sensitivity analysis to investigate the perturbation from the non-identifiability of the covariance parameters, it is shown how one can use some elements of covariance structure. These elements associate conditions for identifiability of the covariance parameters of the responses. Influence of small perturbation of these elements on maximal normal curvature is also studied. The model is illustrated using medical data.     

On the use of extreme value tail modeling for generalized pairwise comparisons with censored outcomes

In randomized clinical trials, methods of pairwise comparisons such as the ‘Net Benefit’ or the ‘win ratio’ have recently gained much attention when interests lies in assessing the effect of a treatment as compared to a standard of care. Among other advantages, these methods are usually praised for delivering a treatment measure that can easily handle multiple outcomes of different nature, while keeping a meaningful interpretation for patients and clinicians. For time-to-event outcomes, a recent suggestion emerged in the literature for estimating these treatment measures by providing a natural handling of censored outcomes. However, this estimation procedure may lead to biased estimates when tails of survival functions cannot be reliably estimated using Kaplan–Meier estimators. The problem then extrapolates to the other outcomes incorporated in the pairwise comparison construction. In this work, we suggest to extend the procedure by the consideration of a hybrid survival function estimator that relies on an extreme value tail model through the Generalized Pareto distribution. We provide an estimator of treatment effect measures that notably improves on bias and remains easily apprehended for practical implementation. This is illustrated in an extensive simulation study as well as in an actual trial of a new cancer immunotherapy.     

On the Bayesian estimation and influence diagnostics for the Weibull-Negative-Binomial regression model with cure rate under latent failure causes

The purpose of this paper is to develop a Bayesian approach for the Weibull-Negative-Binomial regression model with cure rate under latent failure causes and presence of randomized activation mechanisms. We assume the number of competing causes of the event of interest follows a Negative Binomial (NB) distribution while the latent lifetimes are assumed to follow a Weibull distribution. Markov chain Monte Carlos (MCMC) methods are used to develop the Bayesian procedure. Model selection to compare the fitted models is discussed. Moreover, we develop case deletion influence diagnostics for the joint posterior distribution based on the ψ-divergence, which has several divergence measures as particular cases. The developed procedures are illustrated with a real data set.     

Latent variable model for mixed correlated power series and ordinal longitudinal responses with non ignorable missing values

We propose a joint model based on a latent variable for analyzing mixed power series and ordinal longitudinal data with and without missing values. A bivariate probit regression model is used for the missing mechanisms. Random effects are used to take into account the correlation between longitudinal responses. A full likelihood-based approach is used to yield maximum-likelihood estimates of the model parameters. Our model is applied to a medical data set, obtained from an observational study on women where the correlated responses are the ordinal response of osteoporosis of the spine and the power series response of the number of joint damages. Sensitivity analysis is also performed to study the influence of small perturbations of the parameters of the missing mechanisms and overdispersion of the model on likelihood displacement.