Co-constructing a Conceptual Understanding of System Enactment

Clinical Social Work Journal - System enactments are co-created phenomena characterized by confounding and emotionally charged multi-person interactions that emerge through the convergence of...     

Multiple event times in the presence of informative censoring: modeling and analysis by copulas

Motivated by a breast cancer research program, this paper is concerned with the joint survivor function of multiple event times when their observations are subject to informative censoring caused by a terminating event. We formulate the correlation of the multiple event times together with the time to the terminating event by an Archimedean copula to account for the informative censoring. Adapting the widely used two-stage procedure under a copula model, we propose an easy-to-implement pseudo-likelihood based procedure for estimating the model parameters. The approach yields a new estimator for the marginal distribution of a single event time with semicompeting-risks data. We conduct both asymptotics and simulation studies to examine the proposed approach in consistency, efficiency, and robustness. Data from the breast cancer program are employed to illustrate this research.

     

Inferences on the Means of Two Log-Normal Distributions: A Computational Approach Test

In this article, we propose a novel approach for testing the equality of two log-normal populations using a computational approach test (CAT) that does not require explicit knowledge of the sampling distribution of the test statistic. Simulation studies demonstrate that the proposed approach can perform hypothesis testing with satisfying actual size even at small sample sizes. Overall, it is superior to other existing methods. Also, a CAT is proposed for testing about reliability of two log-normal populations when the means are the same. Simulations show that the actual size of this new approach is close to nominal level and better than the score test. At the end, the proposed methods are illustrated using two examples.     

A new long-term survival model with dispersion induced by discrete frailty

Lifetime Data Analysis - Frailty models are generally used to model heterogeneity between the individuals. The distribution of the frailty variable is often assumed to be continuous. However, there...     

Partial Identification of Economic Mobility: With an Application to the United States

Abstract

The economic mobility of individuals and households is of fundamental interest. While many measures of economic mobility exist, reliance on transition matrices remains pervasive due to simplicity and ease of interpretation. However, estimation of transition matrices is complicated by the well-acknowledged problem of measurement error in self-reported and even administrative data. Existing methods of addressing measurement error are complex, rely on numerous strong assumptions, and often require data from more than two periods. In this article, we investigate what can be learned about economic mobility as measured via transition matrices while formally accounting for measurement error in a reasonably transparent manner. To do so, we develop a nonparametric partial identification approach to bound transition probabilities under various assumptions on the measurement error and mobility processes. This approach is applied to panel data from the United States to explore short-run mobility before and after the Great Recession.     

Blinded continuous monitoring in clinical trials with recurrent event endpoints

In studies with recurrent event endpoints, misspecified assumptions of event rates or dispersion can lead to underpowered trials or overexposure of patients. Specification of overdispersion is often a particular problem as it is usually not reported in clinical trial publications. Changing event rates over the years have been described for some diseases, adding to the uncertainty in planning. To mitigate the risks of inadequate sample sizes, internal pilot study designs have been proposed with a preference for blinded sample size reestimation procedures, as they generally do not affect the type I error rate and maintain trial integrity. Blinded sample size reestimation procedures are available for trials with recurrent events as endpoints. However, the variance in the reestimated sample size can be considerable in particular with early sample size reviews. Motivated by a randomized controlled trial in paediatric multiple sclerosis, a rare neurological condition in children, we apply the concept of blinded continuous monitoring of information, which is known to reduce the variance in the resulting sample size. Assuming negative binomial distributions for the counts of recurrent relapses, we derive information criteria and propose blinded continuous monitoring procedures. The operating characteristics of these are assessed in Monte Carlo trial simulations demonstrating favourable properties with regard to type I error rate, power, and stopping time, ie, sample size.     

A new generalized Weibull distribution in income economic inequality curves

Researchers have been developing various extensions and modified forms of the Weibull distribution to enhance its capability for modeling and fitting different data sets. In this note, we investigate the potential usefulness of the new modification to the standard Weibull distribution called odd Weibull distribution in income economic inequality studies. Some mathematical and statistical properties of this model are proposed. We obtain explicit expressions for the first incomplete moment, quantile function, Lorenz and Zenga curves and related inequality indices. In addition to the well-known stochastic order based on Lorenz curve, the stochastic order based on Zenga curve is considered. Since the new generalized Weibull distribution seems to be suitable to model wealth, financial, actuarial and especially income distributions, these findings are fundamental in the understanding of how parameter values are related to inequality. Also, the estimation of parameters by maximum likelihood and moment methods is discussed. Finally, this distribution has been fitted to United States and Austrian income data sets and has been found to fit remarkably well in compare with the other widely used income models.     

Inference and Decision Making for 21st-Century Drug Development and Approval

ABSTRACT

The cost and time of pharmaceutical drug development continue to grow at rates that many say are unsustainable. These trends have enormous impact on what treatments get to patients, when they get them and how they are used. The statistical framework for supporting decisions in regulated clinical development of new medicines has followed a traditional path of frequentist methodology. Trials using hypothesis tests of “no treatment effect” are done routinely, and the p-value < 0.05 is often the determinant of what constitutes a “successful” trial. Many drugs fail in clinical development, adding to the cost of new medicines, and some evidence points blame at the deficiencies of the frequentist paradigm. An unknown number effective medicines may have been abandoned because trials were declared “unsuccessful” due to a p-value exceeding 0.05. Recently, the Bayesian paradigm has shown utility in the clinical drug development process for its probability-based inference. We argue for a Bayesian approach that employs data from other trials as a “prior” for Phase 3 trials so that synthesized evidence across trials can be utilized to compute probability statements that are valuable for understanding the magnitude of treatment effect. Such a Bayesian paradigm provides a promising framework for improving statistical inference and regulatory decision making.