Search for: All records

Background: Outcome measures that are count variables with excessive zeros are common in health behaviors research. Examples include the number of standard drinks consumed or alcohol‐related problems experienced over time. There is a lack of empirical data about the relative performance of prevailing statistical models for assessing the efficacy of interventions when outcomes are zero‐inflated, particularly compared with recently developed marginalized count regression approaches for such data.Methods: The current simulation study examined five commonly used approaches for analyzing count outcomes, including two linear models (with outcomes on raw and log‐transformed scales, respectively) and three prevailing count distribution‐based models (ie, Poisson, negative binomial, and zero‐inflated Poisson (ZIP) models). We also considered the marginalized zero‐inflated Poisson (MZIP) model, a novel alternative that estimates the overall effects on the population mean while adjusting for zero‐inflation. Motivated by alcohol misuse prevention trials, extensive simulations were conducted to evaluate and compare the statistical power and Type I error rate of the statistical models and approaches across data conditions that varied in sample size ( to 500), zero rate (0.2 to 0.8), and intervention effect sizes.Results: Under zero‐inflation, the Poisson model failed to control the Type I error rate, resulting in higher than expected false positive results. When the intervention effects on the zero (vs. non‐zero) and count parts were in the same direction, the MZIP model had the highest statistical power, followed by the linear model with outcomes on the raw scale, negative binomial model, and ZIP model. The performance of the linear model with a log‐transformed outcome variable was unsatisfactory.Conclusions: The MZIP model demonstrated better statistical properties in detecting true intervention effects and controlling false positive results for zero‐inflated count outcomes. This MZIP model may serve as an appealing analytical approach to evaluating overall intervention effects in studies with count outcomes marked by excessive zeros. 

Abstract Stemming from the high-profile publication of Nissen and Wolski (N Engl J Med 356:2457–2471, 2007) and subsequent discussions with divergent views on how to handle observed zero-total-event studies, defined to be studies that observe zero number of event in both treatment and control arms, the research topic concerning the common odds ratio model with zero-total-event studies remains to be an unresolved problem in meta-analysis. In this article, we address this problem by proposing a novel repro samples method to handle zero-total-event studies and make inference for the common odds ratio. The development explicitly accounts for the sampling scheme that generates the observed data and does not rely on any large sample approximations. It is theoretically justified with a guaranteed finite-sample performance. Simulation studies are designed to demonstrate the empirical performance of the proposed method. It shows that the proposed confidence set, although a little conservative, achieves the desired empirical coverage rate in all situations. The development also shows that the zero-total-event studies contain meaningful information and impact the inference for the common odds ratio. The proposed method is used to perform a meta-analysis of the 48 trials reported in Nissen and Wolski (N Engl J Med 356:2457–2471, 2007) as well 

Abstract Well-known debates among statistical inferential paradigms emerge from conflicting views on the notion of probability. One dominant view understands probability as a representation of sampling variability; another prominent view understands probability as a measure of belief. The former generally describes model parameters as fixed values, in contrast to the latter. We propose that there are actually two versions of a parameter within both paradigms: a fixed unknown value that generated the data and a random version to describe the uncertainty in estimating the unknown value. An inferential approach based on CDs deciphers seemingly conflicting perspectives on parameters and probabilities. 

Approximate confidence distribution computing (ACDC) offers a new take on the rapidly developing field of likelihood-free inference from within a frequentist framework. The appeal of this computational method for statistical inference hinges upon the concept of a confidence distribution, a special type of estimator which is defined with respect to the repeated sampling principle. An ACDC method provides frequentist validation for computational inference in problems with unknown or intractable likelihoods. The main theoretical contribution of this work is the identification of a matching condition necessary for frequentist validity of inference from this method. In addition to providing an example of how a modern understanding of confidence distribution theory can be used to connect Bayesian and frequentist inferential paradigms, we present a case to expand the current scope of so-called approximate Bayesian inference to include non-Bayesian inference by targeting a confidence distribution rather than a posterior. The main practical contribution of this work is the development of a data-driven approach to drive ACDC in both Bayesian or frequentist contexts. The ACDC algorithm is data-driven by the selection of a data-dependent proposal function, the structure of which is quite general and adaptable to many settings. We explore three numerical examples that both verify the theoretical arguments in the development of ACDC and suggest instances in which ACDC outperform approximate Bayesian computing methods computationally. 

Abstract The flexibility and wide applicability of the Fisher randomization test (FRT) make it an attractive tool for assessment of causal effects of interventions from modern-day randomized experiments that are increasing in size and complexity. This paper provides a theoretical inferential framework for FRT by establishing its connection with confidence distributions. Such a connection leads to development’s of (i) an unambiguous procedure for inversion of FRTs to generate confidence intervals with guaranteed coverage, (ii) new insights on the effect of size of the Monte Carlo sample on the estimation of a p-value curve and (iii) generic and specific methods to combine FRTs from multiple independent experiments with theoretical guarantees. Our developments pertain to finite sample settings but have direct extensions to large samples. Simulations and a case example demonstrate the benefit of these new developments.