Health-related quality of life has become an increasingly important indicator of health status in clinical trials and epidemiological research. Moreover, the study of the relationship of health-related quality of life with patients and disease characteristics has become one of the primary aims of many health-related quality of life studies. Health-related quality of life scores are usually assumed to be distributed as binomial random variables and often highly skewed. The use of the beta-binomial distribution in the regression context has been proposed to model such data; however, the beta-binomial regression has been performed by means of two different approaches in the literature: (i) beta-binomial distribution with a logistic link; and (ii) hierarchical generalized linear models. None of the existing literature in the analysis of health-related quality of life survey data has performed a comparison of both approaches in terms of adequacy and regression parameter interpretation context. This paper is motivated by the analysis of a real data application of health-related quality of life outcomes in patients with Chronic Obstructive Pulmonary Disease, where the use of both approaches yields to contradictory results in terms of covariate effects significance and consequently the interpretation of the most relevant factors in health-related quality of life. We present an explanation of the results in both methodologies through a simulation study and address the need to apply the proper approach in the analysis of health-related quality of life survey data for practitioners, providing an R package.

Patient‐reported outcomes (PROs) are currently being increasingly used as primary outcome measures in observational and experimental studies since they inform clinicians and researchers about the health‐status of patients and generate data to facilitate improved care. PROs usually appear as discrete and bounded with U, J, or inverse J shapes, and hence, exponential family members offer inadequate distributional fits. The beta‐binomial distribution has been proposed in the literature to fit PROs. However, the fact that the beta‐binomial distribution does not belong to the exponential family limits its applicability in the regression model context, and classical estimation approaches are not straightforward. Moreover, PROs are usually measured in a longitudinal framework in which individuals are followed up for a certain period. Hence, each individual obtains several scores of the PRO over time, which leads to the repeated measures and defines the correlation structure in the data. In this work, we have developed and proposed an estimation procedure for the analysis of correlated discrete and bounded outcomes, particularly PROs, by a beta‐binomial mixed‐effects model. Additionally, we have implemented the methodology in thePROregpackage in R. Because there are similar approaches in the literature to address the same issue, this work also incorporates a comparison study between our proposal and alternative methodologies commonly implemented in R and shows the superior performance of our estimation procedure. This paper was motivated by the analysis of the health‐status of patients with chronic obstructive pulmonary disease, where the main objective is the assessment of risk factors that may affect the evolution of the disease. The application of the proposed approach in the study leads to clinically relevant results.

In this paper, we propose an alternative distribution to model count data exhibiting uni/bimodality. It arises as a weighted version of the beta-binomial distribution, which is defined by a parametric weight function that admits up to two modes for the resulting probability mass function. Like the baseline beta-binomial distribution, the proposed distribution performs well in modeling overdispersed binomial data. Structural properties of the new distribution are studied. Raw moments are derived, which are used to describe the dispersion behavior relative to the mean and the skewness behavior. Parameter estimation is carried out using the maximum likelihood method. A simulation study is conducted in order to illustrate the behavior of the estimators. Finally, two applications illustrating the usefulness of the proposal are presented.