A Bayesian Modeling Framework for Health Care Resource Use and Costs in Trial-Based Economic Evaluations.

Individual-level data are routinely used in trial-based economic evaluations to assess the effectiveness and costs of a given intervention. While effectiveness measures are often expressed via utility scores derived from health-related quality-of-life instruments (e.g., EQ-5D questionnaires), information on different types of health care resource use (HRU) measures (e.g., number and types of services) are collected to compute the costs. Partially complete HRU data, particularly for self-reported questionnaires, are handled via ad hoc methods that rely on some assumptions (fill in a zero) that are typically hard to justify. Although methods have been proposed to account for the uncertainty surrounding missing data, particularly in the form of multiple imputation or Bayesian methods, these have mostly been implemented at the level of costs at different times or over the entire study period, while little attention has been given to how missing values at the level of HRUs should be addressed and their implications on the final analysis. We present a general Bayesian framework for the analysis of partially observed HRUs in trial-based economic evaluations, which can accommodate the typical complexities of the data (e.g., excess zeros, skewness, missingness) and quantify the impact of missingness uncertainty on the results. We show the benefits of our approach with a motivating example and compare the results to those from more standard analyses fitted at the level of cost variables after adopting some ad hoc imputation. This article highlights the importance of adopting a comprehensive modeling approach to handle partially observed HRU data in economic evaluations and the strategic advantages of building these models within a Bayesian framework.HighlightsMissing health care service data in trial-based economic evaluations are often removed or imputed using quite restrictive assumptions (e.g., no use of service).We propose a flexible Bayesian approach to account for missing health care service uncertainty and compare the results with models fitted at more aggregated levels (e.g., total costs) using a real case study.Our results show that, depending on the (assumed) missingness assumptions and the level of data aggregation at which analyses are performed, results may be considerably changed.When feasible, analyses should be conducted at the most disaggregated level to ensure that all available information collected in the trial is used in the analysis without relying on (often) restrictive ad hoc imputation approaches.