Health Economics in Pragmatic Clinical Trials
(Week of November 24, 2025)
(Week of November 24, 2025)
Module 6-1: Health Economics in Pragmatic Clinical Trials (20-Minute Video)
This module introduces the foundations of health economic evaluation within pragmatic clinical trials, explaining how economic analyses help decision-makers allocate limited healthcare resources by comparing costs, outcomes, and real-world value. It walks through key concepts such as cost-effectiveness, cost-utility, QALYs, costing methods, resource use, and the importance of integrating economic evaluation early in trial design.
** The video's content and narration were generated with the assistance of artificial intelligence, with human guidance and oversight throughout the process. **
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Summary: This article outlines the major approaches used to measure health outcomes in economic evaluations, emphasizing how these methods help determine whether healthcare interventions provide good value for the resources invested. Traditional clinical endpoints, such as reductions in disease events or changes in physiological measures, are straightforward but limit comparability across different types of treatments. Broader measures of health-related quality of life, including condition-specific scales, generic instruments, and preference-based utility measures such as the EuroQol, allow assessments that capture both morbidity and overall well-being, though they may lack sensitivity to subtle but meaningful changes. The authors also note that some benefits extend beyond health itself, including emotional, social, and autonomy-related gains. Techniques such as willingness to pay and conjoint analysis enable quantification of these wider benefits by incorporating patient or societal preferences. Together, these outcome measurement strategies provide a more complete and rigorous foundation for evaluating the efficiency and value of healthcare interventions.
Summary: This study examined how well missing data are reported and handled in economic evaluations conducted alongside pragmatic clinical trials and found substantial shortcomings that could undermine the reliability of results used for healthcare decision making. Among the one hundred fifty two included studies, most had missing cost or effect data, yet fewer than two thirds reported how much information was missing, and only a small proportion explained the reasons or assumptions behind the patterns of missingness. The median level of missing data was about thirty percent and was even higher in studies with longer follow up periods. Although multiple imputation was the most commonly used method in primary analyses, sensitivity analyses were performed in fewer than forty percent of studies, despite the need to test whether conclusions hold under different assumptions about missing information. The authors conclude that poor reporting quality and the high proportion of missing data in economic evaluations embedded in pragmatic trials threaten the credibility of these analyses and call for more rigorous reporting standards, better methodological practices, and greater attention to missing data in future research.
Abstract: Health economic evaluations are comparative analyses of alternative courses of action in terms of their costs and consequences. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement, published in 2013, was created to ensure health economic evaluations are identifiable, interpretable, and useful for decision making. It was intended as guidance to help authors report accurately which health interventions were being compared and in what context, how the evaluation was undertaken, what the findings were, and other details that may aid readers and reviewers in interpretation and use of the study. The new CHEERS 2022 statement replaces previous CHEERS reporting guidance. It reflects the need for guidance that can be more easily applied to all types of health economic evaluation, new methods and developments in the field, as well as the increased role of stakeholder involvement including patients and the public. It is also broadly applicable to any form of intervention intended to improve the health of individuals or the population, whether simple or complex, and without regard to context (such as health care, public health, education, social care, etc). This summary article presents the new CHEERS 2022 28-item checklist and recommendations for each item. The CHEERS 2022 statement is primarily intended for researchers reporting economic evaluations for peer reviewed journals as well as the peer reviewers and editors assessing them for publication. However, we anticipate familiarity with reporting requirements will be useful for analysts when planning studies. It may also be useful for health technology assessment bodies seeking guidance on reporting, as there is an increasing emphasis on transparency in decision making.
Summary: This document presents the CHEERS 2022 Checklist, an updated international reporting standard for health economic evaluations. Developed by an ISPOR task force, it outlines twenty eight required reporting items that help ensure economic studies are transparent, complete, and useful for decision makers. The checklist covers all elements of an evaluation, including how study populations, interventions, comparators, costs, outcomes, time horizons, uncertainty, and stakeholder engagement should be described. It also specifies expectations for presenting results, discussing limitations, and declaring funding sources and conflicts of interest. By consolidating best practices into a single structured tool, the CHEERS 2022 Checklist supports higher quality reporting and improves the interpretability and comparability of economic evaluations used in health policy and clinical decision making.