Methodological developments in randomized controlled trial-based economic evaluations

Abstract

Economic evaluation is a key contributor to decision making in health care, and it is important that it is carried out as effectively and reliably as possible. Studies carried out alongside randomised controlled trials are required to contribute real-world evidence to the decision-making process. However, the requirement that resource use be measured as well as effectiveness data within a trial results in additional complexity for trialists, and there are a number of methodological areas in which improvement is needed. This article reviews the literature in methodological work carried out to inform economic evaluation studies conducted alongside randomised controlled trials. Recent advances in areas including overall trial design, measuring resource use, measuring outcomes and reporting economic evaluations are discussed.

Keywords::

• clinical trial
• cost
• economic evaluation
• EQ-5D
• health-related quality of life
• randomised controlled trial
• resource-use measurement
• utility measurement

Acknowledgements

The authors would like to thank Joanna Coast and Dyfrig Hughes for their helpful comments.

Financial & competing interests disclosure

This work was funded the MRC Network of Hubs for Trials Methodology Research (ConDuCT Hub, grant numbers G0800800 and MR/K025643/1). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Key issues

• Methodological research to underpin the use of cost-effectiveness studies in randomized controlled trials represents an area of considerable research interest.

• Improvements in the methods used to conduct economic evaluations will lead to greater accuracy and confidence in cost-effectiveness outcomes for decision makers.

• Outcome measurement has received sustained methodological attention in recent years. In particular, the use of the capability approach has opened up many avenues of research.

• Although there are now many instruments available for measuring health-related quality of life outcomes, the EQ-5D remains dominant, with improvements and new versions being developed.

• Advances have been made in methods for measuring resource use and applying unit costs, but less attention has been paid to this area than to the study of outcomes.

• Despite recent advances in providing guidance on reporting many aspects of economic evaluations, reporting is still weaker than it should be.

• Key challenges for further work include establishing the comparability of disease-specific and generic outcome measures for decision making, developing standardized instruments for resource-use measurement and incorporating productivity changes routinely into economic evaluations conducted from a societal perspective.