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Rationale and methodology for trial-based economic evaluation

Stavros Petrou

Economic evaluation provides a means of allocating finite healthcare resources in an efficient manner. It can inform decision-making processes at many levels, from national decision-making bodies, such as the NICE in England and Wales, to decisions by local healthcare providers. A common vehicle for the conduct of economic evaluation is the randomized controlled trial. This paper provides an overview of the methodologies underpinning economic evaluations based on randomized controlled trials. It covers broad design issues, approaches to measuring resource inputs, approaches to valuing resource inputs, approaches to the measurement and valuation of outcomes, the analysis of data inputs, the comparative analysis of costs and consequences, and methods for handling uncertainty and extrapolating cost–effectiveness. The strengths and limitations of trial-based economic evaluations in comparison with other designs are outlined.