made substantial contributions to the design and drafting of the work, approved the version to be published, and agree to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved. Dyfrig Hughes will act as the guarantor of the work presented in this paper.
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Abstract:Trial-based economic evaluations are an important aspect of health technology assessment. The availability of patient-level data coupled with unbiased estimates of clinical outcomes means that randomised controlled trials are effective vehicles for the generation of economic data. However there are methodological challenges to trial-based evaluations, which include the collection of reliable data on resource use and cost, choice of health outcome measure, calculating minimally important differences, dealing with missing data, extrapolating outcomes and costs over time and the analysis of multinational trials. This review focuses on the state of the art of selective elements concerning the design, conduct, analysis and reporting of trial-based economic evaluations. The limitations of existing approaches are detailed and novel methods introduced. The review is internationally relevant but with a focus towards practice in the UK.
Key Messages: Trial-based economic evaluations require careful consideration of design, conduct, analysis and reporting. Areas for improvement include the need for more robust methods to estimate resource utilisation, use of multiple imputation for missing data, and application of appropriate modelling for the assessment of the impact of non-adherence. This review identifies economic methods that may also enhance clinical trials such as for calculating minimally important differences using discrete choice experiments, and undertaking data extrapolation based on modelling.