Conducting Economic Evaluations Alongside Randomised Trials: Current Methodological Issues and Novel Approaches

Abstract

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. 2 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.

Cost-effectiveness of surgical interventions for the management of osteoarthritis: a systematic review of the literature

“…In addition to this, the quality assessment was carried out by one reviewer. Second, studies that were included in this review were conducted across nine countries which may lead to problems such as generalisability and transferability of the study findings to other settings due to differences in factors such as clinical practice, prices and epidemiology of disease [47, 48]. In addition to this, guidance in many countries does not consider economic data to be transferable to their settings in most cases [49, 50].…”

A Bayesian framework for health economic evaluation in studies with missing data

“…A general concern is missing data, for example, because patients are lost to follow‐up or fail to provide complete responses to questions about their health status or resource use. Multiple Imputation has been widely recommended for handling missing data (Briggs, Clark, Wolstenholme, & Clarke, ; Faria, Gomes, Epstein, & White, ; Gomes, Diaz‐Ordaz, Grieve, & Kenward, ; Hunter et al, ; Hughes et al, ), but typically assumes that data are “missing at random” (MAR).…”

Measurement tools of resource use and quality of life in clinical trials for dementia or cognitive impairment interventions: A systematically conducted narrative review

“…Given the finite health care budget, economic evaluations aiming to support decision making about these new treatments in dementia are essential. Ideally, these evaluations should be based on long‐term clinical trial results that capture the benefits and costs of the intervention …”

“…Ideally, these evaluations should be based on long-term clinical trial results that capture the benefits and costs of the intervention. 4 Cost-utility analysis is the most widely used form of economic evaluation. In such analysis, quality-adjusted life year (QALY) is routinely used as the summary measure of health outcomes, which takes both the quantity and quality of life into account.…”