Advances in Addressing Patient Heterogeneity in Economic Evaluation: A Review of the Methods Literature

Gemma E. Shields (), Paul Clarkson, Ash Bullement, Warren Stevens, Mark Wilberforce, Tracey Farragher, Arpana Verma and Linda M. Davies
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Gemma E. Shields: Manchester Centre for Health Economics, University of Manchester
Paul Clarkson: University of Manchester
Ash Bullement: Delta Hat Ltd
Warren Stevens: Medicus Economics
Mark Wilberforce: University of York
Tracey Farragher: University of Manchester
Arpana Verma: University of Manchester
Linda M. Davies: Manchester Centre for Health Economics, University of Manchester

PharmacoEconomics, 2024, vol. 42, issue 7, No 3, 737-749

Abstract: Abstract Cost-effectiveness analyses commonly use population or sample averages, which can mask key differences across subgroups and may lead to suboptimal resource allocation. Despite there being several new methods developed over the last decade, there is no recent summary of what methods are available to researchers. This review sought to identify advances in methods for addressing patient heterogeneity in economic evaluations and to provide an overview of these methods. A literature search was conducted using the Econlit, Embase and MEDLINE databases to identify studies published after 2011 (date of a previous review on this topic). Eligible studies needed to have an explicit methodological focus, related to how patient heterogeneity can be accounted for within a full economic evaluation. Sixteen studies were included in the review. Methodologies were varied and included regression techniques, model design and value of information analysis. Recent publications have applied methodologies more commonly used in other fields, such as machine learning and causal forests. Commonly noted challenges associated with considering patient heterogeneity included data availability (e.g., sample size), statistical issues (e.g., risk of false positives) and practical factors (e.g., computation time). A range of methods are available to address patient heterogeneity in economic evaluation, with relevant methods differing according to research question, scope of the economic evaluation and data availability. Researchers need to be aware of the challenges associated with addressing patient heterogeneity (e.g., data availability) to ensure findings are meaningful and robust. Future research is needed to assess whether and how methods are being applied in practice.

Date: 2024
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DOI: 10.1007/s40273-024-01377-9

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