Comprehensive Review of Methods to Assess Uncertainty in Health Economic Evaluations

Thomas Michael Otten (), Sabine E. Grimm, Bram Ramaekers and Manuela A. Joore
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Thomas Michael Otten: Department of Clinical Epidemiology and Medical Technology Assessment (KEMTA)
Sabine E. Grimm: Department of Clinical Epidemiology and Medical Technology Assessment (KEMTA)
Bram Ramaekers: Department of Clinical Epidemiology and Medical Technology Assessment (KEMTA)
Manuela A. Joore: Department of Clinical Epidemiology and Medical Technology Assessment (KEMTA)

PharmacoEconomics, 2023, vol. 41, issue 6, No 2, 619-632

Abstract: Abstract Uncertainty assessment is a cornerstone in model-based health economic evaluations (HEEs) that inform reimbursement decisions. No comprehensive overview of available uncertainty assessment methods currently exists. We aimed to review methods for uncertainty assessment for use in model-based HEEs, by conducting a snowballing review. We categorised all methods according to their stage of use relating to uncertainty assessment (identification, analysis, communication). Additionally, we classified identification methods according to sources of uncertainty, and subdivided analysis and communication methods according to their purpose. The review identified a total of 80 uncertainty methods: 30 identification, 28 analysis, and 22 communication methods. Uncertainty identification methods exist to address uncertainty from different sources. Most identification methods were developed with the objective to assess related concepts such as validity, model quality, and relevance. Almost all uncertainty analysis and communication methods required uncertainty to be quantified and inclusion of uncertainties in probabilistic analysis. Our review can help analysts and decision makers in selecting uncertainty assessment methods according to their aim and purpose of the assessment. We noted a need for further clarification of terminology and guidance on the use of (combinations of) methods to identify uncertainty and related concepts such as validity and quality. A key finding is that uncertainty assessment relies heavily on quantification, which may necessitate increased use of expert elicitation and/or the development of methods to assess unquantified uncertainty.

Date: 2023
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DOI: 10.1007/s40273-023-01242-1

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