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A Scoping Review of Item-Level Missing Data in Within-Trial Cost-Effectiveness Analysis
Institution:1. Department of Statistical Science, University College London, London, England, UK;2. Department of Methodology and Statistics, Faculty of Health Medicine and Life Science, Maastricht University, Maastricht, Limburg, The Netherlands;3. London School of Hygiene and Tropical Medicine, London, England, UK;1. Health Economics Unit, Centre for Health Policy, Melbourne School of Global and Population Health, University of Melbourne, Melbourne, Victoria, Australia;2. Murdoch Children’s Research Institute, Melbourne, Victoria, Australia;3. Centre for Health Economics, Monash Business School, Monash University, Melbourne, Victoria, Australia;4. Department of Paediatrics, Faculty of Medicine, Dentistry and Health Sciences, University of Melbourne, Melbourne, Victoria, Australia;5. Department of Allergy and Immunology, The Royal Children’s Hospital, Melbourne, Victoria, Australia;1. Hamburg Center for Health Economics, Universität Hamburg, Hamburg, Germany;2. Faculty of Business Administration and Economics/CINCH Health Economics Research Center, Universität Duisburg-Essen, Duisburg, Germany;1. Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy;2. Multiple Sclerosis Centre, Spedali Civili di Brescia, Montichiari, Italy;3. IRCCS Multimedica, Sesto San Giovanni, Italy;4. Department of Medical, Surgical Science and Advanced Technology “GF Ingrassia,” University of Catania, Catania, Italy;1. Erasmus School of Health Policy and Management, Department of Health Care Governance, Rotterdam, The Netherlands;2. Dutch Health Care Institute (Zorginstituut Nederland), Diemen, The Netherlands;1. School of Health and Related Research, University of Sheffield, Sheffield, England, UK;2. Centre for Regional Economic and Social Research, Sheffield Hallam University, Sheffield, England, UK;3. Institute of Health Research, University of Exeter, St Luke’s Campus, Exeter, England, UK
Abstract:ObjectivesCost-effectiveness analysis (CEA) alongside randomized controlled trials often relies on self-reported multi-item questionnaires that are invariably prone to missing item-level data. The purpose of this study is to review how missing multi-item questionnaire data are handled in trial-based CEAs.MethodsWe searched the National Institute for Health Research journals to identify within-trial CEAs published between January 2016 and April 2021 using multi-item instruments to collect costs and quality of life (QOL) data. Information on missing data handling and methods, with a focus on the level and type of imputation, was extracted.ResultsA total of 87 trial-based CEAs were included in the review. Complete case analysis or available case analysis and multiple imputation (MI) were the most popular methods, selected by similar numbers of studies, to handle missing costs and QOL in base-case analysis. Nevertheless, complete case analysis or available case analysis dominated sensitivity analysis. Once imputation was chosen, missing costs were widely imputed at item-level via MI, whereas missing QOL was usually imputed at the more aggregated time point level during the follow-up via MI.ConclusionsMissing costs and QOL tend to be imputed at different levels of missingness in current CEAs alongside randomized controlled trials. Given the limited information provided by included studies, the impact of applying different imputation methods at different levels of aggregation on CEA decision making remains unclear.
Keywords:cost-effectiveness  economic evaluation  missing data  multi-item questionnaire  review  trial
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