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A scoping review of statistical methods for trial-based economic evaluations: The current state of play.

Mohamed El AliliJohanna M van DongenJonas L EsserMartijn W HeymansMaurits W van TulderJudith E Bosmans
Published in: Health economics (2022)
The statistical quality of trial-based economic evaluations is often suboptimal, while a comprehensive overview of available statistical methods is lacking. Therefore, this review summarized and critically appraised available statistical methods for trial-based economic evaluations. A literature search was performed to identify studies on statistical methods for dealing with baseline imbalances, skewed costs and/or effects, correlated costs and effects, clustered data, longitudinal data, missing data and censoring in trial-based economic evaluations. Data was extracted on the statistical methods described, their advantages, disadvantages, relative performance and recommendations of the study. Sixty-eight studies were included. Of them, 27 (40%) assessed methods for baseline imbalances, 39 (57%) assessed methods for skewed costs and/or effects, 27 (40%) assessed methods for correlated costs and effects, 18 (26%) assessed methods for clustered data, 7 (10%) assessed methods for longitudinal data, 26 (38%) assessed methods for missing data and 10 (15%) assessed methods for censoring. All identified methods were narratively described. This review provides a comprehensive overview of available statistical methods for dealing with the most common statistical complexities in trial-based economic evaluations. Herewith, it can provide valuable input for researchers when deciding which statistical methods to use in a trial-based economic evaluation.
Keyphrases
  • clinical trial
  • electronic health record
  • study protocol
  • big data
  • phase iii
  • systematic review
  • randomized controlled trial
  • cross sectional
  • quality improvement
  • artificial intelligence
  • double blind