Using the counterfactual framework to estimate non-intention-to-treat estimands in randomised controlled trials: A methodological scoping review.

Journal: Contemporary clinical trials
Published Date: Apr 11, 2025

Abstract

BACKGROUND: Randomised controlled trials (RCTs) commonly estimate intention-to-treat (ITT) estimands. However, when nonadherence to assigned treatment occurs, ITT estimands reflect the effect of being offered treatment, rather than adhering to it and thus are less useful for clinical decision-making.

Authors

  • Ellie Medcalf
    Sydney School of Public Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia. Electronic address: ellie.medcalf@sydney.edu.au.
  • Fiona Stanaway
    Sydney School of Public Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.
  • Robin M Turner
    Biostatistics Centre, University of Otago, Dunedin, New Zealand.
  • David Espinoza
    National Health and Medical Research Council Clinical Trials Centre, The University of Sydney, Sydney, New South Wales, Australia.
  • Katy J L Bell
    Sydney School of Public Health, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.

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