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Review of Designs for Accommodating Patients’ or Physicians’ Preferences in Randomized Controlled Trials

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Developments in Statistical Evaluation of Clinical Trials

Abstract

The randomized controlled trial (RCT) is regarded as the principal way to collect scientific data on the efficacy of health interventions. Despite the advantages of RCT design in reducing extraneous variation that may confound interpretation of intervention results, the design may not be suitable for interventions in which patients are likely to have a strong preference for a particular treatment. Some designs incorporating patients or physician preferences by allowing at least a subgroup of them to choose their treatment have been proposed. In this chapter, we review various randomized control trials designs for accommodating participants’ and professionals’ preferences. Specifically, we discuss the advantages, limitations, applicability, ethical issues and statistical issues of each design. We also discuss the estimation of treatment effect (a measure of the extent to which treatment difference is attributable to treatments); selection effect (a measure of the extent to which treatment response is influenced by self-selection of treatment by patients); and preference effect (a measure of the extent to which treatment difference is caused by an interaction between the patient’s choice of treatment and the treatment actually received).

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Author information

Authors and Affiliations

  1. Department of Clinical Epidemiology and Biostatistics, McMaster University, Hamilton, ON, Canada

    Afisi S. Ismaila

  2. Department of Medicine, McMaster University, Hamilton, ON, Canada

    Stephen D. Walter

Authors
  1. Afisi S. Ismaila
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  2. Stephen D. Walter
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Correspondence to Afisi S. Ismaila .

Editor information

Editors and Affiliations

  1. Department of Biostatistics, Erasmus Medical Center, Rotterdam, The Netherlands

    Kees van Montfort

  2. Behavioural Science Institute, University of Nijmegen, Nijmegen, The Netherlands

    Johan Oud

  3. Department of Hematology, Erasmus Medical Center, Rotterdam, The Netherlands

    Wendimagegn Ghidey

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Ismaila, A.S., Walter, S.D. (2014). Review of Designs for Accommodating Patients’ or Physicians’ Preferences in Randomized Controlled Trials. In: van Montfort, K., Oud, J., Ghidey, W. (eds) Developments in Statistical Evaluation of Clinical Trials. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-55345-5_17

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