title: Beyond the treatment effect: Evaluating the effects of patient preferences in randomised trials.
abstract: The treatments under comparison in a randomised trial should ideally have equal value and acceptability - a position of equipoise - to study participants. However, it is unlikely that true equipoise exists in practice, because at least some participants may have preferences for one treatment or the other, for a variety of reasons. These preferences may be related to study outcomes, and hence affect the estimation of the treatment effect. Furthermore, the effects of preferences can sometimes be substantial, and may even be larger than the direct effect of treatment. Preference effects are of interest in their own right, but they cannot be assessed in the standard parallel group design for a randomised trial. In this paper, we describe a model to represent the impact of preferences on trial outcomes, in addition to the usual treatment effect. In particular, we describe how outcomes might differ between participants who would choose one treatment or the other, if they were free to do so. Additionally, we investigate the difference in outcomes depending on whether or not a participant receives his or her preferred treatment, which we characterise through a so-called preference effect. We then discuss several study designs that have been proposed to measure and exploit data on preferences, and which constitute alternatives to the conventional parallel group design. Based on the model framework, we determine which of the various preference effects can or cannot be estimated with each design. We also illustrate these ideas with some examples of preference designs from the literature.
authors.affiliation: 1 Clinical Epidemiology and Biostatistics, McMaster University, Hamilton, Ontario, Canada.
authors.forename: S D
authors.initials: SD
authors.lastname: Walter
authors.affiliation: 2 Screening and Test Evaluation Program, Sydney School of Public Health, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: R
authors.initials: R
authors.lastname: Turner
authors.affiliation: 2 Screening and Test Evaluation Program, Sydney School of Public Health, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: P
authors.initials: P
authors.lastname: Macaskill
authors.affiliation: 2 Screening and Test Evaluation Program, Sydney School of Public Health, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: K J
authors.initials: KJ
authors.lastname: McCaffery
authors.affiliation: 2 Screening and Test Evaluation Program, Sydney School of Public Health, University of Sydney, Sydney, New South Wales, Australia.
authors.forename: L
authors.initials: L
authors.lastname: Irwig
mesh_terms: D015444:Exercise; D006801:Humans; D050177:Overweight / Q000178:diet therapy / Q000628:therapy; D057240:Patient Preference; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D012107:Research Design; D057185:Sedentary Behavior; D057212:Therapeutic Equipoise; D016896:Treatment Outcome; D015431:Weight Loss
keywords: Randomised trials; preference effect; selection effect; treatment preferences; trial designs