Stakeholder views regarding ethical issues in the design and conduct of pragmatic trials: study protocol

BMC Medical Ethics
http://www.biomedcentral.com/bmcmedethics/content
(Accessed 24 Nov 2018 )

Study protocol
Stakeholder views regarding ethical issues in the design and conduct of pragmatic trials: study protocol
Randomized controlled trial (RCT) trial designs exist on an explanatory-pragmatic spectrum, depending on the degree to which a study aims to address a question of efficacy or effectiveness. As conceptualized b…
Authors: Stuart G. Nicholls, Kelly Carroll, Jamie Brehaut, Charles Weijer, Spencer Phillips Hey, Cory E. Goldstein, Merrick Zwarenstein, Ian D. Graham, Joanne E. McKenzie, Lauralyn McIntyre, Vipul Jairath, Marion K. Campbell, Jeremy M. Grimshaw, Dean A. Fergusson and Monica Taljaard
Citation: BMC Medical Ethics 2018 19:90
Published on: 20 November 2018
Background
Randomized controlled trial (RCT) trial designs exist on an explanatory-pragmatic spectrum, depending on the degree to which a study aims to address a question of efficacy or effectiveness. As conceptualized by Schwartz and Lellouch in 1967, an explanatory approach to trial design emphasizes hypothesis testing about the mechanisms of action of treatments under ideal conditions (efficacy), whereas a pragmatic approach emphasizes testing effectiveness of two or more available treatments in real-world conditions. Interest in, and the number of, pragmatic trials has grown substantially in recent years, with increased recognition by funders and stakeholders worldwide of the need for credible evidence to inform clinical decision-making. This increase has been accompanied by the onset of learning healthcare systems, as well as an increasing focus on patient-oriented research. However, pragmatic trials have ethical challenges that have not yet been identified or adequately characterized. The present study aims to explore the views of key stakeholders with respect to ethical issues raised by the design and conduct of pragmatic trials. It is embedded within a large, four-year project that seeks to develop guidance for the ethical design and conduct of pragmatic trials. As a first step, this study will address important gaps in the current empirical literature with respect to identifying a comprehensive range of ethical issues arising from the design and conduct of pragmatic trials. By opening up a broad range of topics for consideration within our parallel ethical analysis, we will extend the current debate, which has largely emphasized issues of consent, to the range of ethical considerations that may flow from specific design choices.