Dataset Information

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

ABSTRACT:

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.

Methods

Semi-structured interviews with key stakeholders (e.g. trialists, methodologists, lay members of study teams, bioethicists, and research ethics committee members), across multiple jurisdictions, identified based on their known experience and/or expertise with pragmatic trials.

Discussion

We expect that the study outputs will be of interest to a wide range of knowledge users including trialists, ethicists, research ethics committees, journal editors, regulators, healthcare policymakers, research funders and patient groups. All publications will adhere to the Tri-Agency Open Access Policy on Publications.

SUBMITTER: Nicholls SG

PROVIDER: S-EPMC6247737 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature

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Publications

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

Nicholls Stuart G SG Carroll Kelly K Brehaut Jamie J Weijer Charles C Hey Spencer Phillips SP Goldstein Cory E CE Zwarenstein Merrick M Graham Ian D ID McKenzie Joanne E JE McIntyre Lauralyn L Jairath Vipul V Campbell Marion K MK Grimshaw Jeremy M JM Fergusson Dean A DA Taljaard Monica M

BMC medical ethics 20181120 1

<h4>Background</h4>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 tr ...[more]

PMID: 30458809

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Dataset Information

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

Background

Methods

Discussion

Publications

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

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