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

study protocol

Stuart G Nicholls (Corresponding Author), 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, Monica Taljaard

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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.

Original languageEnglish
Article number90
JournalJournal of Medical Ethics
Volume19
DOIs
Publication statusPublished - 20 Nov 2018

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Pragmatic Clinical Trials
Clinical Protocols
Ethics
pragmatics
stakeholder
Ethicists
Research Ethics Committees
Publications
research ethics
Ethical Analysis
Committee Membership
Health Services Research
Stakeholders
Ethical Issues
Randomized Controlled Trials
hypothesis testing
Learning
Interviews
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Nicholls, S. G., Carroll, K., Brehaut, J., Weijer, C., Hey, S. P., Goldstein, C. E., ... Taljaard, M. (2018). Stakeholder views regarding ethical issues in the design and conduct of pragmatic trials: study protocol. Journal of Medical Ethics, 19, [90]. https://doi.org/10.1186/s12910-018-0332-z

Stakeholder views regarding ethical issues in the design and conduct of pragmatic trials : study protocol. / Nicholls, Stuart G (Corresponding Author); Carroll, Kelly; Brehaut, Jamie; Weijer, Charles; Hey, Spencer Phillips; Goldstein, Cory E; Zwarenstein, Merrick; Graham, Ian D; McKenzie, Joanne E; McIntyre, Lauralyn; Jairath, Vipul; Campbell, Marion K; Grimshaw, Jeremy M; Fergusson, Dean A; Taljaard, Monica.

In: Journal of Medical Ethics, Vol. 19, 90, 20.11.2018.

Research output: Contribution to journalArticle

Nicholls, SG, Carroll, K, Brehaut, J, Weijer, C, Hey, SP, Goldstein, CE, Zwarenstein, M, Graham, ID, McKenzie, JE, McIntyre, L, Jairath, V, Campbell, MK, Grimshaw, JM, Fergusson, DA & Taljaard, M 2018, 'Stakeholder views regarding ethical issues in the design and conduct of pragmatic trials: study protocol', Journal of Medical Ethics, vol. 19, 90. https://doi.org/10.1186/s12910-018-0332-z
Nicholls, Stuart G ; Carroll, Kelly ; Brehaut, Jamie ; Weijer, Charles ; Hey, Spencer Phillips ; Goldstein, Cory E ; Zwarenstein, Merrick ; Graham, Ian D ; McKenzie, Joanne E ; McIntyre, Lauralyn ; Jairath, Vipul ; Campbell, Marion K ; Grimshaw, Jeremy M ; Fergusson, Dean A ; Taljaard, Monica. / Stakeholder views regarding ethical issues in the design and conduct of pragmatic trials : study protocol. In: Journal of Medical Ethics. 2018 ; Vol. 19.
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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.",
author = "Nicholls, {Stuart G} and Kelly Carroll and Jamie Brehaut and Charles Weijer and Hey, {Spencer Phillips} and Goldstein, {Cory E} and Merrick Zwarenstein and Graham, {Ian D} and McKenzie, {Joanne E} and Lauralyn McIntyre and Vipul Jairath and Campbell, {Marion K} and Grimshaw, {Jeremy M} and Fergusson, {Dean A} and Monica Taljaard",
note = "This work is supported by the Canadian Institutes of Health Research through the Project Grant competition (competitive, peer reviewed), award number PJT-153045. Jeremy Grimshaw holds a Canada Research Chair in Health Knowledge Transfer and Uptake and a CIHR Foundation Grant (FDN-143269). Charles Weijer holds a Canada Research Chair in Bioethics. Joanne McKenzie is supported by an Australian National Health and Medical Research Council Career Development Fellowship (1143429). Vipul Jairath hold a personal Endowed Chair at Western University (John and Susan McDonald Endowed Chair). Marion Campbell is based with the Health Services Research Unit which is core-funded by the Chief Scientist Office of the Scottish Government Health and Social Care Directorates. Ian Graham is a CIHR Foundation Grant recipient (FDN# 143237).",
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AU - Nicholls, Stuart G

AU - Carroll, Kelly

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AU - Weijer, Charles

AU - Hey, Spencer Phillips

AU - Goldstein, Cory E

AU - Zwarenstein, Merrick

AU - Graham, Ian D

AU - McKenzie, Joanne E

AU - McIntyre, Lauralyn

AU - Jairath, Vipul

AU - Campbell, Marion K

AU - Grimshaw, Jeremy M

AU - Fergusson, Dean A

AU - Taljaard, Monica

N1 - This work is supported by the Canadian Institutes of Health Research through the Project Grant competition (competitive, peer reviewed), award number PJT-153045. Jeremy Grimshaw holds a Canada Research Chair in Health Knowledge Transfer and Uptake and a CIHR Foundation Grant (FDN-143269). Charles Weijer holds a Canada Research Chair in Bioethics. Joanne McKenzie is supported by an Australian National Health and Medical Research Council Career Development Fellowship (1143429). Vipul Jairath hold a personal Endowed Chair at Western University (John and Susan McDonald Endowed Chair). Marion Campbell is based with the Health Services Research Unit which is core-funded by the Chief Scientist Office of the Scottish Government Health and Social Care Directorates. Ian Graham is a CIHR Foundation Grant recipient (FDN# 143237).

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N2 - 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.

AB - 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.

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JO - Journal of Medical Ethics

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