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
Clinical Epidemiology Program, Ottawa Hospital Research Institute, Civic Campus, 1053 Carling Avenue, Civic Box 693, Admin Services Building, ASB 2-013, Ottawa, ON, K1Y 4E9, Canada.
School of Epidemiology and Public Health, University of Ottawa, Ottawa, ON, Canada.
BMC Med Ethics. 2018 Nov 20;19(1):90. doi: 10.1186/s12910-018-0332-z.
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.
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.
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.
随机对照试验（RCT）的试验设计存在于一个从解释性到实用性的连续谱上，这取决于一项研究旨在解决疗效问题还是有效性问题的程度。正如施瓦茨和勒卢什在1967年所概念化的那样，试验设计的解释性方法强调在理想条件下（疗效）对治疗作用机制进行假设检验，而实用性方法强调在现实世界条件下检验两种或更多可用治疗方法的有效性。近年来，对实用性试验的兴趣及其数量大幅增长，全球范围内的资助者和利益相关者越来越认识到需要可靠的证据来为临床决策提供信息。这种增长伴随着学习型医疗系统的出现，以及对以患者为导向的研究的日益关注。然而，实用性试验存在一些尚未被识别或充分描述的伦理挑战。本研究旨在探讨关键利益相关者对实用性试验的设计和实施所引发的伦理问题的看法。它是一个为期四年的大型项目的一部分，该项目旨在为实用性试验的伦理设计和实施制定指导方针。作为第一步，本研究将填补当前实证文献在识别实用性试验的设计和实施中产生的一系列全面伦理问题方面的重要空白。通过在我们的平行伦理分析中开辟广泛的主题以供考虑，我们将把目前主要强调同意问题的辩论扩展到可能源于特定设计选择的一系列伦理考量。
对关键利益相关者（如试验者、方法学家、研究团队的非专业成员、生物伦理学家和研究伦理委员会成员）进行半结构化访谈，这些利益相关者来自多个司法管辖区，是根据他们在实用性试验方面已知的经验和/或专业知识确定的。
我们预计该研究成果将引起广泛的知识使用者的兴趣，包括试验者、伦理学家、研究伦理委员会、期刊编辑、监管机构、医疗保健政策制定者、研究资助者和患者群体。所有出版物都将遵循三机构开放获取出版物政策。
