Boers Maarten, Idzerda Leanne, Kirwan John R, Beaton Dorcas, Escorpizo Reuben, Boonen Annelies, Magasi Susan, Sinha Ian, Stucki Gerold, Tugwell Peter
From the Department of Epidemiology and Biostatistics, VU University Medical Center, Amsterdam, The Netherlands; Centre for Global Health Research, Institute of Population Health, University of Ottawa, Ottawa; University of Bristol, Academic Rheumatology Unit, Bristol Royal Infirmary, Bristol, UK; Mobility Program Clinical Research Unit, Li Ka Shing Knowledge Institute of St. Michael's Hospital, Toronto, Canada; International Classification of Functioning, Disability, and Health (ICF) Research Branch in cooperation with the World Health Organization (WHO) Collaborating Centre for the Family of International Classifications in Germany; Swiss Paraplegic Research (SPF), Nottwil; Department of Health Sciences and Health Policy, University of Lucerne, Lucerne; and at SPF, Nottwil, Switzerland; Department of Internal Medicine, Division of Rheumatology, Maastricht University Medical Center and Caphri Research Institute, Maastricht, The Netherlands; Department of Medical Social Sciences, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, USA; Respiratory Unit, Alder Hey Children's Hospital, Liverpool, UK; Department of Medicine, University of Ottawa, Ottawa, Canada.
J Rheumatol. 2014 May;41(5):978-85. doi: 10.3899/jrheum.131307. Epub 2014 Mar 1.
The Outcome Measures in Rheumatology (OMERACT) international consensus initiative has successfully developed core sets of outcome measures for trials of many rheumatologic conditions, but its expanding scope called for clarification and updating of its underlying conceptual framework and working process. To develop a core set of what we propose to call outcome measurement instruments, consensus must be reached both on what to measure and how to measure. This article deals with the first part: a framework necessary to ensure comprehensiveness of the domains chosen for measurement. We formulated a conceptual framework of core measurement areas in clinical trials, for discussion at the OMERACT 11 conference.
We formulated a framework and definitions of key concepts adapted from the literature, and followed an iterative consensus process (small group processes and an Internet-based survey) of those involved including patients, health professionals, and methodologists within and outside rheumatology.
The draft framework comprises 4 core "areas": death, life impact (all aspects of how a patient feels or functions), resource use (monetary and other costs of the health condition and interventions), and pathophysiologic manifestations (disease-specific clinical and psychological signs, biomarkers, and potential surrogate outcome measures necessary to assess specific effects). The survey responses (262 of 2293, response rate 11%) indicated broad agreement with the draft framework and the proposed definitions of key concepts, including understandability and feasibility. A total of 283 comments were processed.
In an iterative process, we have developed a generic framework for outcome measurement and working definitions of key concepts ready for discussion at the OMERACT 11 conference.
风湿病疗效评估指标国际共识倡议（OMERACT）已成功为多种风湿病试验制定了核心疗效评估指标集，但其范围不断扩大，需要对其基础概念框架和工作流程进行澄清和更新。要制定一套我们提议称为疗效测量工具的核心指标集，必须就测量内容和测量方法达成共识。本文探讨第一部分内容：确保所选测量领域全面性所需的框架。我们制定了一个临床试验核心测量领域的概念框架，以供在OMERACT 11会议上讨论。
我们根据文献制定了一个框架和关键概念的定义，并对包括患者、卫生专业人员以及风湿病领域内外的方法学家在内的相关人员进行了迭代式共识达成过程（小组讨论和基于互联网的调查）。
框架草案包括4个核心“领域”：死亡、生活影响（患者感受或功能的各个方面）、资源利用（健康状况和干预措施的金钱及其他成本）以及病理生理表现（疾病特异性的临床和心理体征、生物标志物以及评估特定效应所需的潜在替代结局指标）。调查回复（2293份中的262份，回复率11%）表明对框架草案和关键概念的提议定义广泛认同，包括可理解性和可行性。共处理了283条评论。
通过迭代过程，我们制定了一个疗效测量通用框架以及关键概念的工作定义，准备好在OMERACT 11会议上进行讨论。
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