Lochmüller Hanns, Ambrosini Anna, van Engelen Baziel, Hansson Mats, Tibben Aad, Breukel Alexandra, Sterrenburg Ellen, Schrijvers Guus, Meijer Ingeborg, Padberg George, Peay Holly, Monaco Lucia, Snape Mike, Lennox Anne, Mazzone Elena, Bere Nathalie, de Lemus Mencia, Landfeldt Erik, Willmann Raffaella
Children's Hospital of Eastern Ontario Research Institute, University of Ottawa, Ottawa, Canada and Division of Neurology, Department of Medicine, The Ottawa Hospital, Ottawa, Canada; Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, Freiburg, Germany.
Fondazione Telethon, Milan, Italy.
J Neuromuscul Dis. 2019;6(1):161-172. doi: 10.3233/JND-180368.
In the era of patient-centered medicine, shared decision-making (SDM) - in which healthcare professionals and patients exchange information and preferences and jointly reach a decision - has emerged as the gold standard model for the provision of formal healthcare. Indeed, in many geographical settings, patients are frequently invited to participate in choices concerning the design and delivery of their medical management. From a clinical perspective, benefits of this type of patient involvement encompass, for example, enhanced treatment satisfaction, improved medical compliance, better health outcomes, and maintained or promoted quality of life. Yet, although the theory and enactment of SDM in healthcare are well-described in the literature [1-3], comparatively less attention has been devoted to contextualizing questions relating to if, when, and how to include patients in decisions within medical research. In this context, patient involvement would be expected to be potentially relevant for and applicable to a wide range of activities and processes, from the identification of research priorities and development of grant applications, to the design of patient information and consent procedures, formulation of interventions, identification and recruitment of study sample populations, feasibility of a clinical trial, identification, selection, and specification of endpoints and outcomes in clinical trials and observational studies, data collection and analysis, and dissemination of results. To this end, 45 clinicians, healthcare professionals, researchers, patients, caregivers, and representatives from regulatory authorities and pharmaceutical companies from 15 different countries met to discuss the level of involvement of patients with neuromuscular diseases, specifically in the following settings of medical research for neuromuscular diseases: i) registries and biobanks; ii) clinical trials; and iii) regulatory processes. In this report, we present summaries of the talks that were given during the workshop, as well as discussion outcomes from the three topic areas listed above.
在以患者为中心的医学时代，共同决策（SDM）——即医疗保健专业人员与患者交流信息和偏好并共同做出决策——已成为提供正规医疗保健的金标准模式。事实上，在许多地区，患者经常被邀请参与有关其医疗管理设计和实施的选择。从临床角度来看，这种患者参与的益处包括，例如，提高治疗满意度、改善医疗依从性、取得更好的健康结果以及维持或提升生活质量。然而，尽管医疗保健中共同决策的理论和实施在文献中已有详尽描述[1 - 3]，但相对较少关注与在医学研究中是否、何时以及如何让患者参与决策相关的背景问题。在此背景下，预计患者参与可能与广泛的活动和流程相关且适用于这些活动和流程，从确定研究重点和撰写资助申请，到设计患者信息和同意程序、制定干预措施、确定和招募研究样本群体、临床试验的可行性、确定、选择和明确临床试验及观察性研究中的终点和结果、数据收集与分析以及结果传播。为此，来自15个不同国家的45名临床医生、医疗保健专业人员、研究人员、患者、护理人员以及监管机构和制药公司的代表齐聚一堂，讨论神经肌肉疾病患者的参与程度，特别是在神经肌肉疾病医学研究的以下方面：i）登记处和生物样本库；ii）临床试验；以及iii）监管流程。在本报告中，我们呈现了研讨会期间的演讲摘要以及上述三个主题领域的讨论结果。
