The practice of active patient involvement in rare disease research using ICT: experiences and lessons from the RUDY JAPAN project.
The practice of active patient involvement in rare disease research using ICT: experiences and lessons from the RUDY JAPAN project.
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DOI：
10.1186/s40900-021-00253-6
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发表时间：
2021-02-01
影响因子：
--
通讯作者：
Kato K
中科院分区：
文献类型：
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作者：
Hamakawa N;Kogetsu A;Isono M;Yamasaki C;Manabe S;Takeda T;Iwamoto K;Kubota T;Barrett J;Gray N;Turner A;Teare H;Imamura Y;Yamamoto BA;Kaye J;Hide M;Takahashi MP;Matsumura Y;Javaid MK;Kato K
患者在医学研究中的角色正在发生变化，因为越来越多的患者参与，患者报告的结果越来越多地有助于临床决策。信息和通信技术为患者积极参与研究提供了新的机会。这些趋势在欧洲和美国特别明显，但在日本不太明显。本研究的目的是调查日本利用数字平台积极参与医学研究的实践，并分析结果，以阐明可以付诸实践的具体方法。我们开发了RUDY JAPAN系统，这是一个正在进行的罕见疾病医学研究平台，与英国的罕见和未诊断疾病研究（RUDY）项目合作。经过两年的筹备，RUDY JAPAN于2017年12月推出。最初选择骨骼肌通道病作为目标疾病，随后增加了遗传性血管性水肿。通过患者与研究人员的合作，设计了几种患者积极参与的方法，即指导委员会、问卷开发、动态同意和其他沟通策略。我们分析了我们的实践和经验，重点是每种方法如何影响和促进研究项目。RUDY JAPAN以各种方式成功地让患者参与到该研究项目中。虽然不是启动该项目的初始决策阶段的一部分，但从那时起，患者越来越多地参与其中。通过指导委员会（一个为RUDY JAPAN做出重大贡献的管理机构）和问卷制定过程，实现了高水平的患者参与。患者网络论坛、网站和通讯的创建培养了患者和研究人员之间的对话。登记研究本身允许患者通过数据输入参与，并通过动态同意控制数据使用。我们认为，初步结果证明了日本患者积极参与的可行性和实用性。通过RUDY JAPAN实现的合作是由数字技术实现的。它使忙碌的患者和研究人员能够找到会面的空间，共同为指导委员会、问卷编制和各种交流活动工作。虽然在日本患者积极参与的实践仍处于早期阶段，但这项研究证实了如果具备适当的条件，其可行性。(331话）。
The role of patients in medical research is changing, as more emphasis is being placed on patient involvement, and patient reported outcomes are increasingly contributing to clinical decision-making. Information and communication technology provides new opportunities for patients to actively become involved in research. These trends are particularly noticeable in Europe and the US, but less obvious in Japan. The aim of this study was to investigate the practice of active involvement of patients in medical research in Japan by utilizing a digital platform, and to analyze the outcomes to clarify what specific approaches could be put into practice. We developed the RUDY JAPAN system, an ongoing rare disease medical research platform, in collaboration with the Rare and Undiagnosed Diseases Study (RUDY) project in the UK. After 2 years of preparation, RUDY JAPAN was launched in December 2017. Skeletal muscle channelopathies were initially selected as target diseases, and hereditary angioedema was subsequently added. Several approaches for active patient involvement were designed through patient-researcher collaboration, namely the Steering Committee, questionnaire development, dynamic consent, and other communication strategies. We analyzed our practices and experiences focusing on how each approach affected and contributed to the research project. RUDY JAPAN has successfully involved patients in this research project in various ways. While not a part of the initial decision-making phase to launch the project, patients have increasingly been involved since then. A high level of patient involvement was achieved through the Steering Committee, a governance body that has made a major contribution to RUDY JAPAN, and the process of the questionnaire development. The creation of the Patient Network Forum, website and newsletter cultivated dialogue between patients and researchers. The registry itself allowed patient participation through data input and control of data usage through dynamic consent. We believe the initial outcomes demonstrate the feasibility and utility of active patient involvement in Japan. The collaboration realized through RUDY JAPAN was enabled by digital technologies. It allowed busy patients and researchers to find the space to meet and work together for the Steering Committee, questionnaire development and various communication activities. While the practice of active patient involvement in Japan is still in its early stages, this research confirms its viability if the right conditions are in place. (331 words).