The RUDY study platform - a novel approach to patient driven research in rare musculoskeletal diseases
The RUDY study platform - a novel approach to patient driven research in rare musculoskeletal diseases
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DOI：
10.1186/s13023-016-0528-6
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发表时间：
2016-11-08
影响因子：
3.7
通讯作者：
Kaye, J.
中科院分区：
文献类型：
--
作者：
Javaid, M. K.;Forestier-Zhang, L.;Kaye, J.
背景：随着人们认识到诊断和治疗护理方面存在重大差距，对罕见疾病的研究变得越来越普遍。登记被认为是解决罕见疾病的关键研究方法。本报告描述了英国罕见疾病研究 (RUDY) 平台的结构，该平台旨在改进研究流程并解决开展罕见肌肉骨骼疾病研究的许多挑战。RUDY 是一个基于互联网的平台，具有在线注册、初步口头同意、在线捕获患者报告的结果措施和动态同意框架内的事件。描述了数据库结构、安全性和治理框架。 结果：RUDY 已招募 380 名参与者，问卷完成率超过 50%。其中一名参与者退出，两名参与者修改了他们的同意选项。结论：RUDY 的优势包括临床团队负担轻、研究管理成本低、参与者招募多以及数据收集和访问方便。该平台有潜力用作全球其他罕见疾病的模型。
Background: Research into rare diseases is becoming more common, with recognition of the significant diagnostic and therapeutic care gaps. Registries are considered a key research methodology to address rare diseases. This report describes the structure of the Rare UK Diseases Study (RUDY) platform that aims to improve research processes and address many of the challenges of carrying out rare musculoskeletal disease research.RUDY is an internet-based platform with online registration, initial verbal consent, online capture of patient reported outcome measures and events within a dynamic consent framework. The database structure, security and governance framework are described.Results: There have been 380 participants recruited into RUDY with completed questionnaire rates in excess of 50 %. There has been one withdrawal and two participants have amended their consent options.Conclusions: The strengths of RUDY include low burden for the clinical team, low research administration costs with high participant recruitment and ease of data collection and access. This platform has the potential to be used as the model for other rare diseases globally.