Catherine Stratton, Andrew Taylor, Menelaos Konstantinidis, Vanda McNiven, Peter Kannu, Peter Gill, Ian Stedman, Areti Angeliki Veroniki, Martin Offringa, Beth Potter, Durhane Wong-Rieger, John Adams, Kathy Hodgkinson, Alison M Elliott, Alexandra Neville, Marie Faughnan, Sarah Dyack, Pavel Zhelnov, Jennifer Daly-Cyr, Jessie McGowan, Sharon Straus, Maureen Smith, Laura Rosella, Andrea C Tricco
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引用次数: 0
摘要
目标：本综述旨在确定设计、维护和利用罕见病患者登记处（RDPR）的障碍/促进因素；确定患者合作伙伴、其他知识用户（KUs）和研究人员在这些障碍/促进因素方面是否存在差异以及差异如何；并绘制罕见病和 RDPR 的定义图：RDPRs 对于提高对罕见病自然病史和预后因素的了解、评估干预措施以及确定临床试验的潜在参与者至关重要。然而，目前 RDPRs 的功能尚未完全优化。为了提高 RDPR 的质量和功能，必须了解其设计、维护和使用过程中存在的障碍和/或促进因素；患者合作伙伴、其他 KU 和研究人员之间可能存在的差异；以及界定罕见病和 RDPR 的定义范围：任何研究设计或格式（包括实证研究、书籍、手册、评论、社论、指导文件、会议摘要、综述文件和灰色文献）中提及设计、维护或使用 RDPR 的障碍/促进因素的证据都将被考虑纳入：审查将遵循 JBI 的范围审查方法。我们将检索健康科学数据库，包括 Cochrane 图书馆、Embase、MEDLINE、JBI EBP 数据库和 PsycINFO（从开始到现在），以及使用加拿大药物与健康技术局 (CADTH) 灰色事项指南检索灰色文献。两名独立审稿人将筛选标题和摘要、全文文件以及摘要数据。如有分歧，将通过讨论或与第三位审稿人协商解决。将对证据进行描述性综合，并使用 "系统综述和元分析首选报告项目"（Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews，PRIMSA-ScR）进行报告：开放科学框架 https://osf.io/mvf9r。
Barriers and facilitators to designing, maintaining, and utilizing rare disease patient registries: a scoping review protocol.
Objective: The objectives of this review are to identify barriers/facilitators to designing, maintaining, and utilizing rare disease patient registries (RDPRs); determine whether and how these differ among patient partners, other knowledge users (KUs), and researchers; and chart definitions of rare diseases and RDPRs.
Introduction: RDPRs are vital to improving the understanding of the natural histories and predictors of outcomes for rare diseases, assessing interventions, and identifying potential participants for clinical trials. Currently, however, the functionality of RDPRs is not fully optimized. To improve the quality and functionality of RDPRs, it is important to understand the barriers and/or facilitators involved in their design, maintenance, and utilization; how these might differ among patient partners, other KUs, and researchers; and to delineate the range of definitions for rare diseases and RDPRs.
Inclusion criteria: Evidence of any study design or format (including empirical studies, books, manuals, commentaries, editorials, guidance documents, conference abstracts, review documents, and gray literature) referencing barriers/facilitators for designing, maintaining, or utilizing RDPRs will be considered for inclusion.
Methods: The review will follow the JBI methodology for scoping reviews. We will search health science databases, including the Cochrane Library, Embase, MEDLINE, the JBI EBP Database, and PsycINFO, from inception onwards, as well as gray literature using the Canadian Agency for Drugs and Technologies in Health (CADTH) Grey Matters guidance. Two independent reviewers will screen titles and abstracts and full-text documents, as well as abstract data. Disagreements will be resolved through discussion or with a third reviewer. Evidence will be synthesized descriptively and reported using the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRIMSA-ScR).
Review registration: Open Science Framework https://osf.io/mvf9r.
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