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促进临床研究网络的国际互操作性,以应对未确诊和研究不足的罕见疾病。

Fostering the international interoperability of clinical research networks to tackle undiagnosed and under-researched rare diseases.

作者信息

Zanello Galliano, Chan Chun-Hung, Parker Samantha, Julkowska Daria, Pearce David A

机构信息

Institut National de la Santé et de la Recherche Médicale, Paris, France.

International Rare Diseases Research Consortium, Paris, France.

出版信息

Front Med (Lausanne). 2024 Nov 13;11:1415963. doi: 10.3389/fmed.2024.1415963. eCollection 2024.

Abstract

Clinical research is an essential component to advance diagnosis and therapeutic development. In 2022, the International Rare Diseases Research Consortium (IRDiRC) and the European Joint Programme on Rare Diseases (EJP RD) brought together key stakeholders from across the globe to discuss common themes in clinical research networks (CRNs) for rare diseases. Various topics were raised during discussions including current state of CRNs, the need for new CRNs, multi-stakeholder perspectives on value of CRNs, and ways to collaborate on a global scale. Communication and coordination between various groups, taking advantage of existing experiences, can expedite establishment and execution of complex collaborations that will be necessary for CRNs. In this perspective, we discuss opportunities and highlight key considerations for developing successful collaborative CRNs across the globe.

摘要

临床研究是推动诊断和治疗发展的重要组成部分。2022年,国际罕见病研究联盟(IRDiRC)和欧洲罕见病联合计划(EJP RD)召集了全球主要利益相关者,讨论罕见病临床研究网络(CRN)的共同主题。讨论中提出了各种话题,包括CRN的现状、建立新CRN的必要性、多方利益相关者对CRN价值的看法,以及全球范围内的合作方式。各团体之间的沟通与协调,利用现有经验,可以加快建立和开展CRN所需的复杂合作。从这个角度出发,我们讨论了相关机遇,并强调了在全球范围内建立成功的协作性CRN的关键考量因素。

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