罕见疾病临床研究网络:临床试验准备的模型
Joanne M Lumsden1, Tiina K Urv2
1Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences, National Institutes of Health, 6801 Democracy Boulevard, Bethesda, MD 20892-0001, USA.
Therapeutic advances in rare disease
|December 28, 2023
概括
罕见疾病临床研究网络 (RDCRN) 在过去的20年里大大推进了罕见疾病治疗的发展. 这个网络已经产生了高影响力的出版物,并为FDA批准的八种治疗方法做出了贡献,证明了它在罕见疾病研究中的关键作用.
科学领域:
- * 罕见疾病研究和治疗开发.
- *翻译和临床研究基础设施.
- *生物医学研究的生产力和影响分析.
背景情况:
- *为罕见疾病开发治疗方法具有挑战性,需要改进流程和基础设施.
- *国家卫生研究院 (NIH) 支持的罕见疾病临床研究网络 (RDCRN) 建立以加强罕见疾病的诊断,管理和治疗.
- * RDCRN促进以患者为中心的协作,多地点的研究,旨在创新治疗开发方法.
研究的目的:
- * 概述RDCRN在其20年历史中的生产力和影响力.
- *评估网络对罕见疾病研究和治疗开发的贡献.
主要方法:
- *进行了文献计量,共同作者和共同发生的分析.
- *使用了来自全球赠款,专利,出版物,临床试验和FDA批准的药物的全面,精心策划的数据集的数据.
- *使用NIH工作人员可用的工具进行数据分析.
主要成果:
- *RDCRN的生产力非常高,产生了2763个高质量的出版物,被引用超过10万次.
- * 该网络扩大了国际合作和科学贡献.
- * RDCRN的研究已经为FDA批准的8种罕见病治疗方法做出了贡献.
结论:
- * RDCRN计划成功克服了罕见疾病治疗开发方面的重大挑战.
- * 持续的灵活性和创新对于应对未来在推进罕见病科学方面的挑战至关重要.
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