Room for improvement in clinical trials for rare diseases.,Nature Reviews Rheumatology

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Room for improvement in clinical trials for rare diseases.
Nature Reviews Rheumatology ( IF 29.4 ) Pub Date : 2020-03-01 , DOI: 10.1038/s41584-020-0376-6
Anne Musters _{1,

2} , Sander W Tas _{1,

2}

Affiliation

Trials studying rare diseases, including immune-mediated inflammatory diseases such as Castleman disease, hyper IgD syndrome and undifferentiated vasculitis, frequently end in noncompletion and nonpublication. A new cross-sectional analysis identifies underlying reasons, but also calls for action to establish international networks that might facilitate recruitment of patients into trials and to ensure timely publication of research findings to advance the field.

中文翻译：

罕见病临床试验有待改进。

研究罕见疾病的试验，包括免疫介导的炎症性疾病，如 Castleman 病、高 IgD 综合征和未分化血管炎，经常以未完成和未发表的方式结束。一项新的横断面分析确定了根本原因，但也呼吁采取行动建立国际网络，这可能有助于招募患者参加试验，并确保及时发表研究结果以推动该领域的发展。

更新日期：2020-01-29

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