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CLINICAL TRIALS

Room for improvement in clinical trials for rare diseases

Nature Reviews Rheumatology volume 16, pages 131–132 (2020)Cite this article

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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.

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Authors and Affiliations

  1. Department of Rheumatology & Clinical Immunology, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands

    Anne Musters & Sander W. Tas

  2. Amsterdam Infection & Immunity Institute and Amsterdam Rheumatology & Immunology Center (ARC), Amsterdam, Netherlands

    Anne Musters & Sander W. Tas

Authors
  1. Anne Musters
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  2. Sander W. Tas
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Correspondence to Sander W. Tas.

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The authors declare no competing interests.

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Musters, A., Tas, S.W. Room for improvement in clinical trials for rare diseases. Nat Rev Rheumatol 16, 131–132 (2020). https://doi.org/10.1038/s41584-020-0376-6

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