Room for improvement in clinical trials for rare diseases.

Abstract

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.