Noncompletion and nonpublication of trials studying rare diseases: A cross-sectional analysis release_hhxlz7laenedtdzko7lpmcxeea

by Chris A. Rees, Natalie Pica, Michael C. Monuteaux, Florence T. Bourgeois


In this study, over half of clinical trials initiated for rare diseases were either discontinued or not published 4 years after completion, resulting in large numbers of patients with rare diseases exposed to interventions that did not lead to informative findings. Concerted efforts are needed to ensure that participation of patients in rare disease trials advances scientific knowledge and treatments for rare diseases.
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Published in PLoS Medicine by Public Library of Science (PLoS)
ISSN-L 1549-1277
Volume 16
Issue 11
Page(s) e1002966
Release Date 2019-11-21
Container Type journal
Publisher Public Library of Science (PLoS)
Primary Language en (lookup)

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Type  article-journal
Stage   published
Date   2019-11-21
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ISSN-L:  1549-1277
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