Methodological insights from the EPISTOP trial to designing clinical trials in rare diseases-A secondary analysis of a randomized clinical trial.

Methodological insights from the EPISTOP trial to designing clinical trials in rare diseases-A secondary analysis of a randomized clinical trial.

<h4>Background</h4>In clinical research, the most appropriate way to assess the effect of an intervention is to conduct a randomized controlled trial (RCT). In the field of rare diseases, conducting an RCT is challenging, resulting in a low rate of clinical trials, with a high frequency...

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Main Authors: Stephanie Wied, Ralf-Dieter Hilgers, Nicole Heussen, Katarzyna Kotulska, Maya Dirani, Mathieu Kuchenbuch, Sergiusz Jozwiak, Rima Nabbout
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2024-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0312936
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