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

ABSTRACT:

Background

Rare diseases affect as many as 60 million people in the United States and Europe. However, most rare diseases lack effective therapies and are in critical need of clinical research. Our objective was to determine the frequency of noncompletion and nonpublication of trials studying rare diseases.

Methods and findings

We conducted a cross-sectional analysis of randomized clinical trials studying rare diseases as defined by the Genetic and Rare Disease Information Center database that were registered in ClinicalTrials.gov between January 1, 2010, and December 31, 2012, and completed or discontinued by December 31, 2014. Our main outcome measures were the frequency of trial noncompletion and, among completed studies, frequency of trial nonpublication at 2 and 4 years

SUBMITTER: Rees CA 

PROVIDER: S-EPMC6871779 | biostudies-literature | 2019 Nov

REPOSITORIES: biostudies-literature