As an obligation to participants and to ensure evidence-based decision making, results of trials must be reported. Rare diseases are defined by a prevalence of fewer than 200 000 people affected in the United States,1,2 but overall they may affect up to 25 million people in North America.2 The limited number of patients for each rare disease makes research in this field challenging2 and availability of results even more crucial.3 We aimed to describe the characteristics of completed phase 3 or 4 trials of rare diseases registered at clinicaltrials.gov and to assess whether their results were publicly available.
Dechartres A, Riveros C, Harroch M, Faber T, Ravaud P. Characteristics and Public Availability of Results of Clinical Trials on Rare Diseases Registered at Clinicaltrials.gov. JAMA Intern Med. 2016;176(4):556-558. doi:10.1001/jamainternmed.2016.0137