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Research Letter
April 2016

Characteristics and Public Availability of Results of Clinical Trials on Rare Diseases Registered at Clinicaltrials.gov

Author Affiliations
  • 1Centre de Recherche Epidémiologie et Statistique, INSERM U1153, Paris, France
  • 2Centre d'Epidémiologie Clinique, Hôpital Hôtel-Dieu, Assistance Publique–Hôpitaux de Paris, Paris, France
  • 3Faculté de Médecine, Université Paris Descartes, Sorbonne Paris Cité, Paris, France
  • 4French Cochrane Centre, Paris, France
  • 5medical student at Faculté de Médecine, Université Paris Descartes, Sorbonne Paris Cité, Paris, France
  • 6Department of Epidemiology, Mailman School of Public Health, Columbia University, New York, New York
JAMA Intern Med. 2016;176(4):556-558. doi:10.1001/jamainternmed.2016.0137

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.

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