Status of the Pediatric Clinical Trials Enterprise: An Analysis of the US ClinicalTrials.gov Registry

Abstract

BACKGROUND AND OBJECTIVES: Clinical trials are the gold standard for generating evidence-based knowledge in medicine. Recent legislation requiring trials to be registered at ClinicalTrials.gov has enabled evaluation of the clinical trial enterprise as a whole, which was previously not possible. The purpose of this study was to create a snapshot of the pediatric clinical trial portfolio. METHODS: All interventional trials registered at ClinicalTrials.gov from July 2005 to September 2010 were included. Pediatric (ie, enrolling patients aged 0–18 years) trial characteristics, therapeutic area, location, and funding were described. Secondary objectives included describing pediatric trials over time and comparison with nonpediatric trials. RESULTS: During this time, 5035 pediatric trials were registered compared with >10 times as many nonpediatric trials. Neonates/infants were eligible for enrollment in 46.6% of trials versus children (77.9%) and adolescents (45.2%). Nearly one-half of pediatric trials enrolled <100 subjects, and more pediatric trials versus nonpediatric trials evaluated preventive therapies. The proportion of pediatric trials evaluating a drug intervention declined over time, and there were fewer Phase 0 to II versus Phase III to IV trials. Infectious disease/vaccine studies (23%) were the most common, followed by psychiatric/mental health (13%) studies. Many trials enrolled patients outside the United States, and <15% of trials were sponsored by the National Institutes of Health or other US federal agencies. CONCLUSIONS: Analysis of the ClinicalTrials.gov data set allows description of the current scope of pediatric trials. These data may be useful to stakeholders in informing decisions regarding the conduct of trials in children and provide insight into mechanisms to advance pediatric trial infrastructure and methodology toward improving child health.