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January 17, 2017

The Pediatric Research Equity Act Moves Into Adolescence

Author Affiliations
  • 1Computational Health Informatics Program, Boston Children’s Hospital, Boston, Massachusetts
  • 2Department of Pediatrics, Harvard Medical School, Boston, Massachusetts
  • 3Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital, Boston, Massachusetts
JAMA. 2017;317(3):259-260. doi:10.1001/jama.2016.18131

Children continue to be underrepresented as participants in clinical trials, limiting the evidence available to guide treatment decisions. Among new interventional trials registered on ClinicalTrials.gov in 2015, only 6% of 19 239 trials focused on children from birth to 17 years of age, even though this age group comprises about a quarter of the US population. As a result, clinicians frequently use medications tested in adults for the treatment of children and adolescents. In one study, rates of off-label prescribing were estimated to involve 85% of 57 000 hospitalized children nationally.1 Without adequate evidence to support these interventions, children may be exposed to serious unintended harms. Notable examples include the off-label use of verapamil to treat children with supraventricular tachycardia (associated with hypotension and death) and the antimicrobial chloramphenicol administered to infants (leading to fatal circulatory collapse).