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).
Bourgeois FT, Hwang TJ. The Pediatric Research Equity Act Moves Into Adolescence. JAMA. 2017;317(3):259–260. doi:10.1001/jama.2016.18131
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