Laws requiring or encouraging pharmaceutical companies to conduct pediatric clinical trials of medications are helping clinicians gain useful information, according to a report from the Institute of Medicine (IOM). But the authors also identified the need for improvements, such as greater transparency about trial results and greater clarity about the potential benefits a trial may offer children.
Most drugs have never been studied in children, yet physicians often prescribe products studied in adults to children without the benefit of clinical evidence to support the safety and efficacy of the product for the patient's age group and without evidence-based information on dosing. Although this off-label use is a legal and accepted clinical practice, it can sometimes have unintended consequences. Physiological differences between adults and children may mean that a drug that is safe and effective in adults may not be so in children or that scaling down adult dosing regimens based on weight may not be appropriate. For example, the IOM report (http://tinyurl.com/6oqk65r) notes that in 1982, 16 premature infants died from respiratory complications after receiving intravenous or diluted medications containing benzyl alcohol. No signs of harm had been seen in administering such products to adults or older children.
Kuehn BM. Laws Boost Pediatric Clinical Trials, But Report Finds Room for Improvement. JAMA. 2012;307(16):1681-1682. doi:10.1001/jama.2012.508