Pediatric Clinical Trials: The Need for Regulation

By Dr. Martine Dehlinger-Kremer, VP, Global Medical and Regulatory Affairs

Despite the fact that some labeling for children did exist since 1979, more than 80 percent of listed medications’ labels lacked prescribing information for children because most of them were being used off label. Why? Because 50-90 percent of medicines being used for children were not tested and evaluated in children. Even in hospitals, at least one third of hospitalized children and 90 percent of neonates in ICU were receiving off-label prescriptions. Needless to say, this was not a good situation due to the risks posed for ineffective under-dosing, or worse, adverse effects from overdosing. It was exceptionally hard to determine formulations for pediatric populations and this resulted in delayed access to innovative medicines for children.

But how could this problem be solved? There had been a limited number of clinical trials for children and a limited number of participants even in those trials. There was no suitable trial methodology and no real infrastructure for pediatric trials, in part due to a lack of funding for this research.  All of the regulatory bodies knew this situation had to change.