October 2, 2014
Developing orphan drugs for children is typically challenging due to the limited number of patients, age-dependent dosage and tight regulation to conduct clinical trials on minors. To date, around 500 drugs only are FDA approved for pediatric use. Drug repositioning represents a relatively quicker option to develop drugs for children as clinical testing for new indications does not require initial toxicity studies. On the other hand, testing existing drugs in children will require dose finding and pharmacokinetic studies.
In an article published in Drug Discovery Today, Blatt et al. introduce the Children’s Pharmacy CollaborativeTM, a non-exhaustive database of drugs reported to have been used previously in children. Based on existing databases, such as the Johns Hopkins Clinical Compound Library (JHCCL), the authors identified around 1,250 drugs reported to have been used in minors. This figure suggests that many drugs are therefore used off-label in children. The authors believe this first pediatric-focused database will help identify candidate drugs for repurposing in pediatrics.