Abstract
This study examined the proportion of oral medications used on an off-label basis in pediatric patients and evaluated a novel method for identifying pediatric drug development needs. Using medical big data from Japan Medical Data Center, we identified candidate drugs through criteria-based selection and expert review of prescription information. Among the analyzed medicines, 67.4％ （746/1,107） were not indicated for pediatric use according to their package inserts. Among the medicines used on-label, only 19.0％ explicitly provided pediatric indications in the package insert. Among the 1,107 ingredients, 608 were initially selected using criteria-based selection. Subsequent expert reviews further refined this selection, resulting in a final list of 138 ingredients. The status of the top 20 most prescribed ingredients as of November 2024 was checked. One similar medicinal product had already been developed as a pediatric formulation, whereas two others had been identified by academic societies as requiring drug development. This method, which combines quantitative analysis of medical big data with expert qualitative assessment of candidate pharmaceuticals for pediatric drug development, appears efficient for identifying actual medical needs.
