Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey

Abstract

Background/aim: Pediatric patients, especially those with rare diseases, represent a population that has a high tendency towards offlabel drug use (OLDU) and needs a more careful practice of pharmacotherapy than in adults. We aimed to investigate biotechnological drug use in children with rare diseases requiring OLDU. Materials and methods: This retrospective study examined all single-diagnosed OLDU applications (n= 5792) for 4992 children (