Cases of drug repositioning in children's orphan drugs: Licenced drugs versus unlicenced magistral preparations

Pharmacological treatments of several paediatric diseases are limited by the lack of medicinal products properly indicated for the children. A solution for overcoming such issue may be found in drug repositioning, based on an established clinical use of off-label medicines or extemporaneous preparations. This study aims to discuss the use of licenced products and magistral preparations, which can be compounded in hospital pharmacies, intended to treat rare diseases affecting childhood. A huge cost gap is observed between compounded and industrial drug products, suggesting a clear need in reconsidering the cost-effectiveness of repositioned products, and finding a balance between the manufacturers' economic sustainability and the patients’ access to therapies.