Drug safety in paediatric oncology. The contribution of therapeutic trials to pharmacovigilance

Malignancies in children are rare and have so far not been a focus of industrial drug development. Over the last decades, however, it was children in particular who benefited from new cytotoxic agents as paediatric centres, in joining the therapeutic trials of the German Society of Paediatric Oncology and Haematology (GPOH), have systematically introduced them. At present, over 90% of children with cancer are being treated in over 25 therapeutic studies, and more than 70% are being cured long-term. Such treatment is often given off-label, so observations on drug safety can mainly be obtained only within this system. This paper provides a summary of current legal regulations for documenting drug safety; it presents cases exemplifying the contribution of the aforementioned structures and studies on drug safety in children; and it makes a concluding proposal regarding the continued development of paediatric oncological pharmacovigilance.