Health-care professionals who prescribe medicines have the professional duty to choose medicines that are in the best interest of their individual patient, irrespective if that patient is an adult or a child. However, the availability of medicines with an appropriate label for pediatric use is lagging behind those for adults, and even available pediatric drugs are sometimes not suitable to administer to children. Consequently, health-care professionals often have no other option than to prescribe off-label medicines to children. An important reason for use of off-label medicines is to improve access to (innovative) treatments or to address medical needs and preferences of patients, especially when no other options are available. However, off-label use of medicines is in general not supported by the same level of evidence as medicines licensed for pediatric use. This may result in increased uncertainty on efficacy as well as the risk for toxicity and other side effects. In addition, liability may also be of concern, counterbalanced by professional guidelines.
Conclusion: The purpose of this joint EAP/ESDPPP policy statement is to offer guidance for HCPs on when and how to prescribe off-label medicines to children and to provide recommendations for future European policy.
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- BNF-c :
British National Formulary for Children
- CJEU :
Court of Justice of the European Union
- EAP :
European Academy of Pediatrics
- ESDPPP :
European Society for Developmental Perinatal and Pediatric Pharmacology
- GP :
- HCP :
- PICU :
Pediatric intensive care unit
Post-transplant lymphoproliferative disease
- NICU :
Neonatal intensive care unit
- NKFK :
Dutch Expertise Centre for Pharmacotherapy in Children
- RTU :
Recommendations for use
- SmPC :
Summary of product characteristics
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Tjitske van der Zanden, BSc managing director of the Dutch Expertise Centre for Pharmacotherapy in Children (NKFK), is kindly thanked for her expertise and critical review of the manuscript.
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Schrier, L., Hadjipanayis, A., Stiris, T. et al. Off-label use of medicines in neonates, infants, children, and adolescents: a joint policy statement by the European Academy of Paediatrics and the European society for Developmental Perinatal and Pediatric Pharmacology. Eur J Pediatr 179, 839–847 (2020). https://doi.org/10.1007/s00431-019-03556-9
- Off-label medicines
- European guidance
- Rational medicine use