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

Abstract

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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Abbreviations

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 :

General practitioner

HCP :

Health-care professional

PICU :

Pediatric intensive care unit

PTLD:

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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Acknowledgments

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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This paper was conceptualized by Lenneke Schrier and John van den Anker. The first draft of the manuscript was written by Lenneke Schrier, and all authors commented on previous versions of the manuscript. All authors made substantial contributions to this policy statement and revised it critically for important intellectual content. All authors read and approved the final manuscript to be published. All authors agree to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

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Correspondence to Lenneke Schrier.

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This article does not contain any studies with human participants or animals performed by any of the authors. Therefore, ethical approval or informed consent does not apply.

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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

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Keywords

  • Off-label medicines
  • European guidance
  • Rational medicine use
  • Pediatrics