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Review
. 2022 Feb;44(1):264-269.
doi: 10.1007/s11096-021-01303-5. Epub 2021 Sep 8.

Regulating pediatric off-label uses of medicines in the EU and USA: challenges and potential solutions : Comparative regulation framework of off label prescriptions in pediatrics: a review

Benedetta Guidi #  1 , Andrea Parziale #  2 , Luca Nocco  3 , Aniello Maiese  4 , Raffaele La Russa  5 , Marco Di Paolo  4 , Emanuela Turillazzi  4
Affiliations
Review

Regulating pediatric off-label uses of medicines in the EU and USA: challenges and potential solutions : Comparative regulation framework of off label prescriptions in pediatrics: a review

Benedetta Guidi et al. Int J Clin Pharm. 2022 Feb.

Abstract

Background Off-label uses of medicines are common in pediatrics. The literature reports that at least one-third of children in hospital and up to 90% of newborns in neonatal intensive care units receive off-label prescriptions. Moreover, the lack of data on safety and efficacy in the pediatric population may sometimes increase the risk of adverse drug reactions. Objective This paper aims to (a) map the main gaps in the existing EU and US regulatory frameworks for pediatric drug development and off-label use and (b) propose potential solutions for further discussion. Conclusion The selected jurisdictions aim to limit off-label prescribing, but implementation levels generally seem low, including in pediatric settings. Subject to further research, we propose potential concerted actions and initiatives of international cooperation to fill this gap. In particular, regulators and pediatric societies could strengthen manufacturers' incentives to develop pediatric medicines, pediatricians' information about off-label uses, and patients' and parents' awareness.

Keywords: Ethical and legal implications; European Union; Off-label prescription; Off-patent medicines; Pediatric population; United States.

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References

    1. Lenk C. Off-label drug use in paediatrics: a world-wide problem. Curr Drug Targets. 2012;13(7):878–84. - DOI
    1. Russo C, Stout L, House T, Santana VM. Barriers and facilitators of clinical trial enrollment in a network of community-based pediatric oncology clinics. Pediatr Blood Cancer. 2020;67(4):e28023. - DOI
    1. Schiavone S, Neri M, Pomara C, Riezzo I, Trabace L, Turillazzi E. Personalized medicine in the paediatric population: the balance between pharmacogenetic progress and bioethics. Curr Pharm Biotechnol. 2017;18(3):253–62. - DOI
    1. European Commission. Study on off-label use of medicinal products in the European Union.
    1. Bruxelles. 2017. Available online: https://ec.europa.eu/health/sites/health/files/files/documents/2017_02_2... .

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