(5) Guidance for Pediatric Use in Prescription Information for Novel Medicinal Products in The EU and the US

Friday, August 26, 2022

8:00 AM – 6:00 PM CEST

Location: Hall C

Publication Number: 336

Presenting Author(s)

Helle Christiansen
– Copenhagen Centre for Regulatory Science, University of Copenhagen, Aarhus University, Copenhagen, Denmark

Co-Author(s)

Christine E. Hallgreen
– Copenhagen Centre for Regulatory Science, University of Copenhagen, Copenhagen, Denmark
Sven Frokjaer
– Copenhagen Centre for Regulatory Science (CORS), Denmark
Marie Louise De Bruin
– Professor, Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Faculty of Science, Utrecht University, 3584CG Utrecht, The Netherlands, Netherlands

Background: Mandatory pediatric legislation has been implemented in both the European Union (EU) and the United States (US) to increase research and availability of drugs to be used in the pediatric population. Differences in the legislative framework can cause different pediatric requirements for similar indications granted for similar drugs across jurisdictions and give rise to differences in the guidance for pediatric use provided for clinicians in the two regions.

Objectives: The objective of the study was to determine regional divergence in guidance for pediatric use for novel medicinal products approved by the EU and the US.

Methods: We compared the guidance for pediatric use in the EU Summary of Product Characteristics (SmPC) and the US Prescription Information (USPI) on the level of indications granted as of March 2020 for novel medicinal products approved in both regions between 2010 and 2018.

Results: The guidance for pediatric use differed for 18% (61/348) of the listed indications covering 21% (45/217) of the products, but without the guidance being contradictory. Where guidance differed, an equal share was observed for indications with a higher level of information for pediatric use in one region over the other (49% (30/61) in the US; 51% (31/61) in the EU). The discrepancies in pediatric information could be explained by differences in regulations for 21% (13/61) of the indications. Only a few conditions and diseases (EU n=4; US n=1) were observed to cover potential pediatric use outside the approved adult indication.

Conclusions: Although the EU Paediatric Regulation more often provides regulators a mandate for requiring pediatric drug development as compared to the US PREA, this was not reflected in the prescription information approved by the two regulatory authorities.