Module232025

12/05/2025

Why Paediatric Clinical Development?

The Organisation for Professionals in Regulatory Affairs

3

M Dehlinger-Kremer, 14 May 2025

3

Why are Evaluation of Medicines to be Performed in the Paediatric Population?

Current off-label use

- Approximately 50-70% of medicines given to children have not been tested in children 1, 2 - Up to 90% of drugs used in neonatal intensive care units are administered off-label 3 - Imprecise and variable dosing - High use of “Extemporaneous Formulations”

Regulatory requirements

- Regulations are requesting drug developers, when applicable, to evaluate new treatments in the paediatric population

1. Hang TJ, Orenstein L, Kesselheim AS & Bourgeois FT Completion Rate and Reporting of Mandatory Pediatric Post marketing Studies Under the US Pediatric Research Equity Act. JAMA Pediatr 173, 68–74 (2019). [PubMed: 30452498] 2. Hoon D et. al., Trends in Off-Label Drug Use in Ambulatory Settings: 2006-2015. Pediatrics, 16 Sep 2019,144(4):e20190896 3. Balan S, HassaliMAA & MakVSL Two decades of off-label prescribing in children: a literature review. World J Pediatr 14, 528–540 (2018). [PubMed: 30218415]).

The Organisation for Professionals in Regulatory Affairs

4

M Dehlinger-Kremer, 14 May 2025

4