EFGCP Better Medicines for Children Pre-Conference 2023

Pediatric Dose Optimization


Event Workshop
Type Virtual Event
Language English
Date(s) 12 Oct. 2023
Day(s) 1 Day(s)
Duration 4 hours

Dose optimization is integral to maximizing pediatric medicines development. Variability in pharmacokinetic and pharmacodynamic parameters across the pediatric population by size and/or age can increase the complexity associated with identifying the optimized dose for use of a new medicine. Understanding how to design and optimize selection of a pediatric dose leveraging existing data, employing dose-finding designs that maximize small patient numbers, or utilize quantitative approaches, is essential to maximizing safety, effectiveness and tolerability of new pediatric medicines.

This workshop has been designed to provide pediatric researchers, trial sponsors, and regulatory reviewers an opportunity to actively engage with experts on the fundamentals of developing a strategy for pediatric dose optimization.

Attendees will participate in interactive small group discussions utilizing case examples. Experts will guide attendees through the development of an anonymized pediatric dosing strategy utilizing two case examples – determining the relevance of available data for use in the pediatric program design, analysing whether model-informed approaches are appropriate, and/or evaluating and proposing study designs.

This event is complementary to the Better Medicines for Children Conference 2023, held in Brussels, Belgium on 17 & 18 October 2023. For more information on the Paediatric Conference 2023, see the EFGCP Better Medicines for Children Conference 2023 Webpage.

Event Format: Virtual Zoom Event - Not Recorded


Day 1 - Half Day Afternoon
13:00 Welcome & Introduction to the pre-Conference Workshop
Christina Bucci-Rechtweg, Novartis
13:10 Lecture 1 – Why is Dose Optimization Integral to Pediatric Medicines Development?
John van den Anker, Children’s National USA & Universitats-Kinderspital beider Basel
13:25 Lecture 2 – What is Pediatric Dose Optimization?
Jeff Barrett, Aridhia
13:40 Lecture 3 – How is Dose Optimization Used in Pediatric Medicines Development?
S. Y. Amy Cheung, Certara
13:55 Introduction to the Breakout Sessions
Breakout Session One
14:00 Case Study 1 - Audience Split
14:50 Report Back
15:05 Summary of Case Study
15:15 Break
Breakout Session Two
15:25 Case Study 2 - Audience Split
16:15 Report Back
16:30 Summary of Case Study
16:40 Parting Comments & Key Take-aways
17:00 Thank you and closure
Christina Bucci-Rechtweg, Novartis