EFGCP Better Medicines for Children Pre-Conference
The Fundamentals of Paediatric Extrapolation
In Spring 2022, the International Council for Harmonization (ICH) published the E11A draft guideline establishing a framework for extrapolation from existing data to the pediatric population. The new guideline aims to promote international harmonization of methodologies and strategies to incorporate pediatric extrapolation into overall drug development plans, improving the speed of access to new drugs for pediatric patients while limiting the number of children required for enrolment in clinical trials. The extrapolation framework consists of a pediatric extrapolation concept, and the creation and execution of an extrapolation plan.
This workshop has been designed to provide pediatric researchers, trial sponsors, and regulatory reviewers an opportunity to actively engage with experts to learn the fundamentals of developing a pediatric extrapolation concept and plan.
Attendees will participate in interactive small group discussions learning how to assess the similarities and differences of disease between a reference and a target population. Experts will also guide attendees through the development of an extrapolation plan - determining the relevant studies to be performed, analyzing whether model-informed approaches are appropriate and evaluating potential study designs. Workshop attendees will learn about the use of bridging biomarkers, Bayesian strategies and single arm pharmacokinetic/pharmacodynamic (PK/PD) studies and their application in pediatric extrapolation.
Opening of Conference
Welcome and Introduction to the pre-Conference Workshop
Christina Bucci-Rechtweg, Novartis Pharmaceuticals Corporation
Lecture 1 - Why Extrapolation is Used in Paediatric Medicines Development
Dionna Green, Director, Office of Pediatric Therapeutics, Office of the Commissioner, FDA
Lecture 2 - What Extrapolation is Used in Paediatric Medicines Development
Andrew Thomson, Statistician, EMA
Lecture 3 - How Extrapolation is Used in Paediatric Medicines Development
Skip Nelson, Senior Director, Pediatric Drug Development (CHILD) & Pediatric Strategy Lead, Pediatric Development Team, Immunology, Janssen
Introduction of the Breakout Sessions
Moderator(s): Ashley Strougo, Sanofi
Breakout Session 1 - Case Study 1 "Developing an Extrapolation Concept and Plan & Handling Uncertainties"
Break-Out Session 1 Report Back
Summary of Case 1 Outcome
Breakout Session 2 - Case Study 2 "Developing an Extrapolation Concept and Plan & Handling Uncertainties"
Break-Out Session 2 Report Back
Summary of Case 2 Outcome
Parting Comments & Key Take-aways
Lily Mulugeta, Associate Director, Division of Pediatric & Maternal Health, FDA
End of the Pre-Conference Workshop