Pediatric Extrapolation: Unlocking the Clinical Pharmacology Toolbox to Accelerate Drug Access for Our Little Patients

Session Description

Pediatric patients often experience a delay in accessing novel medications compared to adults, leading to unmet medical needs or off-label drug use. The delay in drug availability mainly originates from the requirement for conducting one or more phase 3 clinical trials in the pediatric population upon approval of the adult indication. These trials typically have slow enrollment rates that could last for years. In addition, having regular clinic visits as well as clinical/laboratory assessments, and the possibility of receiving placebo in a trial impose a significant clinical and mental burden on participating pediatric patients and parents.

Pediatric extrapolation offers an alternative, where the efficacy of study drug in the pediatric patients is extrapolated from the respective adult patient populations or other pediatric patient populations using modeling and simulation approaches. Appropriate application of pediatric extrapolation can be used in lieu of, or reduce the sample size of, phase 3 trials and can help expedite the pediatric drug development timelines, ultimately enabling earlier access to novel medications for pediatric patients.

This session will provide an overview of pediatric extrapolation and the recent trends and advances of its use in pediatric drug development. In addition, two case studies will be presented, showcasing the utilization of extrapolation to enable expedited approval for small molecule and biologic drugs for pediatric indications in the children/adolescent and neonate/infant populations.

Learning Objectives

1. Understand the principles of pediatric extrapolation and perspectives on when it can be used.
2. Understand the recent trends and advances in the use of pediatric extrapolation.
3. Understand how strategy and application of pediatric extrapolation are made under real-world pediatric drug development scenarios.

Session Schedule

Introduction
Yuli Qian, PhD and Aarti Sawant, PhD

The Evolution of Pediatric Extrapolation to Support Pediatric Drug Development
Gilbert Burckart, PharmD

The Art and Power of Extrapolation: Bringing Upadacitinib to Pediatric Arthritis Patients Three Years Ahead of Schedule
Mohamed-Eslam Mohamed, RPh, PhD

Protecting Our Littlest Patients from RSV: PK-based Extrapolation of Nirsevimab Efficacy to Cover All Infants
Lindsay Clegg, PhD

Panel Discussion