Online Program Home
  My Program

All Times EDT

Abstract Details

Activity Number: 304 - Extrapolation as a Default Strategy in Pediatric Drug Development: Implications to Clinical Trial Designs and Statistical Analysis
Type: Topic Contributed
Date/Time: Wednesday, August 5, 2020 : 10:00 AM to 11:50 AM
Sponsor: Biopharmaceutical Section
Abstract #312866
Title: A Quantitative Method to Evaluate Exposure-Response Similarity Between Pediatrics and Adults to Support Efficacy Extrapolation
Author(s): Junshan Qiu and Margaret Gamalo-Siebers and Freda Cooner and Junjing Lin and Yodit Seifu and Yaning Wang*
Companies: FDA/CDER and Eli Lilly & Co and Amgen Inc. and Takeda and and FDA/CDER/DPM
Keywords: Exposure-Response; Similarity; Pediatrics; Non-parametric

US FDA has allowed extrapolation of efficacy from adults and an abbreviated pediatric development program where there is an expectation of a similar exposure-response (E-R) relationship in addition to a similar disease progression and treatment response in children, when compared to adults. Historically, the assessment of E-R similarity was based on visual inspection of two E-R curves. This talk will discuss a more quantitative method to compare exposure-response relationships between pediatric and adult populations to inform decision making in pediatric drug development. This method clarified that non-inferiority instead of similarity is required to extrapolate adult efficacy to pediatric patients. It also provided reliable objective criteria for non-inferiority assessment and decision making for efficacy extrapolation in pediatric population.

Authors who are presenting talks have a * after their name.

Back to the full JSM 2020 program