Notes from presentations delivered at the Pediatric Drug Development: Use of Exposure Matching and Exposure-Response for Extrapolation of Efficacy in Pediatric Product Development have been provided below for the convenience of the attendees.
Opportunities and Challenges in Pediatric Drug Development and Regulatory Science
Gil Burckart, PharmD
Associate Director for Pediatrics
Office of Clinical Pharmacology
Food and Drug Administration
Pediatric Extrapolation: Using Exposure As A Surrogate for Efficacy
Robert "Skip" Nelson, MD, PhD
Senior Pediatric Ethicist and Lead Medical Officer
Office of Pediatric Therapeutics
Office of the Commissioner
Food and Drug Administration
FDA Perspective: Exposure-Response Assessments and Applications to Drug Development in the FDA
Kevin Krudys, PhD
Pharmacometrics Reviewer
Office of Clinical Pharmacology
Food and Drug Administration
What Constitutes A Meaningful Endpoint for Establishing Exposure-Response Similarity Between Adults and Pediatric Patients?
Lynne Yao, MD
Associate Director
Division of Pediatric and Maternal Health
Food and Drug Administration
Case Examples: Exposure-response to Support Extrapolation of Efficacy of IBD for Children
Kerry Jo Lee, MD
Pediatric Gastroenterologist, Gastroenterology and Inborn Errors Products
Office of New Drugs
Food and Drug Administration
Assessing Quality and Quantity of Data to Establish Exposure-Response Similarity Between Adults and Pediatric Patients: PEACE Initiative
Angela Men, MD, PhD
Neurology Team Leader
Food and Drug Administration
Case Examples: Extrapolation of Efficacy for JIA in Children
Satjit Brar, PharmD, PhD
Team Leader, Clinical Pharmacology
Food and Drug Administration
Dealing with Uncertainty of Extrapolation Assumptions
Tarek Leil, PhD
Head, Quantitative Clinical Pharmacology Group
Bristol-Myers Squibb
Experience in FDA Submissions with Matching Pediatric Drug Exposure to Adult Drug Exposure
Lily Mulugeta, PharmD
Pediatric Clinical Pharmacologist
Office of Clinical Pharmacology
Food and Drug Administration
Case Examples: Extrapolation of Efficacy of GERD in Children
Insook Kim, PhD
Clinical Pharmacology Reviewer
Food and Drug Administration
The Pediatric Trials Network: Experience with Matching Drug Exposure in Infants and Neonates to Adult Drug Exposure (not available)
Daniel Gonzalez, PharmD, PhD
Assistant Professor
Department of Pharmacotherapy and Experimental Therapeutics
University of North Carolina Eshelman School of Pharmacy
Methods for Determining Similarity of Exposures Between Adult and Pediatric Patients and Trial: Design Considerations
Marc R. Gastonguay, PhD
President & Chief Executive Officer
Metrum Research Institute
When Does Exposure Matching Require Additional Consideration?
Jeff Barrett, PhD
Vice President, Interdisciplinary Pharmacometrics Program
Sanofi