This collaborative workshop was hosted by the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) and the U.S. Food and Drug Administration (FDA). 

The presentation slides are available for download below, and the recordings of each presentation are accessible online.

FDA’s Division of Pediatrics and Maternal Health in collaboration with the Division of Anesthesiology, Addition Medicine, and Pain Medicine (DAAP) and the University of Maryland CERSI, hosted this two-day public workshop. The purpose of this public workshop was to discuss the current state of therapies to treat acute pain in children, identify data gaps, and consider methods to improve the current drug development paradigm for acute pain in patients under two years of age (e.g., use of pediatric extrapolation, and novel clinical trial designs).  The workshop was designed to focus on drugs with well-established mechanisms of action (NSAIDs, acetaminophen, local anesthetics, opioids), rather than drugs with novel mechanisms of action.



Wednesday, October 13, 2021
10:00 a.m. – 1:30 p.m. ET


Welcome &  Introduction: Setting the Scene

Lily Mulugeta, Pharm.D.
Division of Pediatrics and Maternal Health, FDA

Peter Stein, M.D.
Office of New Drugs, FDA


General Landscape of Extrapolation of Efficacy in Pediatric Drug Development and Ethical Considerations in Pediatric Drug Trials

Beth Durmowicz, M.D.
Office of Pediatric Therapeutics, FDA


Session 1: Extrapolation of Efficacy
Goals: To define an age limit for using PK-based extrapolation (i.e. exposure-matching)
Moderators: Lily Mulugeta, Pharm.D., (FDA) and Lisa Wiltrout, M.D. (FDA)


Development of Nociception and Pain

Suellen Walker, Ph.D.
UCL GOS Institute of Child Health


Pain Epidemiology in Neonates and Infant Patients

Types of pain encountered in each age cohort as compared to older pediatric patients, number of patients available for clinical trials, etc.

Ricardo Carbajal, M.D.
Hospital Armand Troussea


Developmental Pharmacology of Analgesics

What do we know about maturation of targets? Are there maturation-related changes to drug disposition or targets that may suggest need for lower or higher drug exposures in this age group to achieve response similar to older pediatric patients?

Chris McPherson, Pharm.D.
Washington University School of Medicine, St. Louis


Extrapolation of Adult Efficacy Data to Pediatric Patients

Given the available data, is there a biological reason to believe that drugs with well-established MOA (or drugs with well-established MOAs [opioids, acetaminophen, and local anesthetics]) would be less effective at similar concentrations in pediatric patients less than two years of age compared to older children? If so, in which age group and what are the uncertainties? Assessment of PK and safety would be required in all age groups. 


John van den Anker, M.D.
Children's National Hospital, Washington, D.C.


Panel Discussion
Moderators: Lily Mulugeta and Charles Berde



John Alexander (FDA), Yun Xun (FDA), Lisa Wiltrout (FDA), Tamorah Lewis, Suellen Walker, Gary Walco, Ellen Fields, Kanecia Zimmerman, John van den Anker


Closing Remarks


Charles Berde


Thursday, October 14, 2021
10:00 a.m. – 3:00 p.m. ET

Session 2: Trial Design Considerations in Acute Pain
Goals: Trial design and endpoint considerations in age cohorts where exposure-matching cannot be used as the basis for extrapolation of efficacy
Moderators: Lily Mulugeta (FDA) and Charles Berde


Welcome & Introductory Remarks: Setting the Scene (DPMH)

Lisa Wiltrout, M.D.
Division of Anesthesiology, Addiction Medicine, and Pain Medicine, FDA


Trial Design Considerations for Acute Pain in Neonates and Infants

Academic Perspective
Carolina Donado-Rincon
Boston Children's Hospital

Joe Kossowsky 
Boston Children's Hospital

Industry Perspective
Edress Darsey
Pfizer Pediatric Center of Excellence

Regulatory Perspective
Lisa Wiltrout, M.D.
Division of Analgesics and Anesthetics, FDA

Academic Perspective Slides

Industry Perspective Slides


Outcome Measures in Acute Pain Clinical Trials in Neonatal & Infant Populations

Monique van Dijk


PTN COA Development and PTN/NICHD Trials for Off Patent Drugs

Kanecia Zimmerman, M.D.
Duke University/Pediatric Trials Network


Brain-derived Approaches to Assess Neonatal & Infant Pain

Rebecca Slater, Ph.D.
University of Oxford


Innovative Trial Designs Including Bayesian Approaches

Brian Anderson



 Lunch Break


Considerations for PK/PD Studies

Amy Cheung, Ph.D.


Q&A and Moderated Panel Discussion
Moderators: Lily Mulugeta and Charles Berde

Rigo Roca (FDA), Gerri Baer (FDA), Lynne Yao (FDA), Jinglin Zhong (FDA), James Yates (GSK), Ellen Fields, Edress Darsey (Pfizer), Dina Metwally (UMD), Gary Walco, Kanecia Zimmerman, Tamorah Lewis


 Closing Remarks/Future Direction

Lynee Yao, M.D.



This workshop is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award U01FD005946 totaling $25,000 with 100 percent funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.