Grand Rounds July 24, 2026: The Pediatric Trials Network: Innovating to Make Drugs Safe and Effective for Children (Rachel Greenberg, MD, MB, MHS; Danny Benjamin, MD, PhD, MPH)

Posted on by Sophia Ramirez

Speakers

Rachel Greenberg, MD, MB, MHS
Professor of Pediatrics
Duke University
MPI, Pediatric Trials Network

Danny Benjamin, MD, PhD, MPH
Kiser-Arena Distinguished Professor of Pediatrics
Duke University
Deputy Executive Director, Duke Clinical Research Institute
PI, Pediatric Trials Network

Keywords

Pediatric Clinical Trials; Off-Patent Therapeutics; Pediatric Trials Network; Platform Trials; Labeling; Dosing

Key Points

  • The Pediatric Trials Network (PTN) was established in 2010 to address the lack of pediatric labeling for off-patent therapeutics. It provides infrastructure for investigators to conduct trials that improve pediatric labeling and child health. The network performs studies to improve dosing, safety information, labeling, and ultimately, child health.
  • Since its inception, PTN has enrolled over 14,000 participants, with 301 sites in 45 states. Over the course of 54 studies, they have studied 21 therapeutic areas, submitted 34 products to the FDA, and instigated 26 label changes. They have developed innovative workarounds for several challenges associated with pediatric clinical trials, including limited blood volume; a limited number of available participants; and onerous contracting or start-up.
  • One such innovation was the implementation of 4 platform trials: POPs, CUDDLE, SCAMP, and LAPS. These trials answer more than one question at a time by evaluating multiple treatments or diseases within the same protocol.
  • The presenters shared several lessons learned over the course of the platform trials, pertaining to infrastructure; stakeholders and partners; flexibility; and the ability to pivot. They also discussed recent challenges that the network has faced, including a 40% reduction in funding and resulting trial terminations.

Discussion Themes

Platform trials offer a 10-to-1 savings ratio. While a traditional industry-led pediatric study might cost $25 million per molecule, the PTN can achieve similar results for approximately $2 million per molecule by studying them at scale.

The network continues to focus on reaching rural populations and families where clinical trial participation traditionally conflicts with daily life.