Meeting Summary
Published: April 2024
The MRCT Center hosted a 2-day hybrid meeting in Washington DC in October 2024 to examine the potential benefits, challenges, and opportunities of platform trials for pediatric populations. In addition to consideration of overarching issues, three diseases – pediatric oncology, major depressive disorder (MDD), and multidrug-resistant tuberculosis (MDR-TB) – were chosen to represent different conditions, epidemiology, settings, therapeutic challenges, and patient populations, and to illuminate different potential approaches and solutions.
With ~80 pediatric and platform trial experts from Europe, the UK, Asia, Australia and the US in attendance, the discussants were successfully identified knowledge gaps and practical challenges that impact pediatric platform trial planning and execution. Each of the three subgroups recommended actionable approaches to address identified issues, and the respective groups continued to meet and take concrete steps, furthering the momentum created at the workshop, with the MRCT Center remaining actively involved with each disease-specific subgroup to facilitate the next steps.
We appreciate the financial support of several companies, without whom this workshop would not have been possible. The workshop was supported in part by voluntary contributions from AstraZeneca, Johnson & Johnson, and Sanofi. Our thanks to all the individuals who contribute their time and expertise to the planning and conduct of this workshop and without whom this event would not have been possible. We are delighted to share the comprehensive workshop report, a detailed summary of the meeting proceedings.
Related Resource:
Podcast Episode: Advancing Pediatric Platform Trials – A Conversation with Dr. Danny Benjamin
Recorded during the MRCT Center’s October 2024 conference, Advancing Pediatric Platform Trials: Streamlining Development, Maximizing Impact, this keynote conversation features Dr. Danny Benjamin, Kiser-Arena Distinguished Professor of Pediatrics at Duke University Medical Center.
Discussion Highlights:
- The historical evolution of pediatric drug development and key regulatory milestones
- The role and achievements of the Pediatric Trials Network (PTN)
- How platform trials can increase efficiency and reduce burden in pediatric research
- Ethical and operational challenges unique to pediatric platform trials
- Real-world examples demonstrating the value of shared trial infrastructure
- Future directions to drive innovation, collaboration, and regulatory alignment
🔗 Listen to the full episode: EP 1: Advancing Pediatric Platform Trials – A Conversation with Dr. Danny Benjamin