Advancing Platform Trials for Pediatric Drug Development

Advancing Platform Trials for Pediatric Drug Development

Dates: October 29 – 30, 2024; 8:00 AM – 4:00 PM

We are delighted that you will be attending the MRCT Center’s invitation-only two-day workshop, Advancing Pediatric Platform Trials: Streamlining Development, Maximizing Impact, on October 29-30, 2024, at The Ven at Embassy Row, Washington, DC. You will be part of a diverse group of multi-stakeholder participants, each with deep expertise in critical areas related to the design and conduct of pediatric platform trials.

In addition to keynote addresses and panel discussions, three disease areas have been selected to address various challenges and opportunities in pediatric drug development: major depressive disorder, multi-drug-resistant tuberculosis, and pediatric hematological malignancies.

This solution-focused workshop aims to reinforce the strengths of platform trials by advancing the principles, ethical foundations, and operational considerations for conducting these trials in registration studies of pediatric investigational products. The goal is to promote efficiencies in FDA and EMA approval processes, including labeling. The workshop will include carefully structured small and large group discussions, exploring opportunities such as sharing control data, incorporating patient-centric approaches in the design phase, reducing participant enrollment numbers, and streamlining trial conduct.

Additional Workshop Objectives:

  • Understand how study design parameters—such as eligibility criteria, outcome measures, endpoints, data and information sharing, and the handling of company-confidential information—are managed and could be optimized.
  • Identify and develop approaches to address statistical, regulatory, and operational challenges associated with multi-sponsor pediatric platform trials.
  • Explore regulatory perspectives on the feasibility and acceptability of multi-sponsor pediatric platform trials.

Johnson & Johnson, AstraZeneca, and Sanofi have provided sponsorship grants for this independent program. Sanofi is part of the Steering Committee of the MRCT Center. Johnson & Johnson and AstraZeneca are part of the Executive Committee.

Venue and Travel

Book using your group rate for MRCT Center Pediatric Platform Block  

  • The Ven at Embassy Row, Washington DC, A Tribute Portfolio Hotel 
    2015 Massachusetts Avenue NW, Washington, DC 20036 

Room Locations at The Ven

Workshop materials and pre-reads

Platform Trials

MDR-TB


Hematologic Oncology


MDD

MDD: Pediatric Platform Design