Pediatric Rheumatic Diseases Drug Development: Challenges & Opportunities

Moderator(s)

• SA

  Stacy Ardoin, MD

  Nationwide Children's Hospital

Speaker(s)

• JA

  John Alexander, MD, MPH

  US Food and Drug Administration
• NN

  Nikolay Nikolov, MD

  FDA
• RR

  Rebecca Rothwell, PhD

  FDA
• 

  Carolyn Yancey, MD

  FDA

Clinical trials in pediatric rheumatic diseases present scientific and operational challenges. Yet, few opportunities exist to expose investigators to regulatory considerations around these complex pediatric research trials. The ACR, in collaboration with the Food and Drug Administration, present this session on regulatory and clinical trial design considerations in pediatric rheumatology. This interactive talk will address unique aspects of leveraging adult data and provide an overview of innovative trial design. The session will allow for an open dialogue between regulators and academic researchers to evaluate and discuss paths to advance the development of therapies for pediatric rheumatic diseases.

Learning Objectives:

• Describe the regulatory framework for the development of therapies for pediatric diseases
• Explain how innovative uses of methods such as extrapolation and modeling and simulation can leverage existing adult data and decrease the need for pediatric data to facilitate the study of therapies in pediatric populations
• Discuss the challenges with applying traditional trial design framework in pediatric settings
• Identify additional considerations specific to trial designs in the pediatric setting
• Prepare an overview of potential alternative designs and/or drug development programs, potentially with illustrative examples

Presentations:

• 12:00 PM – 12:20 PM ET

  Drug Development in Pediatrics: Regulatory Framework & Extrapolation of Adult Data
  Carolyn Yancey, MD – FDA
• 12:20 PM – 12:40 PM ET

  Trial Design Considerations in Pediatric Rheumatic Diseases
  Rebecca Rothwell, PhD – FDA
• 12:40 PM – 1:00 PM ET

  Q&A and Panel Discussion: Representation from FDA (Rheumatology, Pediatrics and Statistics) and ACR Membership
  John Alexander, MD, MPH – US Food and Drug Administration; Nikolay Nikolov, MD – FDA; Rebecca Rothwell, PhD – FDA; Carolyn Yancey, MD – FDA