Pan-Canadian Research Connect Community – Practical Considerations for Clinical Trials in Rare Disease Drug Development – Apr 9, 2025

Session Description

Pan-Canadian Research Connect Webinar

Rare disease clinical trials present unique challenges, requiring innovative solutions to ensure successful study design, regulatory compliance, and patient engagement. This session will provide an invaluable opportunity to explore these complexities with leading experts in the field.

Learning Objectives:

• Understand the Unique Challenges of Rare Disease Clinical Trials
  Identify key obstacles such as small patient populations, limited natural history data, and diagnostic delays.
• Describe Regulatory Frameworks and Incentives
  Explore the role of regulatory agencies (e.g., FDA, EMA) and incentives like Orphan Drug Designation in advancing rare disease research.
• Analyze Study Design Considerations
  Compare traditional and innovative trial designs (e.g., adaptive trials, basket trials, N-of-1 studies) and their applicability to rare disease research.

Our Distinguished Speaker & Moderator:

• Dr. Rob Freishtat, President & Co-Founder of Uncommon Cures, a leader in rare disease clinical research, biotechnology innovation, and clinical trial transformation.
• Dr. Stelios Georgiades, Director of the Offord Centre for Child Studies at McMaster University, renowned for his work in autism and neurodevelopmental research.

The webinar will offer an engaging, thought-provoking discussion on the evolving landscape of rare disease research and the strategies needed to overcome key challenges.