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Session 7, Track A: Navigating the Novel: Multi-faceted Challenges in Rare Disease Treatment in Canada
Session Chair(s)
Amber McLeod, PhD
Immunology, Virology, and Specialty Head, Regulatory Affairs
Abbvie Corporation, Canada
My Dang, MBA
Director, Regulatory Affairs
Cencora, Canada
This session will explore regulatory challenges in developing treatments for rare diseases, emphasizing pathways and expert advice to navigate these hurdles. Attendees will recognize diverse challenges associated with first-in-class treatments, analyze the impacts of small patient populations and novel manufacturing on submission strategies, and evaluate the benefits of early scientific engagement and interagency collaboration. Panelists will address unique challenges, share case studies, discuss evidence generation for approval, and highlight the importance of characterization and scientific exchange.
- When Every Patient Counts: Regulatory Challenges in Rare Diseases: A Sponsor’s Perspective
Annie Bergevin, Novartis Pharmaceuticals Canada - Health Canada’s Approach for Rare Disease Drug Review
Bassam Haidar, Health Canada - Challenges in Rare Disease Treatment in Canada: Development and Access
Mauricio Ede, Incyte Corporation
Learning Objective :
At the conclusion of this session, participants should be able to:
- Identify and explore the unique challenges in developing rare disease drugs where guidelines may not fully apply
- Evaluate innovative evidence generation and approval strategies for rare drug therapies
- Appreciate the role of collaboration in regulatory processes to harmonize expectations for advanced therapies
Speaker(s)
Health Canada’s Approach for Rare Disease Drug Review
Bassam Haidar, PhD
Health Canada, Canada
Manager, Metabolic and Musculoskeletal Drugs Division, Pharmaceutical Drugs Dire
When Every Patient Counts: Regulatory Challenges in Rare Diseases: A Sponsor’s Perspective
Annie Bergevin, MSc
Novartis Pharmaceuticals Canada Inc., Canada
Regulatory Affairs Franchise Lead
Challenges in Rare Disease Treatment in Canada: Development and Access
Mauricio Ede, MD, PhD
Incyte Biosciences Canada, Canada
Head of Medical Affairs
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