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Session 6, Track A and B: Insights into Rare Diseases: Pioneering Pathways for Patient Access and Drug Development
Session Chair(s)
Brenda Gryfe, MSc
Regulatory Consultant
Flying Moose Technologies , Canada
Amber McLeod, PhD
Immunology, Virology, and Specialty Head, Regulatory Affairs
Abbvie Corporation, Canada
Alysha Croker, PhD
Director, Centre for Policy, Pediatrics and International Collaboration, BRDD
Health Canada, Canada
This session will discuss the challenges in rare disease drug development and provide updates on some exciting programs aimed at advancing therapeutic options for rare diseases. It will focus on Health Canada's National Strategy for Drugs for Rare Diseases, RareKids-CAN Pediatric Rare Disease Clinical Trials and Treatment Network, and the Critical Path Institute's Rare Disease Cures Accelerator - Data Analytics Program. These initiatives aim to increase accessibility and affordability of effective drugs, collect data to support better understanding of rare diseases, and provide drug development tools to address unmet needs in rare disease research.
Learning Objective : At the conclusion of this session, participants should be able to:- Describe the rare diseases space and how it has evolved in Canada over time
- Discuss Health Canada’s current Regulatory environment for rare diseases
- Identify opportunities to collaborate with various initiatives focused on supporting development of new drugs for rare diseases
Speaker(s)
Insights into Rare Diseases: Health Canada Perspective
Alysha Croker, PhD
Health Canada, Canada
Director, Centre for Policy, Pediatrics and International Collaboration, BRDD
Introduction to RareKids-CAN Pediatric Rare Disease Clinical Trials and Treatment Network
Thierry Lacaze-Masmonteil, MD, PhD, FRCPC
Maternal Infant Child and Youth Research Network (MICYRN), Canada
Scientific Director
Speaker
Breanne Stewart, BSN, RN
Maternal Infant Child and Youth Research Network, Canada
Network Director, RareKids-CAN
Critical Path Institute: Rare Disease Cures Accelerator-Data Analytics Platform (RDCA-DAP)
Collin Hovinga, PharmD, MS
Critical Path Institute, United States
Vice President Rare, Orphan Pediatric Diseases Programs
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