Building Canada’s Smart Rare Drug Strategy: Meeting Patient Needs
Drugs for rare diseases did not fit the traditional R&D paradigm and, hence, many were“orphaned” until the US FDA Orphan Drug Act and subsequent EMA Orphan Drug Legislation unlocked them. Regulatory approval processes also evolved to accommodate clinical trial designs specific to small patient populations, resulting in over 600 approved orphan products. Guidelines for health technology assessment were not designed for many of the highly innovative therapies, including drugs for rare diseases and gene therapies.
Webinar Agenda
Canada’s Rare Disease Drug Strategy Event Outline
Patients, patient groups, students and academic researchers registration is complimentary.