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Evidence Generation in Medicines Development for Rare Patient Populations: Challenges and Opportunities
Session Chair(s)
Michelle Rohrer, PhD
Senior Vice President, Global Head of Product Development Regulatory and Policy
Roche, United States
Tomas Salmonson, DrMed, PhD
Partner
Critical Path Institute, United States
This session will focus on challenges and opportunities on pre- and post-authorisation evidence generation in rare patient populations, as identified by patients, regulators, HTA bodies and industry. The discussion will center around the following topics: choice of control, endpoints (biomarkers, clinical outcomes, PROs), therapeutic indication, post authorisation evidence generation/ registries. Following a brief description of key challenges in four topic rounds the panel will bring in perspectives based on concrete cases and examples and discuss on opportunities and solutions. The session will touch but not focus on real world data and as such complement other sessions on that theme.
Speaker(s)
Panel Discussion
Robert Hemmings, MSc
Critical Path Institute, United States
Partner
Panel Discussion
Niklas Hedberg, MPharm
Dental and Pharmaceutical Benefits Agency, TLV, Sweden
HTACG Co-Chair & Chief Pharmacist
Panel Discussion
Dimitrios Athanasiou, MBA
WORLD DUCHENNE ORGANIZATION (WDO) , MDA HELLAS, Greece
Patient Advocate
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