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Evidence Generation in Medicines Development for Rare Patient Populations: Challenges and Opportunities

Track:
DIAmond Sessions

Day & Time:
April 18, 8:30AM - 10:00AM (Central Europe Standard Time)

Session Number:
DMD6

Room Number:
Sydney

Type:
Session

Title:
Evidence Generation in Medicines Development for Rare Patient Populations: Challenges and Opportunities

Chair(s):
Michelle Rohrer, PhD
Vice President, Global Head Regulatory Regions and Policy
Genentech, A Member of the Roche Group , United States

Tomas Salmonson, PhD
Chair CHMP; Senior Scientific Advisor
Medical Products Agency (MPA), Sweden

Description:
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.

Presentation(s) & Speaker(s):
Panel Discussion
Robert Hemmings, MS, MSc
Statistics and Pharmacokinetics Unit Manager
Medicines and Healthcare products Regulatory Agency (MHRA), United Kingdom

Panel Discussion
Niklas Hedberg, MPharm
Chief Pharmacist
Dental and Pharmaceutical Benefits Agency, TLV, Sweden

Panel Discussion
Dimitrios Athanasiou, MBA
Patient Advocate
WORLD DUCHENNE ORGANIZATION (WDO) , MDA HELLAS, Greece