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

Track:
DIAmond Sessions

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

Session Number:
DMD6

Type:
Session

Title:
Evidence Generation in Medicines Development for Fragmented and 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 new innovative ways of evidence generation, such as single arm trials (Phase 1b), indirect comparisons (historic controls) and the impact of next generation sequencing on evidence generation. To discuss these complex topics, regulators, HTA bodies, patients and industry will review challenges and opportunities on the basis of case studies. 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