戻る Agenda
Session 2A Rare Disease/Small Population
Session Chair(s)
Lutz Harnisch, MD
Senior Director, Global Clinical Pharmacology/Pharmacometrics
Pfizer, United Kingdom
The session will highlight EMA’s current approach on the development and approval of medicines for rare diseases/small populations, specifically how this approach translates into a development programme not only supporting the Health Authorities’ approval, but also the acceptance by payers and HTA bodies. What all that means in practice will be illustrated by two recent cases, which not only managed the Regulatory approval, but also successfully went through the HTA assessment, leading to ultimate availability of those new treatment options to children, their parents, and their doctors. Learnings from the two case studies and the experience from the audience will be discussed to better understand what is needed to improve the development and availability of new, innovative medicines for children.
Speaker(s)
Laura Fregonese, MD, PHD, MSC
European Medicines Agency, Netherlands
Scientific Officer
Rare disease/Small population
Stephanie Rosenfeld, DrMed, MD
Sanofi-Aventis Deutschland GmbH, Germany
Director, Evidence Based Medicine/ Health Economics and Outcomes Research
Dunja Pfeiffer
Pierre Fabre Pharma Gmbh, Germany
Head Market Access DACH