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Where is the Orphan Drug Journey Going?
Session Chair(s)
Mark Rutter
Co-Founder, Head of Product Development & Strategy, RxTROSPECT, United States
This session looks at the success of EC Regulation on orphan medicinal products in bringing innovative new therapies to patients with high unmet medical need. We will explore the upcoming changes in the EU orphan environment and what must be maintained and built upon to further stimulate research in this key research area. Session in development
Speaker(s)
Agnes Mathieu-Mendes, RPh
Head of Unit, European Health & Digital Executive Agency (HaDEA), Belgium
Optimisation of the Regulatory Framework for Orphan Medicines to Encourage Innovative Treatments for Patients
Jordi Llinares Garcia, MS
Head of Research and Innovation, European Medicines Agency, Netherlands
Orphan Medicinal Products and the Major Contribution to Patient Care
Virginie Hivert, PharmD, PhD
Therapeutic Development Director, Eurordis-Rare Diseases Europe, France
The Patient’s Perspectives on OMPs and Significant Benefit
Mark Rutter
Co-Founder, Head of Product Development & Strategy, RxTROSPECT, United States
Evolution of the Significant Benefit Framework - An Industry Perspective
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