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US and EU Orphan Drug Regulations: Shaping the Ecosystem for Rare Diseases
Session Chair(s)
Imran Shah, PHD
SVP & Head, Global Regulatory, R&D Quality and Safety (RQS)
EMD Serono, United States
The US Orphan Drug Act and the European regulatory framework have accelerated orphan medicine development, boosting investments in rare disease treatments. This session will gather key stakeholders to discuss successes a and challenges
Learning Objective : Discuss the impact of regulatory policies on the rare disease ecosystem; Discuss the challenges and opportunities associated with orphan drug regulations; Examine the role of regulatory frameworks in fostering innovation and access to therapies for rare diseases; Explain the importance of collaboration between regulatory agencies, industry stakeholders, and patient advocacy groups.
Speaker(s)
Panelist
Amy Comstock Rick, JD
FDA, United States
Director of Strategic Coalitions, Rare Disease Innovation Hub
US and EU Orphan Drug Regulations: Shaping the Ecosystem for Rare Diseases
Anabela Marcal, PHARMD
European Medicines Agency, Netherlands
EMA Liaison Official to the US FDA
Panelist
Segundo Mariz, MD
European Medicines Agency, Netherlands
Panelist
Victoria Gemme, MBA, MS
National Organization for Rare Disorders, United States
Director, Policy and Regulatory Affairs
Panelist
Carolyn Shore, PHD
National Academies, United States
Director
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