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Rising to the Challenge of Developing Novel Orphan Medicines for the Global Market
Session Chair(s)
Cecil J. Nick, MS
FTOPRA, Vice President (Technical)
Parexel Consulting, United Kingdom
One in ten people could be affected by a rare disease; for many there is no treatment, yet modern technology could resolve this. This session investigates the opportunities available to facilitate successful global development of novel orphan medicines.
Learning Objective : Integrate global regulatory considerations to the development of orphan medicines; Describe the challenges in the clinical development of orphan medicines; Apply knowledge of incentives available in the United States, Europe and Japan to maximize successful development of orphan medicines.
Speaker(s)
Panelist
Audrey Gordon, JD
Progeria Research Foundation, United States
President, Executive Director
Rare and Orphaned Disease Drug Products: Convergence of Scientific and Regulatory Paths Leading to Successful Approval
John Ziegler, MD
Premier Research, United States
Medical Director
European Perspective
Spiros Vamvakas, MD
European Medicines Agency, Netherlands
Scientific Advior on Human Medicines
Regulatory Perspective
Gayatri R. Rao, JD, MD
Rocket Pharmaceuticals, Inc., United States
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