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Global Rare Disease Town Hall
Session Chair(s)
James Valentine, JD, MHS
Director
Hyman, Phelps & McNamara, PC, United States
Rare disease drug development is exploding and, in light of both old and new incentives programs to encourage drug development, the challenges of bringing these products to market continue to persist. Exploring the scientific and regulatory issues with experts to provide insights from those with the most experience in both successful and failed rare disease drug proposals.This forum will address the unique regulatory complexities and challenges specific to orphan drug development. It will provide key information about programs available to expedite the development of orphan products and will include audience Q&A.
Learning Objective : Identify unique regulatory complexities and challenges specific to orphan drug development; Describe FDA programs available to expedite the development of orphan products; Recognize FDA requirements related to orphan drug regulation under 21st Century Cures and PDUFA VI.
Speaker(s)
EMA Perspective
Kristina Larsson, MS
European Medicines Agency, Netherlands
Head of Orphan Medicines, Division for Human Medicines Evidence Generation
Perspectives on Rare Diseases and Gene Therapies
Ilan Irony, MD
Janssen Pharmaceutical Companies of Johnson and Johnson, United States
Senior Director, Global Regulatory Leader
FDA Perspective
Lucas Kempf, MD
Parexel, United States
Vice President, RCS