DIA 2013 49th Annual Meeting: Advancing Therapeutic Innovation and Regulatory Science

戻る Agenda

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