In November 2012 over 250 delegates participated in the 3rd Annual World Orphan Drug Congress Europe. This event covers strategies and opportunities from Clinical Development and R&D, Market Access, Pricing & Reimbursement , Corporate Development and Drug Repositioning & Indication Expansion. Attendees at this highly successful event were fortunate enough to hear this exciting presentation delivered by Peter Mueller, EVP R&D, CSO, Vertex Pharmaceuticals.
This presentation covers the following key points:
â¢ Valuable lessons learned during the approval of Kalydeco
â¢ How to prove safety and efficacy quickly and reach primary endpoints with small patient numbers?
â¢ What have been the challenges of conducting the clinical trial?
â¢ Paramount important of strong pre-clinical research in the rare disease space
Why not check out our eBook, showcasing the best presentations from the 2012 event. Download a copy here >
The 4th World Orphan Drugs Congress Europe is a content packed, 2 day event with 2 dedicated tracks and is taking place this year in November. For more information about the event, please visit the website >