Download presentation- Cystic Fibrosis: How to develop powerful clinical data for a small population subset?

download, cystic fibrosis, world orphan drug congress

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:

• Valuable lessons learned during the approval of Kalydeco
• How to prove safety and efficacy quickly and reach primary endpoints with small patient numbers?
• What have been the challenges of conducting the clinical trial?
• Paramount important of strong pre-clinical research in the rare disease space

Download the full presentation here >

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 >

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