“In the past and, perhaps to some extent, today, the specialized care and medications required for the appropriate management of patients with rare diseases, such …
Spoiler Alert: World Orphan Drug Congress, Geneva 2013
The secret is out. The unrivaled networking event for orphan drug developers is once again taking place in Geneva on the 14th-15th November 2013. Whether …
Dr Janet Woodcock on the FDA’s new Breakthrough designation
Janet Woodcock, MD, Director of the FDA’s CDER drug division discusses the FDA’s new Breakthrough Designation for new drug approvals. Source: CurrentMedicine.TV
Dr. Richard Pazdur on the Breakthrough Designation Requirements
“Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, emphasizes the importance of adequate …
The Canadian orphan drug market – Eric Gervais of Medunik Canada
Eric Gervais, Executive Vice President of Medunik Canada, presented at World Orphan Drug Congress USA 2013 on the topic, ‘Myth and reality on the Canadian …
Martine Zimmerman of Alexion on Europe’s regulatory process
Martine Zimmermann, Executive Director Global Regulatory Affairs at Alexion Pharmaceuticals, presented at World Orphan Drug Congress USA 2013 on the topic, ‘Where industry stumbles in …
Identifying biomarkers to articulate the value of an orphan drug: a look into Vertex’s Kalydeco case – Charles Johnson, Vertex
Charles Johnson, VP of Global Medical Affairs at Vertex Pharmaceuticals, presented at World Orphan Drug Congress USA 2013 on the topic, ‘Identifying biomarkers to articulate …
Three Reasons Orphan Drugs Are Hot
“Dr. Coté is the leading national regulatory expert in orphan drug development. With 22 years of Federal service at the FDA, NIH, and CDC, Dr. …