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Cutting Blockbuster Indications into Orphan-Sized Bites
Session Chair(s)
Brigitte Bloechl-Daum
Vice Chair of the Department of Clinical Pharmacology
Medical University of Vienna / Department of Clinical Pharmacology, Austria
With the fragmentation of treatment-eligible populations into ever smaller substrata, targeted therapies present new challenges including evaluation of non-RCT data from small populations, regulatory consequences regarding orphan status, issues of pricing and reimbursement and off- (or near-) label use.
Speaker(s)
Orphans or Orphanisation?
Brigitte Bloechl-Daum
Medical University of Vienna / Department of Clinical Pharmacology, Austria
Vice Chair of the Department of Clinical Pharmacology
Standards of Evidence - From Blockbusters to Orphans
Simon Day, PhD
NDA Group, United Kingdom
Statistical Expert, Regulatory Advisory Board
The End of the Orphan Drug Concept... What's Next?
Ad Schuurman, MBA, MSc
National Health Care Institute (ZIN), Netherlands
Head of the Business Contact Centre & International Affairs
Panel discussion
Hans-Georg Eichler, MD, MSc
Austrian Association of Social Security Bodies, Austria
Consulting Physician
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