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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)
Brigitte Bloechl-Daum
Vice Chair of the Department of Clinical Pharmacology , Medical University of Vienna / Department of Clinical Pharmacology, Austria
Orphans or Orphanisation?
Simon Day, PhD
Statistical Expert, Regulatory Advisory Board, NDA Group, United Kingdom
Standards of Evidence - From Blockbusters to Orphans
Ad Schuurman, MBA, MSc
Head of the Business Contact Centre & International Affairs, National Health Care Institute (ZIN), Netherlands
The End of the Orphan Drug Concept... What's Next?
Hans-Georg Eichler, MD, MSc
Consulting Physician, Austrian Association of Social Security Bodies, Austria
Panel discussion
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