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