Berlin: Orphan Drugs Access And Economics Masterclass
“Rare disease therapies would be highly unattractive under standard market conditions from an investor perspective. The small number of patients cannot financially justify expending resources into orphan therapy research and development, which is a long, expensive, and risky process. As the population of patients with a specific rare disease is very small, the investments must be recouped by increasing the price of the product. It has been acknowledged that, while regulatory incentives have stimulated research and development of orphan therapies on a global level, equitable and timely access to approved orphan medicinal products for rare disease patients remains an issue. Decisions on Pricing and Reimbursement are the exclusive competence of the Member States of the European Union …”
• EU Regulation, Programs, & Mechanisms for Orphan Medicinal Products
• Achieving Sustainable Access For Orphan Drugs
• Methods for Economic Evaluation of Orphan Drugs
• Alternative Ways to Raise Funds & Finance Orphan Drugs
• Regulatory, Pricing & Reimbursement
• Patients & Payers
• Alternative Models: Drug Repurposing.
· Carlos R. Camozzi, Chief Medical Officer, Orphazyme
· Günter Harms, Market Access & Public Affairs Director, Shire
· Camille Métais, Associate Director – Regulatory Affairs, Alexion Pharma International
· Luis Cruz, General Manager Spain & Portugal, CSL Behring.
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