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Rare & Orphan Drug Development: Cost-Efficient Trial Design for Orphan Drugs to Minimize Cash Burn

Date | May 23, 2017 |
Time | 11:00 PM EDT |
Cost | Free |
Online
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Discussion topics include:
- Key challenges to improve cost-efficiency in orphan drugs clinical development
- Regulatory evolution in the review process of Orphan Drugs
- After examples like Sarepta's Exondys51 approval, how will the evaluation of clinical benefits change based on endpoints?
- What aspects of commercially planned clinical trials can be in conflict to minimizing cash-burn during orphan drug development? How do you plan for scale up while improving cost-efficiency?
- What role does multi-stakeholder collaboration in rare disease research play in improving clinical trials cost-efficiency?
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