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

MessageThis Webinar is over
Date May 23, 2017
Time 11:00 PM EDT
Cost Free
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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