Achieving Meaningful Regulatory and Clinical Outcomes for Patients: Strategies in Rare Disease Therapy Development

A collaborative workshop designed to examine practical issues in the structuring of rare diseases therapeutic development programs.

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Case Study Presentation

Session Chair(s)

David H. Schubert

, United States

In this “mock” case study for the treatment of a rare autoimmune disease, we will explore many of the very real and challenging issues in a rare disease development program. We will refer to and interactively discuss this case throughout the workshop as experts and participants address:
• Complex designs of rare disease clinical trials
• Recruitment challenges
• Ways to effectively engage with Regulators
• Ways to involve patients and advocacy groups throughout the clinical development process
• Ways to engage “all” stakeholders to get drugs to patients faster
• Regulatory resources and programs available to facilitate development
• And more