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Optimizing Clinical Trial Design and Execution for Rare Diseases

June 18, 2025
Breakout Session
Orphan and Rare Disease
Rare disease clinical trials face unique challenges, including small patient populations, high variability in disease presentation, and logistical complexities. This panel will explore innovative strategies to enhance trial design and execution, focusing on improving success rates and reducing investment risks. Experts will discuss adaptive trial designs, decentralized approaches, and real-world data integration. Attendees will gain insights into overcoming recruitment barriers, minimizing regulatory hurdles, and implementing cost-effective solutions to streamline the clinical trial process. The discussion will also include case studies and lessons learned from recent trials. By addressing these issues, this panel aims to equip stakeholders with actionable tools to enhance clinical trial outcomes and ensure sustainability in rare disease research.
Moderator
Marshall Summar, MD
CEO
Uncommon Cures
Speakers
Amy Comstock Rick, JD
Associate Director for Rare Disease Strategy, CDER
U.S. Food and Drug Administration
Emil Kakkis, MD and PhD
President and CEO
Ultragenyx
Janet Woodcock, MD
Former Principal Acting Commissioner
FDA

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