At the 2025 World Orphan Drug Congress USA, Katie McCarthy, SVP Clinical and Regulatory Strategy Consulting, had the privilege of moderating a distinguished panel of experts to discuss “The Future of Drug Development in Rare and Orphan Diseases: Navigating Regulatory Challenges and Clinical Innovation.” This panel brought together industry leaders, including Matt Trudeau (President of ITF Therapeutics), Bridgit DeCoursey Bondoc (Partner at Morrison Foerster), and Samantha Zappia (SK Regulatory Solutions), to explore the current landscape of drug development for rare and orphan diseases. The panel delved into the evolving regulatory environment, innovative clinical trial designs, and strategic approaches to advance drug development in these critical areas. Here are ten key takeaways from our insightful discussion:
1. Optimism Reigns Amidst Regulatory Complexity
Despite the evolving regulatory landscape, particularly in the United States, there is continued optimism about the future of drug development for rare and orphan diseases. Creativity, collaboration, and proactive communication with regulatory authorities are essential for navigating the regulatory process effectively. While there have been unsettled nerves regarding the recent changes at the FDA
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