June 2-3, 2025 in Washington, DC
This conference explores cutting-edge methodologies and regulatory advancements in rare disease research. Discussions will highlight innovative study designs, including adaptive, platform, and hybrid trials, as well as novel strategies to address challenges in small patient populations and global trial participation. Experts will share real-world case studies on alternative data sources for regulatory decision-making, such as patient-centric data, AI-driven drug discover, and the use of natural history and real-world data. Additionally, sessions will focus on collaborative research networks and strategies to accelerate rare disease drug development. Attendees will gain valuable insights into the future of clinical research, regulatory innovation, and data-driven decision-making in rare diseases.
Do you know of someone leading innovative work in rare disease research that is poised to accelerate progress in drug development? Please share your speaker ideas with us for one or more topics below!
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