In this free webinar, learn about the rare disease landscape, including 2024 approval trends. Attendees will gain insights into the key regulatory guidances published within the past year and their impact on rare disease clinical research. The featured speakers will share study design strategies to ensure efficiency and compliance. Attendees will also hear a medic's perspective on what the changes will mean for patients and investigators.
TORONTO, Feb. 5, 2025 /PRNewswire-PRWeb/ -- Rare disease drug development is evolving, driven by innovation and regulatory advancements. With many devastating rare diseases still lacking approved treatments, regulatory agencies are taking significant steps to support innovators, advance research and improve patient outcomes. Through updated and newly developed guidances, they aim to provide a roadmap for navigating the complexities of this specialized field.
In this webinar, they will highlight key regulatory guidances published within the past year, addressing artificial intelligence (AI), accelerated approval pathways and fostering diversity, equity and inclusion (DEI) in clinical research. Attendees will gain insights into the challenges these guidances aim to address, their potential to transform the rare disease landscape, and strategies for incorporating any necessary changes into drug development programs.
Register for this webinar to explore how the rare disease landscape is changing with guidance from AI, accelerated approval pathways and fostering DEI in clinical research.
Join experts from , Adam Bloomfield, MD, FAAP, Vice President, General Medicine, Rare Disease and Pediatrics, Medical Affairs; Leslie Wetherell, Executive Director, Program Strategy, Rare Disease and Pediatrics; Paulla Dennis, Executive Director, Program Strategy, Rare Disease, Pediatrics, Cell and Gene Therapy, for the on Tuesday, February 25, 2025, at 11am EST (4pm GMT/UK).
For more information, or to register for this event, visit .
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