February 25, 2025
11 a.m. EST
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, we’ll 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. You’ll 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 your drug development program.
Attendees will gain insights into:
- The rare disease landscape, including 2024 approval trends
- New guidances and their impact on rare disease clinical research
- Study design strategies to ensure efficiency and compliance
- A medic’s perspective on what the changes will mean for patients and investigators
Speakers:
Kenneth Ndugga-Kabuye, M.D., FACMG, Vice President, Cell & Gene Therapy
Paulla Dennis, Executive Director, Program Strategy, Rare Disease, Pediatrics, Cell and Gene Therapy
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