FAST represented the Angelman syndrome community this week in a discussion with senior leadership at the U.S. Food and Drug Administration focused on the future of rare disease research and development.
Ryan Fischer, Chief Operating Officer, and Jennifer Panagoulias, Head of Regulatory and Policy, participated on behalf of FAST in the FDA Rare Disease Roundtable. FAST was one of 15 advocacy organizations from across the rare disease community invited to join the discussion about the challenges and opportunities shaping the path from research to potential therapies.
FAST appreciates FDA’s engagement with rare disease patient organizations and the opportunity to ensure the Angelman syndrome community’s perspective was represented.
For Angelman syndrome, this conversation comes at an important time. As multiple therapeutic programs advance, the field is moving from scientific possibility toward the practical questions that shape development: how to define meaningful change, how to measure it reliably, and how to ensure patient and caregiver perspectives are reflected along the way.
Angelman syndrome and many other rare diseases present unique challenges, from small patient populations and complex natural history to limited outcome measures and clinical trial infrastructure needs. These realities make regulatory clarity, consistency, and early dialogue essential.
FAST’s participation reflects its ongoing commitment to ensuring that the patient and caregiver perspective help inform how progress is understood and measured. For the Angelman syndrome community, progress must be grounded in the realities of daily life, including communication, cognition, independence, daily functioning, and caregiver impact.
“We are at an important moment in Angelman syndrome therapeutic development,” said Ryan Fischer, Chief Operating Officer at FAST. “The science is advancing quickly, but progress depends on more than promising science. It also depends on whether we have the tools, measures, data, and development pathways needed to evaluate therapies in rare conditions where patient populations are small and the natural history is complex.”
FAST will continue working alongside a united Angelman syndrome community and the broader rare disease field to help turn scientific progress into meaningful advances for individuals living with Angelman syndrome and their families.