Lessons Learned from our Roundtable with Rare Disease Advocates

https://www.fda.gov/news-events/fda-voices/lessons-learned-our-roundtable-rare-disease-advocates?utm_medium=email&utm_source=govdelivery Lessons Learned from our Roundtable with Rare Disease Advocates There is no substitute for sitting across the table from the people we serve. Recently we had the privilege of joining Acting Commissioner Kyle Diamantas, Acting Chief of Staff Lowell Zeta, and Deputy Commissioner Grace Graham for a roundtable discussion with 15 dedicated rare disease leaders. Hearing directly from patients and family members who navigate these incredibly challenging conditions every day was both grounding and motivating. The conversation reinforced our shared mission and highlighted several key areas where the U.S. Food and Drug Administration must continue to evolve to meet the unique needs of the rare disease community. During the discussion, we emphasized the FDA’s renewed unwavering commitment to the rare disease community. We made it clear that our medical product decisions must always be rooted in rigorous science and driven by the deep expertise of the FDA’s outstanding scientists and reviewers. While leadership sets the strategic vision, the real engine of this work is our incredibly hard-working career scientists across our medical product centers. Their dedication to evaluating complex data under intense timelines is what transforms scientific possibility into real-world hope for patients.