Webinar
Presented on: June 26, 2025
This on-demand webinar, presented on June 26, examines the FDA’s recommendations for long-term safety monitoring of gene therapy recipients—recognizing both the transformative benefits and potential health risks—and highlights the critical need to design follow-up studies around patient needs and preferences to minimize burdens. Building on prior discussions of platform trials and registries, this webinar emphasizes strategies to reduce burden and enhance participant-centered practices. Perspectives include insights from a foundation tackling patient loss to follow-up, expertise on embedding patient-centric culture change into study design, and a patient advocate’s lived experience emphasizing the power of patient voices in shaping compassionate, sustainable LTFU frameworks.