Long-term follow-up after authorization of gene therapy: leveraging real-world data

Abstract excerpt

• Gene therapy requires long-term follow-up (LTFU) studies. • We reviewed regulatory, scientific and operational requirements and considerations. • Assessing requirements and feasibility is essential before designing LTFU studies. • Studies should integrate the patient perspective, including retention in the study. • Identified challenges require harmonized guidance and multiparty collaboration. Gene therapies...

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