Long-term follow-up after authorization of gene therapy: leveraging real-world data
Publication date
2025-05
Authors
de Haart, Karin
Asao, Keiko
Ataher, Quazi
Geier, Jamie
Hillen, Jodie
Huang, Kui
Mol, Peter G M
Rivera, Donna
Wang, Huifen
Yang, Hong
Editors
Advisors
Supervisors
Document Type
Article
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License
cc_by
Abstract
Gene therapies are innovative treatments that could revolutionize healthcare. Given their unique mechanisms of action, methods of manufacturing and administration, and the potential for long-lasting impact, gene therapies require long-term follow-up (LTFU) of safety and effectiveness post authorization, often using real-world data (RWD). With over 2000 gene therapies in development, there is a need for knowledge-building regarding LTFU studies. Here, we review regulatory, scientific, and operational requirements and considerations for LTFU studies using RWD from a global perspective. The insights in this review support post-authorization LTFU studies for gene therapies, thereby contributing to their safe and effective use.
Keywords
effectiveness, efficacy, gene therapy, long-term follow-up, pharmacoepidemiology, real-world data, safety
Citation
de Haart, K, Asao, K, Ataher, Q, Geier, J, Hillen, J, Huang, K, Mol, P G M, Rivera, D, Wang, H, Yang, H, Zhan, S, Hoogendoorn, W E & Bloem, L T 2025, 'Long-term follow-up after authorization of gene therapy : leveraging real-world data', Drug Discovery Today, vol. 30, no. 5, 104337. https://doi.org/10.1016/j.drudis.2025.104337