Karin de Haart, Keiko Asao, Quazi Ataher, Jamie Geier, Jodie Hillen, Kui Huang, Peter G M Mol, Donna Rivera, Huifen Wang, Hong Yang, Siyan Zhan, Wilhelmina E Hoogendoorn, Lourens T Bloem
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引用次数: 0
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.
期刊介绍：
Drug Discovery Today delivers informed and highly current reviews for the discovery community. The magazine addresses not only the rapid scientific developments in drug discovery associated technologies but also the management, commercial and regulatory issues that increasingly play a part in how R&D is planned, structured and executed.
Features include comment by international experts, news and analysis of important developments, reviews of key scientific and strategic issues, overviews of recent progress in specific therapeutic areas and conference reports.
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