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

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

doi:10.1016/j.drudis.2025.104337

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

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^*

^*Corresponding author for this work

Research output: Contribution to journal › Review article › peer-review

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.

Original language	English
Article number	104337
Journal	Drug Discovery Today
Volume	30
Issue number	5
Early online date	19 Mar 2025
DOIs	https://doi.org/10.1016/j.drudis.2025.104337
Publication status	Published - May 2025

Bibliographical note

Keywords

gene therapy
long-term follow-up
safety
efficacy
effectiveness
real-world data
pharmacoepidemiology

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10.1016/j.drudis.2025.104337Licence: CC BY

1-s2.0-S1359644625000509-mainFinal published version, 599 KBLicence: CC BY

Cite this

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title = "Long-term follow-up after authorization of gene therapy: leveraging real-world data",

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.",

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author = "{de Haart}, Karin and Keiko Asao and Quazi Ataher and Jamie Geier and Jodie Hillen and Kui Huang and Mol, {Peter G M} and Donna Rivera and Huifen Wang and Hong Yang and Siyan Zhan and Hoogendoorn, {Wilhelmina E} and Bloem, {Lourens T}",

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doi = "10.1016/j.drudis.2025.104337",

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journal = "Drug Discovery Today",

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AU - de Haart, Karin

AU - Asao, Keiko

AU - Ataher, Quazi

AU - Geier, Jamie

AU - Hillen, Jodie

AU - Huang, Kui

AU - Mol, Peter G M

AU - Rivera, Donna

AU - Wang, Huifen

AU - Yang, Hong

AU - Zhan, Siyan

AU - Hoogendoorn, Wilhelmina E

AU - Bloem, Lourens T

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N2 - 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.

AB - 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.

KW - gene therapy

KW - long-term follow-up

KW - safety

KW - efficacy

KW - effectiveness

KW - real-world data

KW - pharmacoepidemiology

U2 - 10.1016/j.drudis.2025.104337

DO - 10.1016/j.drudis.2025.104337

M3 - Review article

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JO - Drug Discovery Today

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ER -

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

Abstract

Bibliographical note

Keywords

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