The ethics of gene therapy for hemophilia: a narrative review

Lieke Baas, Rieke van der Graaf, Evelien S van Hoorn, Annelien L Bredenoord, Karina Meijer, SYMPHONY consortium

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Gene therapy is expected to become a promising treatment, and potentially even a cure, for hemophilia. After several years of research, the first gene therapy product has been granted conditional market authorization by the European Union in August 2022. The recent progress in the field also has implications on the ethical aspects of hemophilia gene therapy. Reviews conducted in the 2000s mainly identified questions on the ethics of conducting early-phase clinical trials. However, since then, the knowledge on safety and efficacy has improved, and the field has moved toward clinical application, a phase that has its own ethical aspects. Therefore, we conducted a narrative review to take stock of the ethical aspects of hemophilia gene therapy. Based on our analysis of the literature, we identified 3 ethical themes. The theme Living up to expectations describes the existing hopes for gene therapy and the unlikelihood of the currently approved product becoming a permanent cure. In the theme Psychosocial impacts, we discuss the fear that gene therapy will impact the identity of people with hemophilia and their need for psychosocial support. The theme Costs and access discusses the expected cost-effectiveness of gene therapy and its implications on accessibility worldwide. We conclude that it may be necessary to change the narratives surrounding gene therapy, from describing it as a cure to describing it as one of the many treatments that temporarily relieve symptoms and that there is a need to reevaluate the desirability of gene therapy for hemophilia, given the availability of other treatments.

Original languageEnglish
Pages (from-to)413-420
Number of pages8
JournalJournal of Thrombosis and Haemostasis
Issue number3
Early online date2 Jan 2023
Publication statusPublished - 1 Mar 2023

Bibliographical note

.This research received funding from the Netherlands Organization for Scientific Research
(NWO) in the framework of the NWA-ORC call agreement NWA.1160.18.038.

Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.


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