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

Research output: Contribution to journalReview articleAcademicpeer-review

6 Citations (Scopus)
139 Downloads (Pure)

Abstract

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
Volume21
Issue number3
Early online date2 Jan 2023
DOIs
Publication statusPublished - 1 Mar 2023

Bibliographical note

Funding Information:
The SYMPHONY consortium, which aims to orchestrate personalized treatment in patients with bleeding disorders, is a unique collaboration between patients, health care professionals, and translational and fundamental researchers specialized in inherited bleeding disorders and experts from multiple disciplines. It aims to identify the best treatment choice for each individual based on bleeding phenotype. To achieve this goal, work packages (WP) have been organized according to three themes, eg, Diagnostics (WPs 3 and 4), Treatment (WPs 5-9), and Fundamental Research (WPs 10-12). 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. Principal investigator Dr M.H. Cnossen. Project manager: Dr S.H. Reitsma. More information: www.symphonyconsortium.nl. Beneficiaries of the SYMPHONY consortium: Erasmus MC and Erasmus MC Sophia Children's Hospital, University Medical Center Rotterdam, project leadership and coordination; Sanquin Diagnostics; Sanquin Research; Amsterdam University Medical Centers; University Medical Center Groningen; University Medical Center Utrecht; Leiden University Medical Center; Radboud University Medical Center; Netherlands Society of Hemophilia Patients (NVHP); Netherlands Society for Thrombosis and Hemostatis (NVTH); Bayer B.V. CSL Behring B.V. Swedish Orphan Biovitrum (Belgium) BVBA/SPRL. L.B. Rvd.G. A.L.B. and K.M. designed the study. L.B. drafted the manuscript. Rvd.G. A.L.B. and K.M. critically reviewed the manuscript and provided important intellectual inputs. E.Sv.H. provided important intellectual input to the section costs and access. L.B. and E.Sv.H. screened the articles of the initial systematic search. All authors reviewed and approved the final version. K.M. reports speaker fees from Alexion, Bayer, and CSL Behring; participation in trial steering committee for Bayer; participation in data monitoring and endpoint adjudication committee for Octapharma; all outside the scope of this review. She received consulting fees from Uniqure for participation in a writing committee for a gene therapy study, and she is investigator in a number of gene therapy trials. All fees are paid to her institution. All other authors have no conflicts of interest to declare.

Funding Information:
The SYMPHONY consortium, which aims to orchestrate personalized treatment in patients with bleeding disorders, is a unique collaboration between patients, health care professionals, and translational and fundamental researchers specialized in inherited bleeding disorders and experts from multiple disciplines. It aims to identify the best treatment choice for each individual based on bleeding phenotype. To achieve this goal, work packages (WP) have been organized according to three themes, eg, Diagnostics (WPs 3 and 4), Treatment (WPs 5-9), and Fundamental Research (WPs 10-12). 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. Principal investigator Dr M.H. Cnossen. Project manager: Dr S.H. Reitsma. More information: www.symphonyconsortium.nl . Beneficiaries of the SYMPHONY consortium: Erasmus MC and Erasmus MC Sophia Children’s Hospital, University Medical Center Rotterdam, project leadership and coordination; Sanquin Diagnostics; Sanquin Research; Amsterdam University Medical Centers; University Medical Center Groningen; University Medical Center Utrecht; Leiden University Medical Center; Radboud University Medical Center; Netherlands Society of Hemophilia Patients (NVHP); Netherlands Society for Thrombosis and Hemostatis (NVTH); Bayer B.V., CSL Behring B.V., Swedish Orphan Biovitrum (Belgium) BVBA/SPRL.

Publisher Copyright:
© 2023 The Authors

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