Ethical aspects of hemophilia gene therapy: a qualitative interview study with stakeholders

Abstract

Background

There are great expectations for the potential role of gene therapy in the treatment of hemophilia. At the same time, developments in the field of hemophilia gene therapy have always raised ethical issues. It remains unknown how these ethical issues are perceived by stakeholders, particularly regarding the most recent developments in the field.

Objectives

To obtain insight into stakeholders’ morally reasoned opinions on gene therapy for hemophilia.

Methods

We conducted qualitative research with Dutch people with hemophilia ( n = 13), parents of children with hemophilia ( n = 5), physicians ( n = 4), nurses ( n = 3), a regulator ( n = 1), and a representative from a pharmaceutical company ( n = 1). We conducted semistructured interviews based on a topic list and reported the results according to the Consolidated Criteria for Reporting Qualitative Research guidelines.

Results

We identified 3 main themes. The theme freedom and independence describes the hope people with hemophilia have of increasing their freedom through gene therapy, as well as concerns that gene therapy increases their dependence on their treatment center. The theme trust and altruism describes how people with hemophilia have a high level of trust in their physician and treatment center as well as in scientific research. As a result of this trust, they are willing to participate in research to help other people with hemophilia. The theme incremental benefits describes doubts respondents have about the added value of gene therapy compared to standard treatment.

Conclusion

Stakeholders embrace the theoretical potential of gene therapy, while several people with hemophilia question the added value of the current gene transfer products for themselves.

Essentials

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  It is unknown how stakeholders perceive the ethical aspects hemophilia gene therapy.

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  We conducted an interview study with Dutch stakeholders to identify ethically relevant themes.

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  Freedom and independence, trust and altruism, and incremental benefits are important ethical themes.

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  People with hemophilia hope to enhance their autonomy, but it is unclear if gene therapy will enable this.