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.