Development of a shared decision-making tool for gene therapy in hemophilia A in Italy

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Background: Gene therapy offers a transformative treatment option for hemophilia A (HA) by restoring endogenous factor VIII production. Shared decision-making (SDM) is a collaborative process that allows patients and clinicians to weigh the risks and benefits of complex treatment options such as gene therapy. However, no SDM tools exist for the specific needs of Italian persons with HA. Materials and Methods: An Expert Advisory Board comprising nine experts (including hematologists, a psychologist, a methodology expert, a linguist and patient advocacy group representatives) met three times between March and July 2024 to discuss gene therapy for HA in Italy, and develop an SDM tool to determine the suitability of gene therapy for HA. Results: The resulting modular SDM tool allows for structured interactions between clinicians and patients, while remaining sensitive to each patient’s level of readiness and understanding. The tool includes three components: Role Definition, Agenda Alignment, and Intention Setting, with supporting materials to guide patient-clinician dialogue and various resources (questionnaires, brochures, and visual aids). Conclusions: Despite requiring confirmation in further studies, this SDM tool may represent a useful component of patient-centered care for HA in the Italian healthcare setting.

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