Supporting Patient-Centred Decisions on Gene Therapy for Haemophilia A: A Conceptual Model

The emergence of gene therapy for Haemophilia A (HA) represents a significant therapeutic milestone. By offering a potential one-time treatment that restores endogenous factor VIII production, gene therapy introduces new hope—yet also new complexities—for people with haemophilia (PWH) and their care teams. This is not a decision to be taken lightly. The long-term consequences, the absence of re-treatment possibilities and the evolving evidence base require a shared and carefully navigated path forward between clinicians and patients [1]. To support this process, we developed a conceptual shared decision-making (SDM) tool designed specifically for the Italian context. This tool was created by a multidisciplinary Expert Advisory Board comprising haematologists fromdifferent regions of Italy, a psychologist, a linguist, a methodology expert and representatives of national patient organizations. The group brought together diverse scientific, clinical and experiential perspectives to design a flexible framework capable of fostering dialogue, reflection and informed choice in the context of gene therapy.