Abstract
PURPOSE: As gene therapies for hemophilia are incorporated into clinical practice, shared decision-making (SDM) is recommended for implementation. SDM tools may facilitate informed decision-making for gene therapy and other novel therapies. OBJECTIVE: To inform the development of SDM tools for hemophilia gene therapy. PATIENTS AND METHODS: Men with severe hemophilia were recruited from the National Hemophilia Foundation (NHF) Community Voices in Research (CVR). Semi-structured interviews were completed, and the interviews were transcribed verbatim for quantitative and qualitative analysis. RESULTS: Twenty-five men with severe hemophilia A participated. All participants reported treatment with prophylaxis, nine (36%) on continuous prophylaxis with clotting factor, one (4%) on intermittent prophylaxis with clotting factor, and 15 (60%) on continuous prophylaxis with emicizumab. Ten (40%) indicated that they are excited about gene therapy, 12 (48%) indicated that they are hopeful about gene therapy, one (4%) indicated that they are worried or scared about gene therapy, and one (4%) indicated that they do not have strong feelings about gene therapy. Participants reported engaging Hemophilia Treatment Center, family, and the hemophilia community in their decision-making process. The most reported information needs are efficacy, safety, cost/insurance, mechanism of action, and follow-up. In addition, key information themes that emerged were patient testimonials, hard data and statistics, and comparison to other products. Twenty-two (88%) indicated that a SDM tool would be useful when discussing gene therapy with their hemophilia team. Two indicated that they do their own research, and the tool would not add anything. One needed more information to provide an answer. CONCLUSION: These data highlight the utility of a SDM tool for hemophilia gene therapy and key information needs. Data including comparison to other treatments should be provided along with patient testimonials in a transparent format. Patients will engage the Hemophilia Treatment Center, family, and community members in the decision-making process.