OBJECTIVES: Prophylaxis with factor replacement (Factor VIII or Factor IX) is the current standard of care for people with haemophilia (PwH). With the development of gene therapies for PwH, research is needed to inform the design of a discrete choice experiment (DCE) and to examine the preferences of PwH for different treatment characteristics.

METHODS: Semi-structured interviews were conducted with patient representatives (N=14) and clinical experts (N=6) including haematologists (n=2), nurse specialists (n=2), a psychology specialist, and a physiotherapist in the UK. Interviewees answered open-ended questions and were asked to rank, by importance, a predefined list of 18 treatment attributes with inclusion of any additional treatment attributes not mentioned on the predefined list. Interviews were transcribed and then coded by three researchers, with each transcript double-coded to ensure quality of the analysis of data. A data matrix was developed, and themes were generated from the interview data. The importance of treatment characteristics was prioritised by assigning points based on ranking (rank of 1 = 6 points, decreasing to 1 point for a rank of 6 and no points allocated to a rank of ≥7). This system allowed an attribute to have maximum of 120 points.

RESULTS: Impact on factor level (73 points), uncertainty regarding long-term risks (52 points), impact on daily life (40 points), frequency of monitoring (29 points), and impact on ability to participate in physical activity (26 points) were ranked as the most important treatment attributes. The key themes identified included “the effect of gene therapy on daily life” and the importance of “informed decision making.”

CONCLUSIONS: Our research indicated that treatment benefits, quality-of-life impact, and treatment risks are important factors to inform treatment selection for PwH in the UK. Further research in larger patient cohorts is needed to understand the relative importance for each haemophilia treatment attribute.