OBJECTIVES

Patient and caregiver preferences for varying hemophilia A treatment characteristics relevant to their decision-making can be assessed via discrete choice experiment (DCE) techniques. Identifying these characteristics and their levels is a critical step in DCE design. Herein, we report on the identification of key characteristics and levels to be used in a large quantitative DCE study, combining literature review and qualitative research.

METHODS

A literature review was conducted to identify treatment characteristics reported in previous patient and caregiver preference studies in hemophilia. Based on literature review findings and expert opinions, a short list of characteristics was selected for in-depth, cognitive interviews with hemophilia A patients and caregivers for further evaluation. Patients’ and caregivers’ comprehensibility of characteristic descriptions were assessed, followed by rating of importance in treatment decision-making (0 = “not important” to 10 = “very important”) and ranking (1 = “most important” to 6 = “least important”) of relative importance.

RESULTS

A total of 18 treatment characteristics were identified in the literature review. Based on discussions with clinical experts, the following 6 characteristics were included for subsequent qualitative research: bleed protection, joint health, safety, dosing, real-world evidence, and product type (factor and non-factor therapy). Patient and caregiver cognitive interviews showed that all characteristics were rated ≥ 6, suggesting overall high importance in treatment decision-making. The top 3 ranked attributes were bleed protection (average rank: 1.4), safety (2.9), and joint health (3.5).

CONCLUSIONS

The current literature review and qualitative research identified 6 characteristics important for hemophilia A patients’ and caregivers’ treatment decision-making, with bleed protection, safety, and joint health as the most important characteristics. A quantitative DCE study among hemophilia A patients and caregivers (N = ~250) is currently ongoing to further assess how the respondents value the relative importance of these 6 characteristics, when considering hemophilia A treatments.