OBJECTIVES : In Canada, current standard-of-care treatments for hemophilia A include on-demand treatment of bleeds as they occur, with/without regular intravenous prophylactic infusions. With the introduction of recently approved treatment options, including subcutaneous therapies such as emicizumab, understanding societal perspectives regarding treatment attributes is warranted. In this study, the objective was to elicit preferences for features of hemophilia A treatments amongst adults living in Canada.

METHODS : A discrete choice experiment (DCE) was conducted. Attributes were informed by qualitative interviews in n=10 individuals with hemophilia A and/or caregivers and included the following features: frequency of prophylaxis (including on-demand only option), subcutaneous vs. intravenous administration, efficacy (bleeds per year), and risk of complications. Participants were shown infographics describing disease and attribute features and independently completed 10 choice scenarios. Results were analyzed with conditional logit regression, overall, and stratified by treatment experience.

RESULTS : The DCE was completed by n=219 participants (mean age 44.7 years; 50.9% female). Thirteen percent of participants had experience with intravenous infusions, 10% with subcutaneous injections, and 6% with both. In the overall sample, there was a significant preference for treatments with fewer bleeds, lower risk of treatment complications, less frequent injections, subcutaneous treatments, and prophylaxis over on-demand. The odds ratio for preferring a subcutaneous prophylaxis vs. on-demand was 4.11 (p<0.001), and 3.61 (p=0.006) for intravenous prophylaxis vs. on-demand. Exploratory post-hoc analyses suggested that preference for subcutaneous vs. intravenous may be more pronounced in subgroups with prior real-world experience with intravenous or subcutaneous therapies; however, differences were not statistically significant.

CONCLUSIONS : In Canadian general population respondents, preference was expressed for hemophilia A treatments having favorable efficacy and safety profiles, subcutaneous treatment, and less-frequent prophylaxis. Preference for subcutaneous over intravenous treatment modality warrants further investigation, and potential exploration among a patient, rather than general population, sample.