To assess patient preferences for benefits and risks in hemophilia A treatment.
A systematic literature search and pretest interviews were conducted to determine the most patient-relevant endpoints in terms of effects, risks, and administration of hemophilia A treatments. A Best–Worst Scaling (BWS; Case 3 or multiprofile case) approach was applied in a structured questionnaire. Patients were surveyed by interviewers in a computer-assisted personal interview. Treatments in the choice scenarios comprised bleeding frequency per year, application type, risk of thromboembolic event risk, and inhibitor development. Each respondent answered 13 choice tasks, including 1 dominant task, comparing 3 treatment profiles. Data were analyzed using a mixed logit model (random-parameters logit).
Data from 57 patients were used. The attributes “bleeding frequency per year” and “inhibitor development” had the greatest impact on respondents’ choice decisions. Patients disliked being at risk of inhibitor development more than being at risk of thromboembolic events. The type of application, whether intravenous or subcutaneous, was of less importance for patients. There was a significant preference variation for all attributes.
Patients value low frequency of bleeding per year and low risk of development of inhibitors the most. An increase of risk and frequency would significantly decrease the impact on choice decisions. The type of application does not seem to influence the choice decision very much compared with the other attributes. Regarding preference heterogeneity, further analysis is needed to identify subgroups among patients and their characteristics. This may help to adapt individually patient-tailored treatment alternatives for hemophilia A patients.
Axel C. Mühlbacher Andrew Sadler Björn Lamprecht Christin Juhnke