Preferences towards the characteristics of coagulation factor concentrates used to treat hemophilic patients with inhibitors : the patients', physicians' and pharmacists' perspective

Background: Management of hemophilia with inhibitors is particularly complex and costly. Although modern technologies are shown to be highly effective, no agreement exists yet on how to optimally treat these patients. Treatment is the result of interactions between patients/caregivers, physicians, pharmacists, budget holders. Aim: To evaluate preferences toward characteristics of coagulation factor concentrates for inhibitors patients from a sample of patients/caregivers, haematologists, pharmacists with experience in storing/delivering coagulation factor concentrates. Methods: A Discrete Choice study was conducted. Possible products were described with 7 previously selected characteristics: viral safety, risk of anamnestic response, possibility of major surgery, frequency of infusions in prophylaxis, number of injections to stop bleeding, time to stop bleeding, time to pain recovery, cost (expressed as increase of health care taxes), added to estimated willingness to pay. The respondents were asked to choose among 16 sets, each one describing 2 hypothetical products. Data were analysed with a logistic model. Results: Thirty seven patients/caregivers, 39 physicians, 25 pharmacists were interviewed. All the patients had severe hemophilia A, historical median peak titre = 164 (5–16,400) BU mL-1. Among all the respondents, the hypothesized direction of preferences was confirmed (e.g. lower number of injections preferred to higher number), every attribute was considered important (P < 0.01). However, each subgroup expressed different strengths of preferences for some attributes. Conclusion: Decisions on products (e.g. NovoSeven vs. Feiba) and regimen treatments (e.g. prophylaxis vs. on-demand) must take into account clinical needs of patients. Understanding the preferences assigned to different option characteristics can help to optimize the overall benefits of treatments