title: Physician preferences for medication attributes for the prophylactic treatment of patients with severe haemophilia A with inhibitors to factor VIII.
abstract: Prophylaxis may be beneficial for patients with severe haemophilia A who have developed inhibitors to factor VIII. The aim of this study was to determine physicians' preferences for medication attributes in the prophylactic treatment of this patient population. Haematologists from Europe (EU) and the United States (US) participated in a discrete choice exercise to explore their preferences for medication attributes (efficacy, cost, scientific evidence, dosing frequency and administration time) associated with prophylaxis for severe haemophilia A in patients with inhibitors to factor VIII. Physicians' preferences for medication attributes were assessed through completion of 25 trade-off tasks that included a choice between two hypothetical medications each comprised of one randomized level of each medication attribute. Participants also completed a sociodemographic questionnaire. Data were analysed using a random effects logit model. Participants (N = 36: US = 19; EU = 17) were 80.6% men, had a mean of 19.8 years (SD ± 8.1) [range 6-35] of practice experience. The physicians treated an average of 5.7 (± 5.5) patients with severe haemophilia A and inhibitors per month and reported treating 36.2% of these patients prophylactically. The most important medication attributes for prophylactic treatment were efficacy [Relative Importance (RI) = 35.0%] and scientific evidence (RI = 34.1%), whereas treatment cost (12.0%), dosing frequency (10.8%) and administration time (8.2%) were less important. Results were similar across the EU and US. Efficacy and scientific evidence are the primary considerations for physicians' choice of prophylactic medications for use in this patient population.
authors.affiliation: United BioSource Corporation, Bethesda, MD, USA. heather.gelhorn@unitedbiosource.com
authors.forename: H
authors.initials: H
authors.lastname: Gelhorn
authors.forename: E
authors.initials: E
authors.lastname: Merikle
authors.forename: S
authors.initials: S
authors.lastname: Krishnan
authors.forename: L
authors.initials: L
authors.lastname: Nemes
authors.forename: C
authors.initials: C
authors.lastname: Leissinger
authors.forename: L
authors.initials: L
authors.lastname: Valentino
mesh_terms: D000293:Adolescent; D000328:Adult; D019774:Blood Coagulation Factor Inhibitors / Q000097:blood*; D002648:Child; D005060:Europe; D005169:Factor VIII / Q000037:antagonists & inhibitors / Q000627:therapeutic use*; D005260:Female; D006401:Hematologic Agents / Q000627:therapeutic use*; D006467:Hemophilia A / Q000097:blood / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D008297:Male; D010818:Practice Patterns, Physicians' / Q000706:statistics & numerical data*; D012044:Regression Analysis; D014481:United States; D055815:Young Adult
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D006401:Hematologic Agents; D005169:Factor VIII