title: Estimates of utility weights in hemophilia: implications for cost-utility analysis of clotting factor prophylaxis.
abstract: Estimates of preference-weighted health outcomes or health state utilities are needed to assess improvements in health in terms of quality-adjusted life-years. Gains in quality-adjusted life-years are used to assess the cost-effectiveness of prophylactic use of clotting factor compared with on-demand treatment among people with hemophilia, a congenital bleeding disorder. Published estimates of health utilities for people with hemophilia vary, contributing to uncertainty in the estimates of cost-effectiveness of prophylaxis. Challenges in estimating utility weights for the purpose of evaluating hemophilia treatment include selection bias in observational data, difficulty in adjusting for predictors of health-related quality of life and lack of preference-based data comparing adults with lifetime or primary prophylaxis versus no prophylaxis living within the same country and healthcare system.
authors.affiliation: National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, 1600 Clifton Rd. NE, Mail Stop E-64, Atlanta, GA 30333, USA.
authors.forename: Scott D
authors.initials: SD
authors.lastname: Grosse
authors.forename: Shraddha S
authors.initials: SS
authors.lastname: Chaugule
authors.forename: Joel W
authors.initials: JW
authors.lastname: Hay
mesh_terms: D000328:Adult; D001779:Blood Coagulation Factors / Q000191:economics / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D003695:Delivery of Health Care / Q000191:economics; D006467:Hemophilia A / Q000188:drug therapy* / Q000191:economics; D006801:Humans; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D015983:Selection Bias
chemical_list: D001779:Blood Coagulation Factors
keywords: health utilities; hemophilia; prophylaxis; quality of life; quality-adjusted life-years