Estimates of utility weights in hemophilia: implications for cost-utility analysis of clotting factor prophylaxis

Expert Rev Pharmacoecon Outcomes Res. 2015 Apr;15(2):267-83. doi: 10.1586/14737167.2015.1001372. Epub 2015 Jan 14.

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.

Keywords: health utilities; hemophilia; prophylaxis; quality of life; quality-adjusted life-years.

Publication types

  • Review

MeSH terms

  • Adult
  • Blood Coagulation Factors / economics
  • Blood Coagulation Factors / therapeutic use*
  • Cost-Benefit Analysis
  • Delivery of Health Care / economics
  • Hemophilia A / drug therapy*
  • Hemophilia A / economics
  • Humans
  • Quality of Life
  • Quality-Adjusted Life Years*
  • Selection Bias

Substances

  • Blood Coagulation Factors