A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study

Health Econ Policy Law. 2011 Jul;6(3):405-33. doi: 10.1017/S1744133110000344. Epub 2010 Dec 21.

Abstract

Policy debate about funding criteria for drugs used to treat rare, orphan diseases is gaining prominence. This study presents evidence from a discrete choice experiment using a convenience sample of university students to investigate individual preferences regarding public funding for drugs used to treat rare diseases and common diseases. This pilot study finds that: other things equal, the respondents do not prefer to have the government spend more for drugs used to treat rare diseases; that respondents are not willing to pay more per life year gained for a rare disease than a common disease; and that respondents weigh relevant attributes of the coverage decisions (e.g. costs, disease severity and treatment effectiveness) similarly for both rare and common diseases. The results confirm the importance of severity and treatment effectiveness in preferences for public funding. Although this is the first study of its kind, the results send a cautionary message regarding the special treatment of orphan drugs in coverage decision-making.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biomedical Research
  • Choice Behavior*
  • Cost-Benefit Analysis
  • Decision Making
  • Female
  • Health Care Costs
  • Health Expenditures / statistics & numerical data*
  • Health Policy / economics*
  • Humans
  • Male
  • Models, Econometric
  • Multivariate Analysis
  • Orphan Drug Production / economics*
  • Pilot Projects
  • Prescription Drugs / economics*
  • Rare Diseases / drug therapy
  • Rare Diseases / economics*
  • Students
  • United States
  • Universities
  • Young Adult

Substances

  • Prescription Drugs