Background: Expensive drugs for rare diseases (i.e. orphan drugs) often do not meet traditional cost-effectiveness criteria and thus put further strain on limited healthcare budgets. Failing to provide medically necessary care to patients, however, violates one of the underlying tenets of most public health insurance systems-equity. This has led payers to consider the value that society places on the treatment of rare diseases, given the opportunity cost, when deciding on whether to fund specific treatments.
Aims: In this article we aim to illustrate two factors that make the measurement of societal value in this area particularly difficult: the low level of public awareness of, and engagement with, the orphan-drug issue, and the 'zero-sum' framing commonly used to describe the policy challenge posed by orphan drugs.
Method: We illustrate these challenges using data from an original survey of 2,005 Canadian adults. Respondents completed two tasks in which they were asked to choose between funding the treatment of patients suffering from either rare or common diseases.
Results: Respondents were more likely to display choice aversion and unstable preferences if they had not completed a university degree and when a 'zero-sum' frame was used to introduce the choice sets.
Conclusions: The results suggest that studies in which the stated opportunity costs of funding orphan drugs focus exclusively on reductions in funding for other drugs or treatments may only provide a limited understanding of citizens' policy preferences in the area of rare diseases.