Evaluating the impact of moral arguments on societal values for orphan drug coverage

As a member of the CIHR New Emerging Team for Rare Diseases, Nick is exploring citizens’ attitudes and behaviour as they relate to the challenge of providing accessible treatments for patients suffering from rare diseases. The development of accessible orphan drugs poses a dual problem. On the one hand, the small potential market for orphan drugs for rare diseases makes it difficult for many innovators to access sufficient private capital to develop new orphan drugs, resulting in a lack of treatment options for many rare disease patients. Nick is exploring how patient groups and the general public may be able to address this funding gap. Specifically, he is investigating the extent to which crowdfunding – an emerging approach to online fundraising – might be used to help “de-risk” early-stage orphan drug research by allowing investigators to fund exploratory studies that make it possible for them to attract subsequent grant funding or private investment.

On the other hand, provincial health insurance plans are faced with a decision-making challenge due to the fact that orphan drugs for rare diseases are often very expensive. This poses a moral dilemma, since the same funds used to treat one individual with a rare disease could provide less costly medications for many more Canadians. Because taxpayers must ultimately bear the cost of funding these treatments through the public insurance system, empirically estimating society’s willingness to pay for expensive treatments for rare diseases, despite the significant opportunity costs, is a key element of democratic decision-making. Using data from an online survey of adult Canadians, Nick is exploring societal preferences for orphan drug funding by analyzing respondents’ choices in different scenarios in which provincial health authorities must decide whether or not to fund a specific treatment through the provincial insurance system.