| Summary: | Background: Expensive drugs for rare diseases pose a great challenge for decision-makers involved in determining their funding status due to their extremely high treatment costs, the small patient populations, the uncertainty of evidence for treatment effectiveness and a moral obligation to provide treatment where there is no alternative. The decision to fund a medication is traditionally and primarily guided by two factors: the drug’s cost and its effectiveness. This efficiency-based method is useful in guiding funding decisions for common medications, however is insufficient for rare diseases as they are unlikely to meet current cost-effectiveness thresholds, and moreover does not consider values associated with rarity including disease severity, unmet need and equity. Decision-makers are using values to determine rare disease funding status; however, it is unknown whether these decisions reflect societal priorities. Should society value rarity, this may provide decision-makers with the justification for the funding of expensive drugs which serve a disproportionately small population. Objectives: Identify the public values on health technologies, and the criteria society feels are most important for guiding priority-setting for health technology assessment and drug-reimbursement; Determine the relative importance of rarity; Determine whether society values rarity and accepts the opportunity costs associated with funding high-cost medications. Methods: We conducted an online survey of 2211 Canadians where multiple methodologies including simple ranking with an analytic hierarchy process, measures of attitudes towards equity statements, and trade-off questions were employed. Results: The values most important to Canadians for drug-reimbursement prioritization are drug safety, effect on quality and quantity of life, and certainty of efficacy. Factors related to rare diseases such as severity and equity are important factors to Canadians; however, rarity alone was of relatively low importance for the priority-setting of orphan drug treatment. Although society was not found to be willing to accept the opportunity cost for funding a rare disease if it is at the cost of a common condition, they prefer funding of a rare disease over a non-health benefit. Equity and equal access to drug treatments are important factors to Canadians, though rarity alone is not justification for prioritizing a drug treatment for funding.
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