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Rare Diseases

Engaging publics on reimbursement decision-making for expensive drugs for rare diseases

Current health technology assessment emphasize allocative efficiency, under which few of expensive drugs for rare diseases (EDRDs) would be reimbursed by provincial government drug plans. Decision makers are faced with the high costs and uncertainty of evidence for treatment effectiveness versus a perceived obligation to provide treatment where there is no good alternative. As innovation and the number of available drug treatments increase, there will be a greater need to balance high costs within a constrained budget and with societal needs, preferences and values. The collaborating knowledge users are key actors in the formation of recommendations of whether to fund drugs through the Common Drug Review and BC provincial ministry. They are interested incorporating public values and input. The research question is: How do respondents to an online public engagement assess these priorities and alternative scenarios related to funding expensive drugs for rare diseases?

A ten-minute, cross-Canada survey was developed to further explore and to assess regional variations in societal values and attitudes associated with reimbursement decision-making for EDRDs. The survey used a dynamic, interactive online platform – Metroquest – as a novel approach to engaging publics on this topic. Results will be used to understand how societal values might be incorporated into and impact reimbursement decision-making processes for EDRDs and to help inform future public engagement activities.