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Assessing the Scope of Patient Engagement in Canada
Case Study: Research on Patient Engagement in Rare Disease Drug Policy

he Promoting Rare-Disease Innovations through Sustainable Mechanisms (PRISM) research initiative was established in 2011 with the goal of generating new information to support government decision-makers in the design of policies and processes to facilitate the development, introduction, and use of drugs for rare diseases (DRD) to benefit health outcomes and wiser use of Canadian healthcare resources.

The diversity, breadth, and multi-sectoral nature of PRISM’s scope of research has been unique. In particular, PRISM has been singular in its efforts to collaborate with and bring together a broad range of stakeholders from multiple sectors to discuss issues. This has allowed PRISM to examine a complex range of issues relevant to rare disease drug policy in Canada, bringing together and collaborating with a broad range of stakeholders in the process.

While the group has carried out research on a wide range of topics, PRISM’s approach to patient engagement research (i.e., Citizen’s Juries) has been unique and serves as a model for meaningful patient engagement. Overall, this work has demonstrated that there is significant potential value for decision-makers in involving patients in various policy decision-making activities as a potential means of reducing uncertainties that exist around DRDs.

For instance, in the context of systems for DRD funding decisions, Citizen’s Juries proved to be a practical means of understanding values that are important to consider in rare disease decision-making. According to PRISM’s research, while prevalence (i.e., “rarity”) matters, the severity of disease and degree of benefit achieved through treatment are also key factors in assessing a product for funding.

PRISM’s work has also examined the role of patients in the overall policy lifecycle of a DRD product. Research found that patient involvement in decision-making can provide decision-makers with important insights throughout the drug’s lifecycle, helping regulators reduce the uncertainties that exist around DRDs, as well as informing payers as they design and implement managed access programs.

Informed by these insights, the research team created a patient-directed policy framework to support development of policies that ensure the usefulness of patient input throughout the lifecycle of DRDs. This could be used by regulators to enhance their understanding of patient and caregiver willingness to accept potential harm (risk) in their treatment with new DRD products. It could also be used by drug plan managers considering managed access programs as a means of providing patients with access to new DRDs while maximizing data collection to support more definitive coverage decisions.