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Rare Diseases

Informing Future Orphan Drug Coverage Using Scenario Studies (iFOCUSS)

It has been estimated that approximately 1 in 12 Canadians suffer from 1 of over 7,000 different rare diseases. Orphan drugs used in the treatment of rare diseases routinely cost more than $500,000 per year. In Canada, many patients’ medicines are covered by a private health insurance payer, or in the absence of one, a provincial health program. However, for a growing sub-set of patients with rare conditions orphan drugs are not always an insured benefit primarily due to financial constraints. Typically provincial Health Ministries use cost-effectiveness to inform their coverage decisions; however, if those methods were strictly applied, then almost no orphan drugs would be covered and many patients would go untreated.

How then should decision-makers decide on which orphan drugs to cover in a future that projects more Canadians with rare diseases and an increasing number of expensive drugs? To assess if the current systems and decision- making processes are ensuring sustainable access to orphan drugs, our research will explore how such systems and processes will respond to current trends and future challenges relating to medicine, biotechnology, and economic funding arrangements. We are undertaking a research project that uses scenario study methods to help decision-makers to explore these uncertain futures, and to use those explorations to collectively draft policy recommendations for orphan drugs that are sustainable, fair, transparent and future orientated. Instead of simply looking at the current state of a technology, we will develop scenarios in the form of stories, narratives, or news items of the future and deploy them in a focus group setting to engage stakeholders in thinking about how current technological and social contexts will unfold to influence the trajectory of orphan drugs. By exploring how decision-makers respond to diverse future scenarios we will be able to highlight best practices for coverage adjudication going forward.