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On January 27th, Health Canada announced that all Canadians, especially patients with rare diseases or interested stakeholders, will be invited to take place in a series of engagement activities to share their views on what a national rare disease drug strategy might look like. The claim is that feedback from this process will help inform the development of the emerging national strategy in Canada (see our post in this blog from September 1st, 2020 for more on that) and help ensure that patients receive faster and more effective access to the drugs available to help treat their rare diseases.

In order to help structure these discussions, Health Canada released a discussion paper that highlights some important background information on rare disease drug access in Canada and helps to better highlight the importance of developing a national drug strategy. According to the discussion paper “a national strategy on high-cost drugs for rare diseases, with agreed on priorities and processes, will help patients better access the drugs they need. By consolidating efforts, a strategy would also allow governments and funders to make more timely decisions about coverage, plan more effectively and ease the strain on public and private drug plans and the health care system across the country” (Health Canada, 2021, p. 8). In addition, the paper highlights that by developing a rare disease drug strategy, Health Canada would be better equipped to “...promote more cooperation among federal, provincial and territorial governments along the lines of the pan-Canadian Pharmaceutical Alliance’s work, to further reduce costs and increase access (Health Canada, 2021, p. 8). The hope is that the emerging strategy could improve consistency across the provinces and territories and that a “ common process for monitoring and assessing the real-world impact of new drugs” will ultimately emerge (Health Canada, 2021, p. 8)

The discussion paper then identified three key issues that Health Canada hopes to address through the development of a rare disease drug framework (Health Canada, 2021, p. 10). Interestingly, by structuring the discussion paper in this way, some stakeholders have noted that they feel that Health Canada is directing the discussion, and closing the opportunity for other focus areas that some patients and advocates may feel are also important.

Nevertheless, this discussion paper and the consultation provide a meaningful way for Canadians to engage with this new emerging piece of rare disease policy in the Canadian context. Canadians are encouraged to complete an online question, participate in one of six virtual public town halls, or send feedback by mail or email.

Elsewhere, we have advocated for an increased role of patients and the public in coverage decision-making for rare diseases (Douglas, Wilcox, Burgess and Lynd, 2015). As we pointed out there, patient and public involvement is not a panacea solution to the diverse challenges facing access and availability of rare disease treatments, and that consideration needs to be taken about the goals of the involvement activity when selecting which kind of form of involvement is being chosen and designed (see Table 1 from Douglas, Wilcox, Burgess, and Lynd, 2015 below)

While our work focused on coverage decision making specifically, when it comes to the engagements on the emerging national rare disease framework, it would most certainly seem that the goals here are developmental, thus the choice of a survey followed by a series of town hall meetings makes sense when trying to set some priorities. While this is not directly related to social pharmaceutical innovation, we can come to think of this as social pharmaceutical policy, in terms of alternative forms of engaging and developing policy in this area. Like all engagement activities, only time will tell what emerges from this approach as all too often engagements take place with little in the way of the marked difference in the policy or practice. That is one area that we will most certainly be keeping our eyes on.

All those engaged in the rare disease drug realm in Canada should consider participating in this discussion. To learn more and participate in the development of this national strategy, please visit:



Douglas, C. M., E., W., Burgess, M., & Lynd, L. D. (2015). Why orphan drug coverage reimbursement decision-making needs patient and public involvement. Health Policy, 119(5), 588-596.

Health Canada. (2021). Building a national strategy for high-cost drugs for rare diseases: A discussion paper for engaging Canadians. Government of Canada.


Written by: Shir Grunebaum (MA) and Conor Douglas (PhD) Please share your thoughts in the comment section below and check out our discussion forum as well:

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