Around the Globe

Canadian Federal Consultations: National Strategy for Drugs for Rare Diseases
Judith Glennie
JL Glennie Consulting Inc.

n January 27, 2021, Health Canada launched a consultation on the development of a National Strategy for High-Cost Drugs for Rare Diseases. The consultations (open until March 26, 2021) are intended to engage Canadians and invite them to share their ideas and views on what a national strategy could look like. The foundation of the consultations is a discussion paper describing key considerations and questions for developing a national strategy for high-cost drugs for rare diseases. Feedback is being solicited through an online questionnaire, written submissions, and/or participation in virtual public town halls.


The February and June 2019 installments of Global Forum provided an overview of the history regarding Canadian policy developments related to drugs for rare diseases (DRDs). After years of discussion and pressure by provincial/territorial governments and patient advocacy groups, Canada’s federal government proposed the development of a Rare Disease Strategy in its March 2019 budget.

The budget contained little information as to the scope of the strategy, but the stated goal was to ensure that patients with rare diseases have access to effective, medically necessary therapies at an affordable cost. The federal government noted that it was looking to develop an approach to gather and evaluate evidence for DRDs, to improve the consistency of decision-making/access to these products across Canada, and to develop strategies for negotiating with manufacturers. They proposed an investment of up to $1 billion over two years (starting in 2022-23), with up to $500 million ongoing per year.

After the initial announcement, there were no further comments made regarding the strategy for well over a year. Then, in September 2020, the federal government reaffirmed its commitment to the strategy as part of the 2020 Fall Economic Statement and the Speech from the Throne. While it was heartening to see that the strategy was still in play, it was not until January 2021 that the potential scope of the strategy was revealed. This has led to the current consultation process.

Federal Consultations

Components of the Discussion Paper

The discussion paper is directed towards a wide range of stakeholders and, as such, provides a great deal of background information regarding what a rare disease is, the challenges of developing and funding DRDs, how DRDs are accessed and funding decisions made, and the rationale for a national strategy for high-cost drugs for rare diseases.

The paper outlines three key issues in creating a national strategy, options for addressing these issues, and then a series of questions related to these options. A general concluding question is also posed for stakeholders to address:

  • Do you have other ideas that might help increase access and lower costs for drugs for rare diseases?

The following summarizes each issue and their related considerations. Further details for each are available in the discussion paper.

1. How to improve patient access to high-cost drugs for rare diseases, and ensure access is consistent across the country.

Options for improving access and improving national consistency for people with rare diseases include:

  • A single framework for decision-making on high-cost drugs
  • A transparent coordinating body
  • Patient and clinician engagement
  • Coordinated support for research on rare diseases in Canada
Questions for Discussion

  1. How can access to high-cost drugs for rare diseases be made consistent to improve patient access to these treatments?
  2. Which of the proposed options, or combination of options, would be the most effective for improving access and improving consistency?
  3. Please explain the option(s) that you selected above.
2. How to ensure decisions on covering high-cost drugs for rare diseases are informed by the best evidence available.

Options for addressing the challenge of covering drugs with limited evidence include:

  • Innovative approval and coverage models
  • A national expert panel
  • A national data system
  • Independent national and international networks
Questions for Discussion

  1. How can decisions on covering high-cost drugs for rare diseases be made when the evidence is limited?
  2. Which of the proposed options, or combination of options, would be most effective for strengthening the evidence base?
  3. Please explain the option(s) that you selected above.
3. How to ensure spending on high-cost drugs for rare diseases does not put pressure on the sustainability of the Canadian health care system.

Options for controlling the impact of high-cost drugs on health system budgets include:

  • Sharing of costs and pooling of risks
  • Investments up front to reduce the risk in early development
  • Pay for performance
  • Supports for “made-in-Canada” innovation
  • International collaboration
Questions for Discussion

  1. Which of the proposed options, or combination of options, would be most effective for getting rare disease treatments to patients?
  2. Please explain the option(s) that you selected above.

Insights and Considerations

It is noteworthy that most of the options identified in the discussion paper relate to activities that are within the scope of the federal government’s role in the health and innovation ecosystem, and/or are activities over which the federal government has significant influence. For instance, regarding the latter, Health Canada is a major funding source for the Canadian Agency for Drugs and Technologies in Health (CADTH), which is the national health technology assessment agency for Canada (except Quebec). Many of the suggestions related to frameworks, coordinating bodies, and clinician and patient engagement (amongst others) are already within CADTH’s scope.

Several other options proposed would require significant collaboration and partnership with provincial/territorial governments, as they are the drug plan managers for DRDs funded by the public purse. Since private payers are also involved in DRD funding, they would also likely be part of initiatives related to innovative funding models.

If one reads between the lines of the discussion paper, it is also important to note what is not in scope for these consultations. While not explicitly stated, it is unlikely that the federal government has the development of a national drug program for DRDs in mind – at least not at this time. Based on the proposed budget for the Rare Disease Strategy (i.e., up to $500 million per year), there would not be enough money to fund a drug program for rare diseases at the current rate of expenditures and expected growth in this market segment. According to PMPRB estimates, Canadian 2019 DRD sales (cancer and non-cancer) per capita are $CAD68.2 (i.e., $CAD2.45 billion, assuming a total population of 36 million). Even if one were to assume that the program would only fund non-cancer DRDs (approximately one-third of this total), the funds available for the strategy would not be sufficient.


The road to a national DRD strategy has been long and arduous for Canadian citizens with rare diseases and their families. While the final scope of the federal government’s role in this area is yet to be finalized, it is heartening to see acknowledgement of the issues, openness to potential solutions, and concrete dialogue with stakeholders. But there are no simple solutions when it comes to matters of healthcare and its funding in the Canadian environment.

Two important factors that will determine whether this strategy comes to fruition. First, Canada currently has a minority government at the federal level, so the timing and outcome of the next federal election will be a key determinant in realizing the national strategy. Second, any policies that relate to healthcare need to be developed in partnership with the provinces, as the latter have responsibility for the funding and delivery of care. Moving forward with a national DRD strategy is important and hopefully something around which all stakeholders can align for the sake of patients and their families.