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Canadian Response to the Online Engagement National Strategy for High-Cost Drugs for Rare Diseases

Our February 8th this blog informed our readers the Health Canada would be conducting an engagement activity ( on the emerging National Strategy for High-Cost Drugs.

After reviewing the discussion paper, and participating in some of the town hall activities members of the Canadian team of the SPIN project, alongside other academics in Canada, submitted the following as a response to that engagement activity.


To the members of the Office of Pharmaceuticals Management Strategies at Health Canada, We are writing as a group of Canadian academics who have collectively been undertaking research on policy challenges facing the rare disease community. We feel it is our duty as academics and as Canadians to provide our insights on the discussion paper “Building a National Strategy for High-Cost Drugs for Rare Diseases: A Discussion Paper for Engaging Canadians” (2021) to engage with all rare disease stakeholders in order to improve outcomes for rare disease patients and all Canadians. We write to raise some critical issues that we believe are either absent or underdeveloped in that discussion paper.

We generally found that the many initiatives/options/approaches included in the discussion paper were not “either/or” options, but rather “all of the above” approaches. For instance, in terms of “options” for addressing the challenge of covering drugs with limited evidence, we see the necessity in all of the options you provided. That is, an innovative approval and coverage models to get treatments to patients without treatment options quicker, as well as a national expert panel to decide which patients would be eligible for one of such models and the kind of data needed, a national data system to collect that real-world evidence to subsequently evaluate the managed access agreement, and independent national and international networks to pool and compare various national data sets on similar conditions or interventions.

Next to this, we would like to stress that we fully acknowledge the challenges that rare disease patients and their families face, and we wish to see associated policy in this area that will work to serve them as a healthcare population while also supporting the long-term healthcare system sustainability across the country.

Our comments fall into four broad categories that call for the National Strategy to:

1. Go beyond high-cost drugs and attend more holistically to screening, care, nonpharmaceutical treatment, and support of caregivers;

2. Focus on the entire pharmaceutical ecosystem rather than primarily on prices;

3. Increase funding for Canadian public pharmaceutical and biotech research and development;

4. Continue engagement and involvement activities that bring patients and other members of the Canadian public into the discussion and decision-making process around drugs for rare diseases.

We will address each of these briefly in turn.

Going beyond high-cost drugs

As has been noted by many participants at Health Canada’s virtual consultations (which many of us have also attended), the framing of this National Strategy as being about “high-cost drugs” in part misses the mark. We trust that the government has come to recognize how exclusionary this framing is and will re-focus the National Strategy so that it is more holistic and attends to “drugs, therapies and non-pharmaceutical interventions for rare diseases”. The National Strategy must not simply accept that all drugs for rare diseases will be high cost. A national approach needs to explore the reason(s) for any high costs and also include a plan to seek cost reductions, as is currently being attempted through changes to PMPRB guidelines.

Given the often-low regional prevalence of people with any given rare disease, one of the goals of this National Strategy must be to help move the provinces, territories, and federal government towards a national rare disease strategy around which they can all come together. Problems of health equity abound in the rare disease space, particularly in the context of the “postal code lottery” and the varying levels of access that different families affected by the same rare disease(s) have to treatments, care, and support services across Canada. We therefore strongly recommend that a fundamental goal of this National Strategy be to eliminate inconsistencies in availability and access to “drugs, therapies and non-pharmaceutical interventions for rare diseases” across the country. Furthermore, the strategy must not overlook the importance of strengthening caregiver support, which is fundamental to improving access in a meaningful way.

Attending to the entire pharmaceutical ecosystem

The National Strategy must position itself in such a way that the entire pharmaceutical ecosystem is within its purview. Public investment in the early stages of research often leads to innovators selling their discoveries to private pharmaceutical companies. These private companies are not required to disclose their development costs and are then able to set those often high-profit margins that this National Strategy is aimed at finding ways to avoid paying into. Public investment into R&D must therefore extend beyond the science and attend to what happens after therapies with potential are discovered. It must focus on incentivizing R&D, supporting commercializing, and generally helping to keep costs low so that the entire pharmaceutical ecosystem can benefit over time. The drug Glybera is of course a prime example of how the pipeline problem has played out in Canada in the past.

Building Canadian not-for-profit pharmaceutical and bio-tech capabilities

The continuing devastation wrought by the COVID-19 pandemic has served as a global reminder that pharmaceutical innovation plays an important role in helping governments create resilient economies. At the same point in time, we have also been reminded of the need to have local R&D and manufacturing capacities to bring treatments made in Canada to Canadians. Building on the need for a National Strategy that seeks to improve access to therapies and help reduce high drug costs, meaningful public investment must be made into pharmaceutical research and development - particularly not-for-profit partnership ventures. In the context of this National Strategy, specific programs must be created to support research into the mechanisms of rare diseases and to help researchers move potential therapeutics forward through the R&D pipeline. A number of excellent examples of this already exist in Canada, such as Canada’s Immunotherapy Network (BioCanRx ) that is tackling rare as well as more common forms of cancers, as well as the M4K Pharma ( ) that is taking an “open science” approach to rare pediatric conditions. Alternative drug development models stand to address some of the challenges related to drugs for rare diseases (including high costs), but support for more of these types of initiatives is needed through the emerging National Strategy.

Reframing the narrative of “cheap and unsympathetic government” through continued patient and public engagement and involvement

We acknowledge the efforts of Health Canada to organize numerous town hall meetings, as well as the online engagement through which we are submitting this letter. This is an important step towards greater patient and public engagement and involvement on the issue of pharmaceutical governance in general, as well as the pressing issues facing rare diseases in particular. We have previously advocated for different forms of patient and public involvement in decision-making processes related to the coverage of drugs for rare diseases (Douglas et al. 2015). While these kinds of initiatives need to be developed, we believe that engagement and involvement activities must be integrated throughout the drug research and development life-cycle (this connects to our previous point regarding made-in Canada not-for-profit pharmaceutical R&D). Not only does this have a positive effect for identifying appropriate targets and outcomes of interventions, collecting natural history data, participating in research, collecting outcome data and real-world evidence, but a larger role for patients in the emerging National Strategy stands to reframe the antagonistic discourse currently pervasive in the Canadian debates on rare diseases. All too often patient complaints regarding a coverage decision, and the associated media coverage, pit patients against governments, Ministries of Health, and other public institutions. In many ways, patients have been mobilized against public institutions, which are being framed as the depriver of life-saving treatment rather than the provider of health services. This framing and media coverage leads to political pressure that can cloud sound health policy decisions. It is well known that the pharmaceutical industry has entire divisions devoted to patient relations, which extend beyond research. Significant efforts are needed to change the narrative of the role of public institutions in the rare disease space. Bringing patients into decision-making processes in meaningful ways can help to achieve this important outcome. Patients need real input in all facets of the R&D life-cycle for there to be any chance of re-orientating the relationship. At the same time, a worst-case scenario exists in which Canadians with needs other than rare diseases begin to turn their backs on patients who are seen to be receiving an unequal share of public resources. Consequently, Canadians at large must also be involved in a systematic, long-term, and institutionalized manner. Considerable expertise exists in Canada for establishing these kinds of involvement mechanisms, and numerous models exist internationally for institutionalized involvement (i.e. patient and public involvement policies at NICE). Of the billion dollars available in the emerging National Strategy, relatively modest expenditures for public and patient involvement activities stand to have a significant impact on interest alignment and reframing the current narrative.

Finally, it is incumbent on us to note the ahistorical nature of this discussion paper. Numerous initiatives related to rare diseases and pharmaceuticals more broadly have started and stopped over the years, all of which are totally absent from this document. While we fully realize the need for brevity in such a discussion paper, we also recognise the need to locate discussions in social and historical context so that the lessons of earlier consultations, initiatives, reports, and policy options are not lost and forgotten. We hope that firm political leadership will be taken on this issue and that the federal, provincial, and territorial governments alike can capitalise on the opportunities that lie ahead with the budget commitments to address some of the significant challenges facing rare disease patients and their communities.

In kind regards,

Conor Douglas, PhD Assistant Professor, York University

Ian Stedman, PhD Assistant Professor, York University

Carlos Novas, PhD Associate Professor, Carleton University

Maz Riverin, PhD Postdoctoral Fellow, Princess Margaret Cancer Centre References



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

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