Like The Hub?
Join our community.

Durhane Wong-Rieger: Rare disease patients suffer while Canadian governments dither


Then-Federal Health Minister Jean-Yves Duclos in Montreal, March 22, 2023, speaking on the national strategy for drugs for rare diseases. Graham Hughes/The Canadian Press.

A commitment was made five years ago after an initial promise made 18 years ago. In February 2019, the federal government announced $1 billion to set up Canada’s Rare Disease Drug Strategy (CRDDS). In March 2023, the Canadian Organization for Rare Disorders (CORD) was proud to stand with the health minister when he announced $1.5 billion for Canada’s Rare Disease Drug Strategy with a spending plan of $1.4 billion for new treatments through provincial/territorial bilateral agreements and $100 million for federal drug plans, research, data collection, and evaluation initiatives.

Tragically, to date, no new drug funding has been made available; CORD has publicly denounced the fact that not a single penny has been spent to fund a single rare disease drug for a single patient!

To understand the magnitude of this delay, it is important to recognize that rare disease is a significant public health issue that directly impacts over 3 million Canadians (1 in 12). Each rare disease, by definition, affects a very small number of citizens.

Most rare diseases are severe, debilitating, and/or life-threatening. While about 70 percent affect children, there are a significant number of adult-onset rare conditions. Among the 7,000 known rare diseases, only 5 percent have an effective drug therapy. Many of these are the first approved therapy or a significant advancement over previous therapies, including some new therapies that could be considered a “cure” because they address the underlying cause of a disease.

it is all the more frustrating and unconscionable that only 60 percent of these therapies are approved here in Canada, due, in part, to Canada’s long, slow, multi-step, highly uncertain coverage process. Only about 25 percent of rare disease therapies approved by Health Canada and recommended by the Canada’s Drug Agency (CDA) and/or Institut national d’excellence en santé et services sociaux (INESSS) ever get to patients through the public drug plans. Rare disease patients with no access to effective therapy undergo suboptimal surgeries or rely on off-label drugs. Many experience avoidable decline in functionality, forcing them to drop out of school or leave their jobs; others suffer preventable life-threatening or life-ending events leading to disability and early death. It is very frustrating that there has been no visible government progress and transparency on the bilateral negotiations for the $1.4 billion in available funds.

Who are the patients in urgent need of approved therapies?

  • Patients with HoFH—a rare form of very high cholesterol that is ineffectively managed with statins and transfusion, leading to cardiovascular disease and premature death. An approved drug therapy significantly lowers cholesterol levels, reducing morbidity and mortality.
  • Children with NF1—large tumours growing next to the nerves that cannot be surgically removed. A targeted drug therapy can reduce the size and growth of tumours.
  • Patients with FOP whose muscles and tendons turn to bone, creating a second skeleton and restricting movement and breathing. A drug therapy researched in Canada reduces the risk of crises that lead to bone formation.
  • Patients with oHCN whose thickened heart muscles restrict blood flow leading to heart failure, stroke, arrhythmias, and sudden cardiac death. A novel therapy targets the underlying pathophysiology by reducing myocardial contractility and reducing risk of serious symptoms.
  • Adults with VHL with proliferating noncancerous tumours in kidney, brain, or pancreas that cannot be surgically removed. A drug therapy can reduce the size of tumour and avoid surgery.
  • Adults with SMA whose symptoms are later onset and thereby excluded from drug therapy, resulting in muscle weakness, fatigue, limited mobility, and difficulty breathing. An effective oral therapy has been approved but is not available to adults over the age of 25 despite demonstrated benefits and availability in other countries.
  • Adults with late-onset Pompé’s Disease who are denied access to treatment. An effective enzyme replacement therapy is approved but not reimbursed.

How to make the most of $1.5 billion? Invest it, one drug at a time

CORD, on behalf of the patient community, cannot wait for negotiations to deliver a master plan to spend $1.4 billion in bilateral agreements. We can and must act to make urgently needed drugs immediately available. CORD has proposed an implementation plan by which rare disease therapies that are approved and recommended can be started immediately with a defined cohort of patients along with a plan for ongoing monitoring, data collection, and assessment of benefit and potential harm.

CORD has convened a Rare Disease Drug Pathways(RDDP) working group, a multi-disciplinary multi-stakeholder group to inform the implementation of the CRDDS that will meet its stated goals and also assure timely, optimal, access to new, emerging, and “state of the art” therapies for rare disease patients The RDDP working group will run case studies and undertake analyses to develop recommendations on the criteria for the deployment of the CRDDS funding so that patients can receive funded access to new and emerging medicines for rare diseases.

Specific objectives

  • Assess and report on effective practices in Canada and other jurisdictions for the funding/reimbursement of rare diseases and other complex, innovative, or targeted therapies.
  • Develop a “flow chart” for the introduction, review, approval, and funding that minimizes the “time to access” and optimizes “availability to patients,” identifying barriers, challenges, and opportunities for improvement with all relevant parties, including Health Canada, CADTH/INESSS, pCPA, federal/provincial/territorial drug plans, private insurers, health system/health care providers, CIHI, industry, and patient organizations.
  • Select rare disease therapies awaiting patient access or requiring updated access that can serve as case studies toward developing consensus on access, management, and funding.
  • Develop consensus-based “strategic pathways” for accessing innovative, complex, and targeted (small population) therapies that address issues of urgent access, conditions for starting therapy, protocols to monitor and manage appropriate use, exceptional access programs, collecting data from clinicians and patients on safety and effectiveness in real-world settings, and adjusting individual use and treatment protocols based on evidentiary feedback, risk-sharing, and risk-mitigation protocols for all stakeholders, including patients, regulators, payers, and industry.

CORD invites all those interested in expediting access to effective therapies for rare disease patients to join us and support our efforts.

This article was made possible by the Canadian Life and Health Insurance Association and readers like you. 

Zachary Patterson: Universities are not going to reform themselves—so now what?


A pro-Palestinian encampment set up at the University of Toronto in Toronto, May 23, 2024. Christopher Katsarov/The Canadian Press.

My two previous columns indicated that both professors and universities are failing to fulfill their respective, unique, roles.

Instead of being producers of dependable, objective knowledge about the world we live in, they have become highly politicized and intent on remaking society according to their own values.

In so doing they have abandoned the foundational truth, knowledge, and merit principles of universities. Moreover, universities have abandoned their commitment to academic freedom.

The abandonment of academic freedom and these principles is not innocuous. The surprising antisemitic intensity and tenacity of support for Hamas on campuses is just the most recent example of what has been percolating in universities for decades if not generations.

Importantly, it is clear that the public does not support the radical ideas promulgated and emerging from campuses. At the same time, Canadian taxpayers lavish enormous resources on higher education. In fact, thirty percent more money goes to universities than to the military!

Some may argue that the excesses of universities are only temporary and that farsighted administrators will be reading the tea leaves and preparing the ground to right the ship. Others argue that universities must be the ones to lead reform.

Reform “from within” is unlikely however for four reasons. First, as found in a study with my colleague Christopher Dummitt, universities are overwhelmingly populated by Left-leaning academics and there is an increasing willingness to cancel dissenting views. Second, university administrators seem to be more liberal than already Left-leaning academics so it’s difficult to imagine reform coming from them.

Third, governing boards do not appear to be up to the task. These boards have either little actual power or are ignored by universities themselves. Moreover, they are more commonly populated by people with little desire to take on the universities.

These positions are typically voluntary and primarily considered to be honourary, despite their potentially important role in the governance of universities. Few members, even if they were sympathetic to reform, are likely to risk reputational damage by taking on radicalized universities, their professors, and their students.

Finally, many governing boards don’t even follow the most rudimentary rules of organizational governance. The most basic rule of independence of a board from universities is commonly ignored, with professors and even students being represented on them.

Professors and universities appear uninterested, unwilling, or incapable of reforming themselves. Governing boards appear to be in no better position to do the same. Since the public devotes enormous amounts of money to universities and doesn’t support the radical ideas coming from campuses, it is clear that their representatives not only have a right to seek to reform them but indeed an obligation.

The legal and funding landscape for universities in Canada

The question then becomes: “How can this much-needed reformation be accomplished?”

There are two starting points. The first is how we can learn from elsewhere. The second is to understand the university governance and funding landscape and what it implies for possible avenues for reform.

Luckily, serious efforts to reform universities are underway in both the U.K. and the U.S. Given the similar legislative and legal environment, the U.K.’s Higher Education (Freedom of Speech) Act 2023 is the most closely applicable to Canada’s universities.

This act was designed to protect and promote academic freedom in universities in the U.K. Importantly, in order to provide teeth to the act, it establishes an academic freedom “tsar” to monitor university policies as they affect academic freedom. The “tsar” will have the power to fine universities found to be in violation of the act.

While the U.K. started early in its moves to reform higher education, the U.S. has been the source of much more activity. Over twenty-five states, most famously Florida, have introduced or passed legislation, very often related to “Diversity, Equity and Inclusion” and thereby the merit principles, to this effect.

In addition to legislation that has been passed or introduced, there has also been a great deal of activity in proposing model legislation. For example, the National Association of Scholars has done a large amount of work on policy recommendations and stand-alone legislation. Similarly, the Manhattan and Goldwater Institutes have introduced model state legislation.

In addition to institutional contributions in this space, many public personalities have provided ideas for the reform of universities. These include Victor David Hanson, Chris Rufo, and Richard Hanania.

It’s beyond the scope of this column to provide a thorough description or analysis of all legislation, model legislation, and other avenues for reform. I mention them to highlight that whatever might be done in Canada, it’s not necessary to start from scratch.

Many people have put a lot of thought into these questions. Moreover, increasingly, we will be able to evaluate the degree to which different approaches have been successful and learn from experience elsewhere.

Avenues for reform are primarily a function of how universities are governed and funded. While the devil may be in the details, the broad brushstrokes are relatively straightforward.

Universities (and education more generally) are unambiguously provincial jurisdiction. Since almost all universities in Canada are public, they are ultimately public instances. They are governed through provincial power and legislation.

Universities are established through provincial legislation such as charters or acts. The relevant legislation then either directly or indirectly determines the governance of the universities. For example, they can delegate responsibility to a board of directors who oversee the management of a university, which is then governed according to university by-laws (e.g. University of Calgary).

University funding is less straightforward since it comes from multiple sources. Ultimately, university funding comes primarily from provincial and federal governments (directly and indirectly) and from students themselves in the form of tuition.

Given that universities are provincial jurisdiction, provinces provide the largest share of funding. This comes mostly in the form of operating funds.

Most provincial governments also provide research funding (e.g. Ontario Research Fund) that pays for equipment and other research costs but, importantly, also “overhead” funds provided to universities. Finally, provincial governments provide grants and student loans to students, which serve as direct or indirect funding for tuition.

While not in federal jurisdiction, the federal government devotes significant funds to universities through its “Tri-Council” agencies and the Canadian Foundation for Innovation with combined expenses of over $4 billion.

As with provincial governments, the federal government provides student loans that end up as tuition for universities. In addition, the federal and provincial governments extend tax benefits to universities (as well as to the tri-council agencies).

This overview of the governance and funding landscape explains the different means by which the public supports higher education in Canada. It also provides a framework to understand how they might be reformed. The concluding column in this series will lay out exactly how this might look.