First Gene Therapy Approved by Health Canada

On December 16th, 2020, Health Canada announced that it would approve its first-ever gene in Canada - Zolgensma for spinal muscular atrophy (SMA). This is a significant development for a number of reasons. First, as a gene therapy Zolgensma offers curative potential, and as such is a windfall for SMA patients (most of who are children) and their families. Spinal muscular atrophy is a motor neuron disease which affects muscles throughout the body (Lunn & Wang, 2008, 2120) with individuals with often experiencing weakness in the legs or arms, and less commonly, individuals may experience difficulty feeding or swallowing (Lunn & Wang, 2008, 2120). In some children, it also affects the respiratory muscles and can lead to an increased tendency for pneumonia and other lung problems (Lunn & Wang, 2008, 2120). According to CURE SMA Canada, approximately 1 in 6000 babies in Canada are affected by spinal muscular atrophy and 1 in 40 people are genetic carriers of this condition (Cure SMA Canada, 2017). The way it works (not scientifically speaking) is that it is a one-time single-dose intravenous injection that can actually cure SMA, not simply treat its symptoms (Dangouloff & Servais, 2019, 1154). Unlike the other major treatment for SMA (nusinersen aka Spinraza), Zolgensma can also be given to adults who would not be able to receive the nusinersen injection due to spinal fusion, a common condition with spinal muscular atrophy (Dangouloff & Servais, 2019, 1154).

It is important to remind our international readers that Health Canada approval does NOT necessarily mean that patients across Canada will be receiving the therapy. What this recent approval signifies is that Zolgensma has been deemed safe and effective enough to be sold in Canada. It is now up to the thirteen individual provinces and territories who are responsible for the organization and delivery of their own provincial and territorial health care system (including drugs and treatments) as to whether they will make this groundbreaking therapy available - and why wouldn’t they?! Well, there are 2.8 million reasons as to why they might at least think twice about it. Zolgensma this is set to be the most expensive ‘drug’ in the world (Dyer, 2020, 580) -although it's not really a drug it's more of a treatment- with an estimated price tag of $2.8 million dollars (CDN) per patient (Black, 2020). However, this treatment is incredibly expensive and this has posed a significant challenge for governments throughout the world (Black, 2020).

The cost is one issue; however, researchers are still not adequately able to determine if this treatment can continue to work in the long-term. It is that uncertainty, in combination with incredibly expensive treatments such as this, that is calling for the Canadian government and the pharma companies to develop (better) risk-sharing mechanisms. Risk-sharing agreements are defined as those in which the “risk is shared by both the payer, which agrees to pay reimbursement costs for the product despite uncertainties about its clinical or health economic value, and the drug manufacturer, which typically agrees to make financial or outcome-related concessions depending on the future performance of the product” (Bastian et al., 2016, 46). These are pre-arranged contracts which rely on performance or financial measures that are tracked over periods of time in a specific population (Bastian et al., 2016, 47), and also represent one of the kinds of social pharmaceutical innovation we are interested in, in terms of alternative regulatory frameworks for coverage. Even before the approval of Zolgensma in the United States in May, there was a significant push for Novartis to explore innovative reimbursement models, including payments that were to be spread out over time and risk-sharing agreements (Weintraub, 2019). Risk-sharing agreements have increasingly become more common for high-cost treatments (Bastian et al., 2016, 44), and while risk-sharing agreements are more rare in Canada and the United States, health authorities in the United Kingdom and Italy have engaged in these types of agreements for many years, yielding both positive and negative outcomes (Bastian et al., 2016, 47). In fact, in a presentation given by CADTH in 2019, CADTH urgently called for the Canadian government to work with the industry “to establish a national risk-sharing models for rare disease and high cost drugs to ensure that all regions in Canada can afford to offer the minimum standard” (CADTH, 2019).

Perhaps these types of agreements will be part of the solution to Canada’s continued difficulty in establishing a national rare disease coverage policy. But are such agreements considered innovative? Or are they simply another financial tool harnessed by pharmaceutical companies? It will, therefore, be very interesting to see how things proceed with Zolgensma in Canada. It will be something that we will watch very closely, as this represents an opportunity for the kind of social pharmaceutical innovation we are trying to understand in the project.

Whichever way it goes, it will surely be better than what has been happening thus far with this treatment. In January of 2020, Novartis, which is a Swiss company that developed Zolgensma, began accepting applications for patients to be awarded this treatment via a lottery system (Forani, 2020). Novartis announced that it would be selecting a new name every two weeks to receive the therapy and that they would distribute 100 free doses worldwide (Forani, 2020). This lottery, which appeared to some as a charitable venture, was highly condemned by health ministers and patients groups throughout the world (Dyer, 2020, 580). SMA, a patient group in Europe, even released a statement stating that they were “alarmed that the programme will make thousands of babies [with spinal muscular atrophy] compete with each other for a lifesaving treatment, splitting tight knit communities” (Dyer, 2020, 580). Health ministers throughout Europe echoed these concerns, indicating that this lottery “proves no sincere commitment to patients and only increases the distress of families concerned. They are given false hope” (Dyer, 2020, 580). Furthermore, this type of lottery system also places a lot of pressure on local governments to offer coverage for these types of drugs. A lottery such as this creates a high level of disparity between patients, which may be viewed as a political tool to call for government support of these treatments. It is these kinds of initiatives that we would like to see change and replaced by more socially innovative approaches that help reduce the vulnerability of rare disease patients.

This approval has now opened the door to other gene therapies,which can be a cornerstone for the treatment of rare diseases (Black, 2020). Historically, gene therapy is an area of therapeutics aimed at curing, or significantly improving, diseases or conditions with few or no treatment alternatives (CADTH, 2018, p. 3). Numerous gene therapies have been approved outside of Canada or are in the advanced stage of clinical research (CADTH, 2018, p. 3), which is promising for rare disease patients and their families. How products life Zolgensma will be dealt with from a regulatory and healthcare systems perspective is to be seen. What we will be looking for is if this new development in treatment can also bring a new socially innovative approach to how medicines are developed, regulated and paid for.


Bastian, A., Dua, D., & Mirzahossein, S. (2016). The use of risk-sharing agreements to manage costs, mitigate risk, and improve value for pharmaceutical products. Journal of Clinical Pathways, 2(2), 43-51.

Black, M. (2020, December 16). Health Canada approves $2.8M treatment for spinal muscular atrophy. Global News.

CADTH. (2018, March 6). Gene therapy: An overview of approved and pipeline technologies. Canadian Agency for Drugs and Technologies in Health. Retrieved January 5, 2021, from

CADTH. (2019, April 15). Managing the "expense" in expensive drugs for rare diseases. Canadian Agency for Drugs and Technologies in Health. Retrieved January 7, 2021, from

CADTH. (2021, January 5). CADTH reimbursement review: Onasemnogene abeparvovec. Canadian Agency for Drugs and Technology in Health. Retrieved January 7, 2021, from

Cure SMA Canada. (2017, December 14). About SMA. Cure SMA Canada. Retrieved January 5, 2021, from

Dangouloff, T., & Servais, L. (2019). Clinical evidence supporting early treatment of patients With spinal muscular atrophy: Current perspectives. Therapeutics and Clinical Risk Management, 15(1), 1153-1161.

Dyer, O. (2020). Health ministers condemn Novartis lottery for Zolgensma, the world’s most expensive drug. British Medical Journal, 368(1), 580.

Forani, J. (2020, January 15). Ontario parents enter lottery for 'world's most expensive drug' to save their daughter's life. CTV News Canada.

Lunn, M. R., & Wang, C. H. (2008). Spinal muscular atrophy. The Lancet, 371(1), 2120-2133.

Weintraub, A. (2019, April 4). Pharma Novartis' SMA gene therapy would not be cost-effective if priced over $1.5M: ICER. Fierce Pharma. Retrieved January 6, 2021, from

Written by: Conor Douglas (PhD) and Shir Grunebaum (MA)

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