441-00283 (Human diseases)

The Government of Canada recognizes the importance of patients having access to drugs that may help treat their serious or life-threatening conditions, including those with rare diseases such as Amyotrophic Lateral Sclerosis (ALS), and is committed to supporting Canadians’ access to safe and effective health products.

In Canada, the management of pharmaceuticals is a shared responsibility among the federal, provincial and territorial governments. The Federal Government is responsible for assessing the safety, efficacy and quality of drugs before authorizing them for sale in Canada, and for monitoring their safety after they enter the Canadian market. The provincial and territorial governments are responsible for the delivery of health care for their residents, including determining which drugs are reimbursed and under what conditions for their eligible populations.

Once Health Canada authorizes a drug for sale in Canada, provincial and territorial drug plans then decide if the drug will be eligible for public reimbursement. To inform this decision, public drug plans use the recommendations from the Canadian Agency for Drugs and Technologies in Health (CADTH) and Quebec’s Institut national d’excellence en santé et en services sociaux (INESSS).

Prescription drugs and drug therapies are evolving quickly, and Canadians want access to the latest, most effective treatment options. The Priority Review of Drug Submissions Policy is available for drugs intended for the treatment, prevention or diagnosis of serious, life-threatening or severely debilitating illnesses or conditions, with an accelerated review target date of 180 days instead of the usual 300 days. Additionally, the Notice of Compliance with Conditions pathway provides access to similar drugs which have promising clinical benefit; this pathway has a 200 day review target. Drug manufacturers requesting a Priority Review must demonstrate that their Drug Submission is for a serious, life-threatening or severely debilitating disease or condition for which there is substantial evidence of clinical effectiveness that the drug provides:

• Effective treatment, prevention or diagnosis of a disease or condition for which no drug is presently marketed in Canada; or,
• A significant increase in efficacy and/or significant decrease in risk such that the overall benefit / risk profile is improved over existing therapies, preventatives or diagnostic agents for a disease or condition that is not adequately managed by a drug marketed in Canada.

In the case of Amylyx Pharmaceuticals Inc.’s submission for AMX0035, the drug manufacturer was not able to meet these criteria; therefore, a Priority Review was not granted by Health Canada. Due to the serious nature of ALS, and despite not meeting the necessary criteria to be assigned priority review status, Health Canada has committed to reviewing the AMX0035 submission as quickly as possible, having planned for the review of this submission and allocated resources accordingly.

To further accelerate access for Canadians to the therapies they require, Health Canada, along with CADTH and INESSS, have also introduced an option for sponsors to seek aligned reviews for drug submissions. Sponsors of qualifying submissions can opt in for an earlier Health Technology Assessment (HTA) review up to 180 days before Health Canada makes its regulatory decision about the drug submission. This aims to reduce time lags between the Department’s market authorization and HTA recommendations, where possible. The current AMX0035 submission is being reviewed as part of an aligned review.

In some circumstances, drugs not yet approved in Canada may be accessed through Health Canada’s Special Access Programme (SAP). The SAP provides practitioners with access to non-marketed drugs in order to treat patients with serious or life-threatening conditions when conventional therapies have failed, are unsuitable or offer limited options. Health Canada has been working to reduce the burden on SAP applicants and to secure access to products as rapidly as possible.

The Government of Canada has made a commitment to improve access to medications. The Regulatory Review of Drugs and Devices Initiative, which began in 2017, consists of a series of projects to support access to medicines, including working closely with our international regulatory partners to jointly review new drugs.

Health Canada is now advancing a Regulatory Innovation Agenda to make its regulations for drugs and devices more agile so that they support innovation while protecting the safety of Canadians. It is building on its experience in responding to the COVID-19 pandemic, where agile regulatory approaches, along with extra surge capacity and funding, were used to help enhance timely access to needed products. For example, Health Canada is modernizing its regulations for clinical trials and for market access, which will help bring more trials to Canada and further enable access to safe, new health innovations. 

Health Canada is also developing a Patient Involvement (PI) Strategy to ensure that patient expertise, experiences, perspectives, needs and priorities are incorporated into Health Canada’s policy and regulatory work around health products in a meaningful way. This strategy is expected to establish a systematic approach for engaging with patients on the topic of health products, including collecting input and integrating feedback in decision-making.

As part of the PI Strategy, Health Canada hosted a Patient Listening Session with the ALS community on December 7, 2021, which allowed Health Canada to ask patient representatives important questions that could inform both its policy and regulatory work. The session was very meaningful and impactful for Health Canada staff, and was appreciated by the participating patient representatives. A summary report of the session will be posted on Health Canada’s website. Ideas generated during the session will be discussed and considered in future policy and regulatory work.

The government recognizes the need to better understand the underlying mechanisms for ALS and investigate potential treatments. That is why, over the past five years, the Federal Government – through the Canadian Institutes of Health Research (CIHR) – has invested approximately $37 million in research relating to ALS. Some notable examples of this research include:

• A $2.8M foundation grant, awarded to Dr. Jean-Pierre Julien of Université Laval to study the pathogenic pathways of ALS and to develop innovative treatment for ALS based on new therapeutic targets.
• A $4.9M foundation grant, awarded to Dr. Guy Rouleau of McGill University to develop a drug discovery platform using ALS relevant cells derived from induced pluripotent stem cells prepared from patients. His research will focus on the causative role for somatic mutations in the spinal cord to explain the cause of a significant fraction of sporadic ALS cases.
• A $1M project grant, awarded to Dr. Honglin Luo and Dr. Neil Cashman of the University of British Columbia to study the role of enteroviral infection in ALS to identify new factors that contribute to the development of ALS and develop new treatments for ALS.
• A $952K project grant, awarded to Dr. Jeehye Park at the Hospital for Sick Children to identify key disease-initiating events and investigate how and why these specific neuronal groups are more susceptible to degeneration. Identification of the disease-initiating events will provide important clues to the development of effective strategies for early disease prevention and therapeutic intervention for ALS.

Moreover, the Government, through CIHR, has a long history of collaborating with partners, such as the ALS Society to advance research on ALS, and will continue to support national and international initiatives that provide the evidence we need to tackle this terrible disease.

Finally, Budget 2019 proposed to invest up to $1 billion over two years, starting in 2022-2023, with up to $500 million per year ongoing, to help Canadians with rare diseases access the drugs they need. We recognize that, for many Canadians who require prescription drugs to treat rare diseases, the cost of these medications can be astronomically high.

To support the development of a national strategy for drugs for rare diseases, a public and stakeholder engagement process was launched in early 2021 and concluded on March 26th, 2021. A What We Heard report summarizing key themes and feedback that emerged during the public and stakeholder engagement was published in July 2021.

The Government of Canada will continue working with provinces, territories and other partners to improve access of medications.