Question Period Note: Amyotrophic Lateral Sclerosis (ALS)

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• Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a rare neurodegenerative disorder that affects the motor neurons responsible for controlling muscles. Affected individuals gradually lose the ability to walk, speak, eat, swallow and eventually breathe. There is no cure for ALS and there are few treatment options available.
• Health Canada has received letters from patients and their supporters expressing their desire to have the review of potential treatments expedited. Media interest has been high.
• Health Canada continues to advance policy and regulatory changes, informed by engagement with partners, industry and patients, to improve Canadian’s access to treatments.
• Health Canada approved Albrioza under the Notice of Compliance with conditions (NOC/c) policy in June 2022. Canada was the first jurisdiction in the world to receive and approve an application for this drug.
• Health Canada approved the oral suspension version of RADICAVA (edaravone) in November 2022, in addition to RADICAVA (edaravone) for intravenous injection was first approved in 2019. The oral suspension formulation provides a treatment option that can be administered at home to avoid frequent, long-term IV infusions.
• The Department also recently accepted Alsitek (masitinib) for review under the advanced consideration NOC/c policy.
• Health Canada recognizes the tragic consequences of Amyotrophic Lateral Sclerosis (ALS), the extreme burden it places on patients and their families, and the urgency for patients to have timely access to treatments.
• We are committed to continuing to work with our regulatory and health care partners, manufacturers and patients, to provide access to these medications while ensuring that the standards of safety, efficacy and quality are met.
• Over the years, the Department has expedited the review of ALS treatments using policy and regulatory initiatives to facilitate earlier access to drugs and made them available to requesting physicians via our Special Access Program.
• Several drugs for ALS have also participated in aligned reviews by Health Technology Assessment organizations (HTA) and Health Canada to minimize any potential delays to patient access between regulatory approval and decisions from provinces and territories on drug reimbursement.
• Health Canada continues to engage the ALS community. A Patient Listening Session in December 2021 allowed us to listen to the community and learn important information to inform policy and regulatory work. A “what we heard” report was published that provides an overview of what was shared with Health Canada at that session.

IF PRESSED ON ACCESS TO UNAPPROVED DRUGS
• There are processes in place for patients to access products that have not yet been approved for sale in Canada.
• The best option is usually for patients to register in a clinical trial. Most clinical trials require an authorization from Health Canada to ensure that the best interests of patients are protected and that a product is administered in accordance with national and international ethical, medical, and scientific standards. Clinical trials can involve a single patient.
• For serious or life-threatening conditions, including ALS, physicians may also request access to unauthorized drugs through the Special Access Program (SAP). The SAP provides access to non-marketed drugs for serious or life-threatening conditions when conventional therapies have failed, are unsuitable or are unavailable and when there is sufficient information available to support the use of the drug.
• Through the SAP, Health Canada has enabled many ALS patients to access unapproved drugs.

IF PRESSED ON PATIENT ENGAGEMENT
• Health Canada recognizes the vital perspectives and expertise that patients bring to the development, testing and regulation of health products. Patients have knowledge of their disease that industry and regulators do not. This is particularly true for rare diseases.
• HPFB is developing a Patient Involvement Strategy to ensure that patient 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
Health Canada held a Patient Listening Session focused on ALS in December 2021, which allowed us to listen to the ALS community and glean important information that could inform both policy and regulatory work. A “what we heard” report was published on our website that provides an overview of what ALS patients shared with Health Canada at that session

IF PRESSED ON THE PRIORITY/EXPEDITED REVIEWS OF ALS DRUGS
• A drug intended to treat a severe incurable disease, such as ALS, may qualify for one of the accelerated approval pathways. Health Canada advises sponsors on how to avail themselves of the Priority Review Policy or the Notice of Compliance with Conditions (NOC/c) Policy, when applicable, to accelerate the review of a drug for an unmet medical need
• The Priority Review of Drug Submissions Policy helps to expedite Health Canada’s review time for drugs. Drug manufacturers requesting a Priority Review must demonstrate that they meet specific criteria to be eligible.
• In August 2021, Health Canada accepted into review an application from Amylyx Pharmaceuticals Inc. for AMX0035. Health Canada reviewed this drug as quickly as possible while ensuring that our high standards of safety, efficacy and quality are met. ALBRIOZA was approved in June of 2022.
• Health Canada is continuously looking for ways to modernize its regulatory framework. To that end, the Department is currently considering additional ways to determine how we prioritize drug reviews to better meet the needs of the Canadian health care system and individual patients.

IF PRESSED ON REIMBURSEMENT
• Provincial and Territorial drug plans, with the exception of Quebec, use recommendations from the Canadian Agency for Drugs and Technologies in Health’s (CADTH), which assess the clinical and cost-effectiveness of drugs and issue non-binding formulary listing recommendations to participating public drug plans.
• While drug coverage, including formulary listing decisions, is primarily a PT responsibility, the federal government continues to work with PT jurisdictions to improve the efficiency of the pharmaceutical system by streamlining decision-making.
• Several drugs for ALS have participated in aligned reviews by Health Technology Assessment organizations (HTA) and Health Canada to minimize any potential delay between regulatory approval and decisions from provinces and territories on drug reimbursement.
Furthermore, the federal government is working with the provinces and territories as an active member of the pan-Canadian Pharmaceutical Alliance (pCPA). The pCPA combines governments’ collective buying power to negotiate lower prices on brand name drugs for all public plans and sets the price point for many generic drugs.

Under the Food and Drug Act and Regulations, all products sold or marketed in Canada and making a therapeutic claim need to be approved by Health Canada. The drug authorization process is initiated when a manufacturer submits an application to Health Canada for review. Every submission is then reviewed to assess the product’s safety, efficacy and quality.

The Priority Review of Drug Submissions Policy helps to expedite Health Canada review time for drugs from 300 days to 180 days. Drug manufacturers requesting a Priority Review must demonstrate that their application is for a serious, life-threatening or severely debilitating disease or condition and 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 October 2018, after a priority review, Health Canada authorized the drug Radicava (edaravone) to treat patients with ALS. Marketing of a drug is at the manufacturer’s discretion and there was a delay in this case. To ensure that ALS patients had access to edaravone as soon as possible following authorization and before marketing, Health Canada worked with the drug manufacturer and Canadian ALS specialists to make the drug available by request from practitioners through Health Canada’s Special Access Program (SAP). On November 5, 2019, the manufacturer began marketing edaravone in Canada. Recently, an oral formulation of edaravone was authorized by Health Canada.

In the case of Amylyx Pharmaceuticals Inc.’s submission for Albrioza, the sponsor was not able to meet these criteria and therefore a Priority Review was not granted. However, Health Canada reviewed the submission as quickly as possible and has since granted an approval under the NOC/c policy.

To accelerate access for Canadians to the therapies they require, Health Canada, along with Canadian Agency for Drugs and Technologies in Health (CADTH) and Quebec’s Institut national d’excellence en santé et en services sociaux (INESSS), 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 Alsitek submission is being reviewed as part of an aligned review and under the NOC/c advanced consideration policy.

The SAP considers requests from practitioners to access drugs that are not available on the Canadian market to treat patients with serious or life-threatening conditions when conventional treatments have failed, are unsuitable or unavailable and when sufficient information is available to support its use. Decisions by the SAP are made on a case-by-case basis. Through the SAP, Health Canada has enabled early access to various product to numerous patients with ALS

SAP does not provide drug funding to Canadians for access to medication nor does it or Health Canada have a role in the drug reimbursement process. Decisions on insurance coverage or funding are under the jurisdiction of the provinces and territories.

The Government of Canada is committed to improving the affordability and accessibility of prescription drugs for all Canadians. That is why we are working with provinces and territories as an active member of the pan-Canadian Pharmaceutical Alliance (pCPA) to combine the governments’ collective buying power to negotiate lower prices on brand name drugs for all public plans, including high cost drugs for rare diseases.

Health Canada approved Albrioza on June 22, 2022 with a Notice of Compliance with Conditions. CADTH listed its reimbursement recommendation on July 21, 2022 and on August 9th, 2022, Pan Canadian Pharmaceutical Alliance (pCPA) engagement letter was sent to Amylyx Canada to initiate active negotiation of Albrioza. PCPA aims to finalize negotiations within 90 business days from the date the initial engagement. As of November 28th, 2022, Albrioza is under active negotiation with the PCPA and provincial public plans.

• In Canada, there are now currently three drugs authorized for ALS:
- RILUTEK (riluzole), an oral tablet, was approved in Canada in 2000, and may extend survival and/or time to tracheostomy in some patients with ALS.
- RADICAVA (edaravone), given by intravenous (IV) infusion in cycles, was approved in Canada in 2019 and is indicated to slow the loss of function in patients with ALS. RADICAVA (edaravone oral suspension) was approved in November 2022.
- ALBRIOZA (phenylbutyrate and taurursodiol), a powder for oral suspension, was approved in 2022 for the treatment of ALS.
- The Department also recently accepted ALSITEK (masitinib) for review under the advanced consideration NOC/c policy.

• Health Canada reviews ALS drugs in an expedited manner. The Priority Review policy may be used for a serious, life-threatening or severely debilitating disease or condition and for which there is substantial evidence that a new drug:

• Provides effective treatment, prevention or diagnosis of a disease or condition for which no drug is presently marketed in Canada; or
• Provides a significant increase in the overall benefit/risk profile over existing therapies for a disease or condition that is not adequately managed by an existing drug