How new drugs become approved and accessible to Canadians

Earlier this year, manufacturer Amylyx announced that it plans to pursue Health Canada approval for its AMX0035 therapy. We’ve updated the blog post we first published back in 2018 about how new drugs become accessible in Canada to help orient you to the steps involved, their purpose, the milestones along the way and the timelines involved.

The bottom line is that it simply takes too long for a drug to move from regulatory approval through to public reimbursement (where a provincial drug plan decides to cover some, or all, of the cost of a drug). Ultimately, we want to see a streamlined series of processes that see Health Canada approved ALS therapies made accessible quickly and equitably regardless of where in the country someone lives. Our advocacy efforts will be ramping up to support this need. In the meantime, here is an overview of the current approach.

How it works

• Health Canada conducts a review of the drug to assess its safety, efficacy and quality.
• The process begins when the drug manufacturer files a New Drug Submission (NDS) with Health Canada. Health Canada must accept the New Drug Submission before regulatory review begins.
• If Health Canada concludes the benefits of the drug outweigh the risks and that the risks can be mitigated, they will issue a Drug Identification Number (DIN) and a Notice of Compliance (NOC). This means the drug is approved to market in Canada and physicians can begin prescribing it. At this point, however, the drug is only accessible by paying for it out-of-pocket or through private insurance.
• If the drug does not receive a Notice of Compliance, the drug’s manufacturer has the option of providing additional information, re-submitting the application at a later date with additional supporting data, or asking Health Canada to reconsider its decision.

How long it takes

Standard review is one year; Priority Review is 180 days. ALS therapies are typically Priority Review.

How it works

The drug manufacturer makes a submission to the Common Drug Review, which is administered by a federal agency called the Canadian Agency for Drugs and Technologies in Health, or CADTH. (In Quebec, this process is managed by the Institut national d’excellence en santé et en services sociaux, or INESSS.)

The Common Drug Review conducts an evaluation of the clinical, economic, patient, and clinician evidence of the drug. It results in recommendations and advice that are used by federal, provincial, and territorial drug plans to make decisions about whether the drug costs will be covered whether through public programs.

Patient input  and, since 2020, clinician input, are key parts of the Common Drug Review. Patient groups are invited to share their experiences, perspectives and expectations for the drug under review and clinicians have the opportunity to comment on the benefits of the therapy from a clinical perspective. Together the patient and clinician input help to inform CADTH about the realities of an ALS diagnosis and how therapies that have even modest clinical impact can make a significant difference in quality of life.

ALS Canada is planning a CADTH patient input submission for AMX0035 and will be inviting the Canadian ALS community to contribute to it. If you would like to participate, sign up for our e-newsletter to be notified.

At the conclusion of the Common Drug Review, one of the following recommendations is made:

• That the drug be reimbursed;
• That the drug be reimbursed with clinical criteria and/or conditions; or
• That the drug not be reimbursed.

The final recommendation is shared with participating drug plans and the manufacturer and is posted on cadth.ca.

Following the completion of the Common Drug Review, the federal, provincial and territorial drug plans can choose to work together through the pan-Canadian Pharmaceutical Alliance (pCPA) to determine what public reimbursement could look like within each jurisdiction based on negotiation with the manufacturer.

Each jurisdiction then typically does its own independent review to determine if and how the drug will be covered by its particular public drug plan. Once the review is complete, the jurisdiction’s health authority (e.g. Ministry of Health) will make the final decision about public drug plan coverage. Federal, provincial and territorial drug plans choosing to cover the therapy in whole or in part will have a Product Listing Agreement with the manufacturer.

Increasingly, private health plans are referencing the CADTH recommendation when making decisions about whether they will cover the drug.

How long it takes

• In 2018 Health Canada, CADTH and INESSS announced an aligned review process where the Common Drug Review will accept submissions six months before they expect to receive a Notice of Compliance from Health Canada.
• The Common Drug Review has a target timeline of approximately six months. However, there are multiple variables that can affect the process and extend it upwards of one year or more.
• Timelines associated with the pan-Canadian Pharmaceutical Alliance and subsequently the Product Listing Agreements with individual federal, provincial and territorial drug plans can easily take up to two years complete, depending on the jurisdiction. Throughout the process there are often ways for patients, caregivers, patient groups and health care professionals to push for an expedited process.

How it works

• While Health Canada approval and reimbursement reviews are in process, and throughout the duration of the drug’s patent, the drug is subject to price controls, which fall under the authority of the Patented Medicine Prices Review Board (PMPRB). The PMPRB is a federal agency that sets the maximum price at which companies sell their drug products in the Canadian marketplace (e.g. to hospitals, pharmacies, public drug plans, private drug plans, out of pocket payers).
• Drug manufacturers must submit specific pricing information to the PMPRB when the drug is first introduced and then on a semi-annual basis. PMPRB publishes information on its price reviews on its website.
• The federal government recently updated the guidelines that set out how patented therapies are priced in Canada. They are set to come into effect on July 1, 2021. ALS Canada is one of many organizations that have voiced concerns about the potential impact of these reforms. We believe that the model used to determine pricing must balance affordability with access.

How I access a therapy if a price has not yet been set?

A final price does not have to be determined by PMPRB before the drug can be purchased by a patient. In general, as long as Health Canada has approved the therapy and supply is available, clinicians are able to prescribe it based on the product monograph. However, depending on the status and outcome of the pCPA negotiations and reimbursement decisions by public and private drug plans, a patient may have to pay out of pocket to ultimately get access.

Case study: Radicava

Here are the timelines associated with the approval and reimbursement of Radicava, the most recent ALS therapy to become available in Canada. ALS Canada made a patient input submission and initiated an advocacy campaign that saw more than 3,600 Canadians email their provincial Health Ministers (or equivalent) to advocate for the province to make a quick decision in favour of covering the drug. As of May 2021, the province of PEI has still not made a reimbursement decision, so if you are a PEI resident please consider contacting your Health Minister.