Question Period Note: TRIKAFTA

• What is Health Canada doing to make Trikafta available to Canadians with cystic fibrosis?

• Health Canada recognizes the importance of patient access to new therapies for serious or life-threatening conditions. We are aware of the many steps involved in the approval and coverage of drugs in Canada, and that this can be frustrating for families who are trying to gain access to treatments for their loved ones as quickly as possible.

• On November 16, Health Canada granted Priority Review status for Trikafta, a new drug used for the treatment of cystic fibrosis, which is marketed by Vertex Pharma. As per Health Canada policy, Vertex Pharma now has 60 days to submit the application for drug authorization.

• For serious or life-threatening conditions, such as cystic fibrosis, physicians may request access to unauthorized drugs through Health Canada’s Special Access Programme (SAP). This continues to be the case for Trikafta. As of November 2020, 160 patients in Canada have accessed Trikafta through this program.
• To help Canadians get better access to effective treatments, Health Canada is working with provinces, territories and other partners to develop a national strategy for high cost drugs for rare diseases. This is an important step in expanding drug coverage for patients with rare diseases, through federal support.

Trikafta, by the drug manufacturer Vertex Pharma, is the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. It is reported to increase lung function by an average of 14%, which is a significant improvement for many patients struggling with the effects of cystic fibrosis.

A study published in the “Journal of Cystic Fibrosis” on August 24, 2020, predicted benefits that will be lost by cystic fibrosis patients should the drug not be available to them by 2021. Access to the drug by next year would result in a 60% decrease in severe cases, 19% decrease in hospitalization, fewer lung transplantations and a 9-year increase in survival. Such results would have a significant impact not only on the personal health of these patients but also on the health care system.

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 by scientists to assess the product’s safety, efficacy and quality.

At this time, Health Canada has not received a new drug submission for Trikafta. However, Vertex Pharma did apply for a request for a Priority Review, which was granted on November 16, 2020. According to Health Canada policy, the company now has 60 days to submit their application.

Health Canada maintains a Submissions Under Review (SUR) List, which includes all new drug submissions and supplemental new drug submissions for new uses that are currently under review, but for which the Department has not made a final decision on authorization. Trikafta will be added to this list if the submission is received and accepted into review.

Products containing compounds found in Trikafta are approved by Health Canada for use in treating Cystic Fibrosis. The products are:

• KALYDECO (Ivacaftor)
• SYMDECO (Ivacaftor and Tezacaftor)
• ORKAMBI (Ivacaftor and Lumacaftor)

(Trikafta contains ivacaftor, tezacaftor and elexacaftor)

All drug products approved for sale in Canada are listed on the Drug Product Database, available at: https://health-products.canada.ca/dpd-bdpp/index-eng.jsp.

The Special Access Program (SAP) considers requests from practitioners treating patients with serious or life-threatening conditions for drugs that are unavailable for sale in Canada when conventional treatments have failed, or are deemed unsuitable for the patient. Decisions by SAP are taken on a case-by-case basis. They are based on the clinical details of the patient’s unique situation as well as the clinical reasons why other marketed therapies may not be suited for a patient. As of November 2020, 160 patients in Canada have accessed Trikafta through SAP.

SAP does not provide drug funding to Canadians for access to medication and does not 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.

Additionally, in August 2019, the Government announced final amendments to the Patented Medicines Regulations. These amendments will give the Patented Medicine Prices Review Board (PMPRB) the tools it needs to protect Canadians from excessive prices and make patented medicines more affordable for all payers - public and private drug plans, as well as Canadians who pay out of pocket for their prescription drugs, including high cost drugs for rare diseases. They will come into force in January 2021.

SYNOPSIS

In October 2019, the US Food and Drug Administration approved Trikafta, the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation, at an annual cost of over $300,000 USD. Although Health Canada has not received a new drug submission for Trikafta, as of November 2020, 160 patients in Canada have accessed this drug through the Special Access Program (SAP). The Department has received many letters from patients and their supporters expressing their desire to have this drug made available in Canada, and media interest is high, particularly because of its use in the pediatric population.
IF PRESSED ON THE DRUG APPROVAL

• Health Canada carefully reviews new drugs to determine that they are safe, effective and of good quality.

• Priority Review is a fast-track status granted to eligible new drug submissions for human use, for serious or life-threatening illness, where there is no other treatment available, or the new treatment promises significant benefits over available treatment options. Priority Review submissions have an accelerated review target of 180 days.

• The actual time for review may vary if Health Canada needs to request additional information from the sponsor in order to reach a final regulatory decision.

• Applications for new drug authorizations appear on Health Canada’s Submission Under Review List. The submission for Trikafta will be published on the list once it is accepted for review.
IF PRESSED ON CONCERNS WITH IMPACTS ON REVENUE TO INDUSTRY AND DRUG ACCESS

• Even with lower prices, revenues from patented drug sales are expected to continue growing over the next ten years in Canada.

• Our Government has also streamlined regulatory processes supporting faster access to the Canadian market for products.

• While Health Canada encourages manufacturers to submit applications, it is the manufacturer’s decision whether to do so.