Question Period Note: TRIKAFTA

• Trikafta, manufactured by Vertex Pharma, is the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Following a Priority Review, Health Canada approved Trikafta on June 18, 2021, for the treatment of cystic fibrosis patients aged 12 and older. In July, Health Canada received a subsequent submission (a Supplemental New Drug Submission) that would expand the indication to include children aged 6 and older.
• The cost of Trikafta runs at over $300,000 annually. In September, some provincial governments announced that they would be funding Trikafta on their public drug plans for eligible patients.
• Health Canada 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.

KEY MESSAGES
• Health Canada recognizes the importance of patient access to new therapies for serious or life-threatening conditions, and is committed to supporting Canadians’ access to safe and effective health products.
• I am happy to share that on June 18, 2021, following a priority review, Health Canada authorized Trikafta for the treatment of cystic fibrosis in patients aged 12 years and older. This approval offers Canadian patients with another treatment option to combat this terrible disease.
• We have also granted Priority Review status to a subsequent submission that seeks to expand the indication of Trikafta to include children aged 6 years and older.
• Health Canada is pleased with CADTH’s positive decision to reimburse Trikafta and with the Pan Canadian Pharmaceutical Alliance’s recent letter of intent. We are equally pleased that several provinces have begun reimbursing this needed treatment.
• 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.
IF PRESSED ON THE DRUG APPROVAL
• Health Canada carefully reviews new drugs to determine that they are safe, effective and of good quality.
• For Trikafta, Health Canada fast-tracked the process using the Priority Review pathway, which applies to eligible submissions for serious or life-threatening illness, where there is no other treatment available, or the new treatment promises significant benefits over available treatment options. These submissions have an accelerated review target of 180 days compared to the regular 300-day target.
• We also aligned our review with our Health Technology Assessment partners to expedite access to this needed treatment.
• Health Canada is fully committed to openness and transparency, which is why we have published the Regulatory Decision Summary. This document describes the rationale for authorizing Trikafta. A more detailed explanation of the recommendation, the Summary Basis of Decision, will be published on our website in October.
• 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). Before its availability on the Canadian market, 253 patients in Canada accessed Trikafta through this program.
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.

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.

Trikafta was issued its Notice of Compliance (NOC)—or drug approval—on June 18 2021.

The Regulatory Decision Summary (RDS) describing the NOC recommendation was posted to the Health Canada website on September 8, 2021 and the more detailed Summary Basis of Decision (SBD) is currently scheduled to be posted to the Health Canada website in October 2021.

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. Before its availability on the Canadian market, 253 patients in Canada had 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.

KEY FACTS

• 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.
• 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.