Cystic fibrosis (CF) is an inherited disease that impacts the whole body. People with CF experience a buildup of thick mucus in the lungs and other organs, which causes problems with breathing, increases risk of infection, and makes it difficult to digest and absorb nutrients from food. Living with CF means a high burden of care for patients, requiring physical therapy to clear airways several times a day, and many pills a day to help absorb nutrients from food and to thin the mucus in the body. In addition to daily therapies, frequent antibiotic use is required to treat infections from bacteria that can grow in the mucus.Reference2 Canadians with CF can expect to visit a specialized clinic 4 times a year for regular checkups.Reference3 This can mean travelling long distances, as only half of Canadians living with CF live within 50 km of a specialized CF clinic; 1 in 3 Canadians travel more than 100 km for routine care.Reference4

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Cystic fibrosis means countless hours of medication, therapies, medical appointments, phone calls and paperwork. Being a medical parent is often a full-time job on its own. — Paula, Caregiver, Ontario

The recent introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators has significantly changed care trajectories. These medications improve the body’s ability to move salt and fluid throughout the body, reducing the thick, sticky mucus. TrikaftaFootnotei is the most recent version of a CFTR modulator. It was approved for use in Canada on June 18, 2021, and quickly became the most common CFTR modulator prescribed in 2022.Reference1 Of the approximately 4,445 Canadians with CF in 2022, over half (56%) were on a CFTR modulator; almost all were Trikafta.Reference1 Due to the high cost of Trikafta, this medication accounted for 25% of the growth in public drug program spending in 2022.Reference5 For more information about public drug program coverage for Trikafta, refer to  CIHI’s Pharmaceutical Data Tool.Reference6  

Prescriptions for Trikafta rose quickly after its approval 

Notes
This graph includes patients with prior exposure to cystic fibrosis transmembrane conductance regulator (CFTR) modulators to show overall use. 
The analysis includes patients from Newfoundland and Labrador, New Brunswick, Ontario, Manitoba, Saskatchewan, Alberta and British Columbia.
Data for patients taking Trikafta that was paid fully through private sources (i.e., private insurance, pharmaceutical companies, clinical trials) will not be captured. It is estimated that the number is small and will have minimal impact on the findings. All results here should be treated as an underestimate.

Source
National Prescription Drug Utilization Information System, June 2021 to December 2022, Canadian Institute for ºìÁì½í¹Ï±¨ Information.

Our analysis captures patients who started to take Trikafta between June 1, 2021, and March 31, 2022, and compares their acute and chronic health system use in the year prior to starting Trikafta with their health system use in the year after starting Trikafta. Regional inclusion is based on data availability and may differ by analysis. Refer to the methodology notes and data tables for more detail.

Clinical trials of Trikafta show improved lung functioning, improved CF respiratory symptoms, increased weight and fewer pulmonary exacerbations — in particular, exacerbations leading to hospitalizations and requiring IV antibiotics.Reference7

The need for either care in the emergency department (ED) or a stay in hospital represents the more acute events experienced by those with CF. In the year after beginning Trikafta, CF patients had fewer visits to the ED and those visits were less likely to end in admission to hospital (a drop of 62%). In the year prior to starting Trikafta, approximately one-third of patients were hospitalized; in the year after starting Trikafta, less than one-sixth of patients were hospitalized. Additionally, for those admitted to hospital, their average length of stay was 40% shorter in the year after starting Trikafta. The decrease in need for acute care was largest among younger age groups. (Refer to the data tables for details.)

Impact on acute care use in the year after starting Trikafta

Notes
Hospitalizations included patients from Newfoundland and Labrador, New Brunswick, Ontario, Manitoba, Saskatchewan, Alberta and British Columbia.  
Patients with prior exposure to CFTRs are excluded from this analysis.
ED visits included patients from Ontario and Alberta.
Commonly used oral antibiotics for acute infections were identified by the clinical advisory group. This analysis includes claims from Manitoba, Saskatchewan and British Columbia. Analysis was performed on supply days (i.e., the number of days the person was taking the medication).

Sources
Discharge Abstract Database and National Ambulatory Care Reporting System, 2020–2021 to 2022–2023; and National Prescription Drug Utilization Information System, 2010 to 2023, Canadian Institute for ºìÁì½í¹Ï±¨ Information.

In the year prior to starting Trikafta, most patients stayed 14 days in hospital, the length of time for a typical course of intravenous (IV) antibiotics. In the year after starting Trikafta, most patients stayed 1 day. Both clinicians and patient advisors identify IV antibiotic therapy as the most common reason for hospitalizations. (Refer to the data tables for details on hospital admissions.) 

Routine care can include regular visits to physicians to monitor lung functioning, lab tests and renewal of medications. (Refer to the data tables for specific changes in standard care.) In the year after starting Trikafta, the following changes occurred:

• The number of patient visits to physiciansFootnoteii  (excluding inpatient care) decreased (-5%). Changes for in-person and virtual visits varied significantly based on where the patient lived. (Refer to the data tables for rates.)
• Routine testingFootnoteiii of pulmonary function and chest X-rays decreased (-16% and -36%, respectively), while laboratory testing for liver and kidney functioning increased 46%; the latter is expected when a new drug is being monitored. (Refer to the data tables for specific laboratory tests.) 
• Use of medications for chronic symptom managementFootnoteiv  of CF decreased. The largest decrease was seen in routine drugs (supply days) for obstructive airways (-42%) and mucolytics (-38%) as people have less mucus build up in their lungs; the smallest decrease was seen in prophylactic use of antibiotics (-9%). Medication for digestive enzymes was the only exception, with a 5% increase in the year post-Trikafta compared with the year prior. (Refer to the data tables for a detailed breakdown of changes.) 

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In the first year following initiation of Trikafta, we followed our patients very closely to assess clinical response and to monitor for side effects like liver toxicity; this should decrease after the first year. In the future, I expect we will see our patients less frequently outside of routine clinic visits as they are sick much less often. — Dr. Bradley Quon, Medical Director, St. Paul’s Hospital Adult CF Clinic

Access to Trikafta and other CF drugs varied across the country, with patients reporting that they experience both logistical and financial barriers. In some regions, Trikafta and other CF medications are accessible from certain pharmacies only. Not surprisingly, given the number of medications people with CF need to take, they reported that travel to multiple pharmacies and coordination of prescription renewals can be a substantial burden. In contrast, in some regions, CF clinics compile the medications and coordinate payment with public drug programs and private insurance providers on behalf of the patient, shifting burden away from the patient.

Patients and families also reported facing financial barriers to accessing Trikafta. The amount a person pays out of pocket for the medication varies, depending on public drug program coverage for high-cost drugsFootnotev (i.e., publicly covered), their personal income (which determines the amount public drug programs will cover) and whether they have access to private health insurance (e.g., through their employer’s benefits). This can create inequities between individuals even if they live in the same province.

In 2022, ºìÁì½í¹Ï±¨reported that public drug programs in Canada spent $285 million on Trikafta.Reference5 According to ºìÁì½í¹Ï±¨data, nearly all Canadians (99%) have at least 90% of their Trikafta costs publicly covered, and 78% have 100% of their costs publicly covered.Footnotevi For medication costs not covered by public drug programs, patients typically rely on a combination of private insurance coverage and out-of-pocket payment. With the high cost of Trikafta, having a small copay or no insurance could result in hundreds or thousands of dollars in out-of-pocket costs each month.

In the Trikafta cohort (patients taking Trikafta), people in the highest income group were more likely to take Trikafta compared with those in the general CF cohort (refer to the image below). ºìÁì½í¹Ï±¨does not hold information on private insurance, but patients we spoke to reported relying on private insurance and paying high copays in order to continue their Trikafta treatment. For some patients, coverage for Trikafta through their employment benefits was a factor when deciding whether to accept a new job or leave their current job. Caregivers of younger children with CF expressed concern over their children’s future access to Trikafta when they are no longer covered as dependants under the parent’s insurance.

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I was recently entertaining applying for a new position. It would mean living in the same city as the CF clinic, closer to our child’s medical teams, but the benefits associated with the job did not have the same medical coverage as my current employer. We could not follow through because of the medical coverage. I worry about what happens when my son is too old to be covered under my insurance. What happens if his job does not have a benefits package that covers Trikafta? What if insurance companies or the governments change their mind about covering Trikafta? — Paula, Caregiver, Ontario

Access to Trikafta can vary by income level

Notes
Trikafta cohort: Patients taking Trikafta.
General CF cohort: Canadian population data by income quintiles for CF patients is not available. The Ontario CF population is used as a proxy for a Canadian comparison.
The analysis includes patients from Newfoundland and Labrador, New Brunswick, Ontario, Manitoba, Saskatchewan, Alberta, and British Columbia. 
Patients with prior exposure to CFTRs are excluded from this analysis.

Sources
Discharge Abstract Database, 2020–2021 to 2022–2023; and National Prescription Drug Utilization Information System, April 1, 2021, and March 31, 2022, Canadian Institute for ºìÁì½í¹Ï±¨ Information.
Arya R, et al. . Journal of Cystic Fibrosis. 2024.

This analysis shows real-world evidence of how health system use for patients with CF changes after starting Trikafta. Other sources are starting to report other impacts of Trikafta for patients with CF:

• The CF registry reported a decline in lung transplants performed since CFTR modulators have became available, and patients have been removed from transplant wait-lists — almost all of whom were on CFTR modulator therapy.Reference1
• CF often comes with reproductive barriers,Reference8 but emerging anecdotal evidence could suggest that Trikafta is removing those barriers. 

The number of Canadians living with CF is forecast to grow as people live longer due to the effectiveness of the drug. The effects of Trikafta are estimated to decrease deaths by 15%, improving median survival age by 9 years.Reference9 Prior to Trikafta,Reference10 the socio-economic cost burden of CF in Canada — including costs to individuals, society and caregivers — was an estimated $414 million. This analysis is a first step in understanding the impacts this new therapy will have on those living with cystic fibrosis.

• Impact of Trikafta on Individuals Living With Cystic Fibrosis — Data Tables
• Drugs for rare diseases
• Data Learnings for Rare Disease Analysis 
• Pharmaceutical Data Tool

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