WHAT IS THE REIMBURSEMENT RECOMMENDATION FOR TRIKAFTA?: Canada’s Drug Agency (CDA-AMC) recommends that Trikafta be reimbursed by public drug plans for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least 1 of the 152 non-F508del mutations in the gene that are identified in Figure 1 of this document, if certain conditions are met. WHAT ARE THE CONDITIONS FOR REIMBURSEMENT? Patients aged 2 to 5 years who start treatment with Trikafta should be evaluated for response after 1 year, and those aged 6 years or older should be evaluated after 6 months. The physician must provide evidence that the patient is benefiting from the treatment. The cost of Trikafta must also be reduced. WHY DID CDA-AMC MAKE THIS RECOMMENDATION? Six clinical studies demonstrated that Trikafta was well tolerated and resulted in clinically meaningful improvements in end points that are important for those living with CF (e.g., lung function, nutritional status, pulmonary exacerbations, and quality of life). For the rare mutations that have not been evaluated in clinical trials (i.e., those where the response to Trikafta has only been assessed in a laboratory), the committee acknowledged the practical challenges with generating clinical evidence. The committee concluded that Trikafta has the potential to provide clinically meaningful benefit for patients with CF caused by rare mutations, on the basis that the laboratory data were considered acceptable by Health Canada and that the biological plausibility of a beneficial treatment effect was supported by the CF specialists who provided input for this review. Based on the CDA-AMC assessment of the health economic evidence, Trikafta does not represent good value to the health care system at the public list price. A price reduction is therefore required. Based on public list prices, reimbursement of Trikafta for patients with at least 1 of the 152 non-F508del mutations in the gene that are identified in Figure 1 is estimated to cost the public drug plans approximately $61 million over the next 3 years.
WHAT IS CF? CF is a progressive, fatal, genetic disease that primarily affects the lungs and digestive system. Those living with CF lose the ability to breathe due to accumulated lung damage, caused by chronic lung infections and inflammation. UNMET NEEDS IN CF: There are significant unmet therapeutic needs for those living with CF. There are no treatments currently available that effectively meet the most important goals of CF therapy: prolonging survival, preventing the need for lung transplant, slowing the decline in lung function over time, and reversing the course of the disease. HOW MUCH DOES TRIKAFTA COST? Treatment with Trikafta is expected to cost $306,810 per patient per year.
