If Trikafta — Vertex Pharmaceuticals’ next-generation triple combination cystic fibrosis (CF) therapy — were to be available in Canada next year, the median age of CF patients could rise by 9.2 years and the number of deaths drop by 15% over the next 10 years, a forecast study reported.
Findings were reported in the study, “Projecting the impact of delayed access to elexacaftor/tezacaftor/ivacaftor for people with Cystic Fibrosis,” published in the Journal of Cystic Fibrosis.
Trikafta is a CFTR modulator — a therapy that aims to correct the defects in the CFTR protein that lead to the onset of CF — that can potentially be given to some 90% of CF patients to alleviate their symptoms.
The therapy combines two CFTR correctors — elexacaftor and tezacaftor — that are designed to help the CFTR protein fold correctly, with ivacaftor, a CFTR potentiator that maintains the CFTR protein in a stable conformation at the cell surface to improve its function.
Trikafta is approved in the U.S. to treat CF patients ages 12 and older carrying at least one copy of the F508del mutation, the most common disease-causing mutation and one estimated to be present in about 90% of all patients. The therapy was also recently approved in the European Union, where it will be available under the name Kaftrio.
Despite efforts by Canadian patient advocates and leaders in CF, the medication is yet to be approved in that country.
Researchers at the Dalhousie University, in collaboration with colleagues at St. Michael’s Hospital, and The Hospital for Sick Children (SickKids), reported the findings of a forecast study that aimed to estimate the benefits of making Trikafta available to those with CF living in Canada.
Predictions were made with the help of a microsimulation model of three different scenarios in which: Trikafta never became available in Canada, Trikafta was made available early (in 2021), or available beginning in 2025.
“To make the forecasts as realistic as possible, we used all the information we know about people living with CF today, and the initial results from the Trikafta clinical trials to project the impact Trikafta may have on the CF population in the future,” Sanja Stanojevic, PhD, assistant professor at Dalhousie in Nova Scotia, and the study’s lead author, said in a university news story.
Taking into account the disease state and the potential treatment effects of Trikafta on CF patients, the study estimated that if introduced in 2021, the therapy would reduce the number of patients in Canada with severe lung disease by 60% by 2030.
In this scenario, the number of people with mild lung disease would increase by 18%, and the number of pulmonary exacerbations (symptom worsening) would drop by 19% through 2030.
If made available by 2021, Trikafta might lower the number of CF-related deaths across Canada by 15%, and increase patients’ median age of survival by 9.2 years by 2030, the study indicated. Fewer lung transplants would likely be required for those with severe disease.
If patients are unable to have access to the medication before 2025, there is a possibility that these positive changes in CF lung health and survival will be mitigated.
“The expected benefits of therapy are cumulative, therefore delayed access to elexacaftor/tezacaftor/ivacaftor will result in preventable health care utilization and deaths,” the researchers wrote.
“It’s heartbreaking to know that these stats exist yet someone, like myself, has absolutely no way of getting their hands on something that could completely change my life,” said Jeremie Saunders, host of the Sickboy podcast who has CF.
This frustration is also shared by physicians caring for CF patients.
“It is frustrating as a clinician to know that there is a medication that has the potential to improve the health of over 90 per cent of individuals living with CF, but be unable to prescribe it,” said Anne Stephenson, MD, PhD, a respirologist and CF researcher at St. Michael’s Hospital, and senior author of the study.
According to Cystic Fibrosis Canada, delays in accessing Trikafta in the country are due to several reasons, including changes in the regulations governing the Patented Medicines Prices Review Board (PMPRB), the Canadian agency responsible for determining if a medication is excessively costly.
This opinion is also shared by Vertex, which is reported to consider market uncertainty stemming from these regulatory changes as part of the reason it has not yet submitted an application to Health Canada requesting Trikafta’s approval.
CF is one of the most common fatal genetic diseases in Canada, where about 4,300 people are estimated to have the disease.
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