After years of fighting to have lifesaving medications covered for cystic fibrosis (CF) patients in Canada, local mother-daughter duo Beth and Madi Vanstone are celebrating this week as the province has agreed to provide coverage for the expensive medication under its publicly funded drug program.
Patients who are eligible and qualify for Trikafta, a specialized CF medication, will have access to the medication through public drug programs, with eligibility criteria yet to be specified. At list price, Trikafta costs approximately $300,000 per patient per year.
"She cried when I told her!" Beth Vanstone said of her daughter's reaction to the news. "I am so happy for all the lives that will be saved and changed, the weight lifted from families shoulders, it's a big relief."
A CF official agreed.
"Our community has fought hard for this day and sadly lost many loved ones along the way," said Dr. John Wallenburg, chief scientific officer of Cystic Fibrosis Canada in a news release.
"While we are pleased with the news, we also need to ensure that access to Trikafta is granted to everyone eligible under Health Canada’s indications. We ask the provinces to follow Cystic Fibrosis Canada’s clinician-developed guidelines and provide access to all Canadians living with CF who would benefit from this life-changing treatment," said Wallenburg.
In June 2021, Health Canada approved the use of Trikafta, but the Canadian Agency for Drugs and Technologies in Health (CADTH) had outlined strict criteria recommendations for public funding to gain access to the drug. If adopted, the recommendations would exclude 27 per cent of the CF patient community, mostly affecting youth.
The recommendations received negative feedback from the CF community patient groups and clinicians.
The Vanstones, who are from Bradford, and Simcoe-Grey MPP Jim WIlson, met with Minister of Health, Christine Elliott last month to discuss the negative impacts of adopting the recommendations from CADTH.
The trio first met with Elliott nine years ago when they convinced the Ontario government to approve coverage for CF drug Kalyedco, after a lengthy public battle. Elliott was the provincial health critic at the time.
Had Kalydeco not been funded by OHIP, the Vanstones would need to pay $350,000 yearly for the drug. Even with private insurance and participation in a drug study, they were looking at a cost of about $60,000 per year.
Elliott said the move is "life-changing" for CF patients.
“Our government has taken urgent action to ensure all cystic fibrosis patients will have more timely access to the effective and life-changing treatments they need,” Elliott said.
“Providing coverage for Trikafta is one more way our government is building a sustainable, modern, and connected health care system that will expand coverage to new and innovative treatments and provide high-quality health care to patients for years to come.”
Madi Vanstone, 19, will now be able to transition from her current CF drug Kalydeco medication to Trikafta, as her condition has worsened over the past year and her doctors are recommending the switch.
"Trikafta is the single greatest innovation in cystic fibrosis history and it has the power to transform the lives of thousands of Canadians," said Kelly Grover, president and CEO of Cystic Fibrosis Canada. "The cystic fibrosis community in Ontario has fought long and hard to get this drug into their hands. Access to Trikafta will mean longer and healthier lives for so many people and the ability to plan for a future that many feared they might not live to see."
According to CF Canada, it is estimated that one in every 3,600 children born in Canada has CF. More than 4,300 Canadian children, adolescents, and adults with cystic fibrosis attend specialized CF clinics.
