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Cystic fibrosis patients upset over recommended restrictive criteria for life-saving drug

'By narrowly defining accessibility and continuation criteria CADTH’s recommendation overlooks many of Trikafta’s significant benefits,' says chief scientific officer for CF Canada
2021-02-05 Beth Vanstone crop
Beth Vanstone is shown in a supplied photo.

Last month the Cystic Fibrosis (CF) community was relieved and overjoyed when Health Canada announced its approval for the life-saving drug TRIKAFTA for those aged 12 and up with CF and at least one F508del mutation.

But their excitement was short-lived after learning of CADTH's (Canadian Agency for Drugs and Technologies in Health) draft review released earlier this week. The review outlined strict criteria recommendations for public funding to gain access to the drug. 

CADTH is an independent, not-for-profit organization that provides research and analysis to healthcare decision-makers. 

According to a press release from Cystic Fibrosis Canada, if the recommendations are adopted by the provinces, it could effectively exclude over 25 percent of eligible Canadians with CF.

One of the recommendations is to make funding available only to those with below 90 percent lung function. 

"This of course totally disregards the fact that the disease affects every organ in the body," points out CF Advocate and leader for CF Get Loud, Beth Vanstone. 

To remain on the drug and have it funded, CADTH is recommending that patients must gain five percent lung function in the first six months of use in order for the prescription to be renewed and to maintain the five percent indefinitely.

Vanstone says this is problematic, as most healthy adults' lung function naturally declines with aging, like hearing and eyesight.

"So if you are fortunate enough to have access to this lifesaving drug you are essentially not granted the normal aging process," said Vanstone. "It is very short-sighted and discriminatory."

"CF survival has dramatically improved over the years precisely because an aggressive regimen of therapy is initiated immediately on diagnosis. Why is TRIKAFTA different? By narrowly defining accessibility and continuation criteria CADTH’s recommendation overlooks many of TRIKAFTA's significant benefits.” said Dr. John Wallenburg, chief scientific officer, Cystic Fibrosis Canada.

He warned, “If adopted by the provinces, CADTH’s draft recommendation will create an absurd situation in which patients will need to become  demonstrably sicker to get this medicine, and those who receive the drug and experience important clinical benefits could see it pulled right out from under their feet.”  

CADTH says their drug reimbursement recommendations are developed in accordance with a robust and publicly available process, and patient groups provide essential input at different stages of the process.

"In particular, the stakeholder feedback period for draft recommendations is an essential component of the review process because it allows patient groups and clinician groups to comment on draft recommendations before they are finalized," said Andrea Tiwari, Marketing and Communications, CADTH. 

For the review, three patient groups and three clinician groups provided input. Tiwari says CADTH also convened with a panel of CF specialists to advise on TRIKAFTA's place in therapy and advise on the evidence's appraisal and interpretation. Additionally, participating public drug plans identified potential implementation issues during the review and deliberations.

"As our review of TRIKAFTA is ongoing, we can’t comment on stakeholder feedback until we formally receive the input and the expert committee has the opportunity to review the feedback during its deliberations, should a request for reconsideration come forward," said Tiwari. 

A spokesperson for Vertex, the company that manufactures the TRIKAFTA drug, thanked CADTH for their collaboration on the process, through aligning the timing of their review with Health Canada, collaborating with INESSS, and including the opinions of a clinical panel in the recommendation.

"While the Canadian Drug Expert Committee (CDEC) issued a positive recommendation to list TRIKAFTA, Vertex will be requesting CADTH reconsider the initiation and continuation criteria that were recommended," the Vertex spokesperson confirmed. 

Cystic Fibrosis Canada is also working with CF clinicians to have CADTH to expand its criteria recommendations. 

“CADTH’s recommendations are not aligned with the recommendations of Canada’s CF physicians, who almost universally disagree with the limitations CADTH has placed on access and continuation,” said Dr. Wallenburg. “We are counting on the provinces to do the right thing and save lives by immediately funding Trikafta for all Health Canada approved indications."

He added, "Canadians with CF have one of the highest rates of survival in the world, a testament to the quality of Canada’s CF physicians, the real experts in this rare disease. Let’s ensure they aren’t handcuffed by these shortsighted recommendations and that they are the ones making the important clinical decision of who should access this life-changing drug.”  

Patient groups have until July 22 to submit their feedback to CADTH on the recommendations. 

"CF Get Loud will be responding. We are collecting stories from patients to say how they are affected," said Vanstone. 

What frustrates Vanstone most is that CADTH has been involved in the CF modulator review process for over a decade and is familiar with how the disease affects the body. She wondered why such strict criteria were set for TRIKAFTA. 

"When the recommendation was going in, they (CADTH) ask for patient input and hundreds of people submitted stories. They have all the information to make educated decisions, and yet they disregarded all of the things that affect [patients]. We aren't happy with it," said Vanstone. "It's very disappointing to see this."

Vanstone has been in touch with MP for Simcoe-Grey Jim Wilson, who has been advocating for the CF Community for many years and has secured a meeting with Minister of Health Christine Elliott to discuss the possibility of funding without adopting the recommendations from CADTH. 

Elliott was health critic at the time when Vanstone and her daughter Madi convinced then-premier Kathleen Wynne and the Ontario government to approve coverage for her life-saving CF drug, Kalydeco. 

"We've been in this fight a long time and I am really hoping she has the understanding and the compassion to see past this very restrictive criteria and lead Canada by showing Ontario is siding with CF patients to gain access," said Vanstone. "That's my hope."

To view CADTH's draft recommendation, click here