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Volunteer Advocate


Letter to the Honourable François Legault and Christian Dube
September 9, 2021

The following letter was sent by Cystic Fibrosis Canada to The Honourable François Legault and Christian Dubé on Tuesday, September 7th 2021. 

Re: INESSS recommendation for Trikafta

We are writing on behalf of physicians that specialize in treating cystic fibrosis. 

Cystic fibrosis (CF) is a common fatal genetic disease affecting >1300 Quebec children and young adults. There is no cure.  CF has various effects on the body, but mainly impacts the digestive system and lungs. The degree of CF severity differs from person to person; however, because of the destruction of the lungs and loss of lung function, lung transplantation is required at a young age (<35 years)

A life-changing drug, Trikafta, markedly improves the function of CFTR, the protein which, when defective, causes the manifestations of CF. INESSS has recommended that access to Trikafta be limited to only those patients with lung function <90%. We strongly disagree with this opinion and want to share with you our reasoning. 

CF lung disease starts in infancy. Even with newborn screening, therapy that does not include CFTR modulators like Trikafta does not alter the progression of lung disease. The studies of Trikafta in those 12 years of age and older demonstrated changes in lung function using spirometry, which is insensitive to changes in lung structure. Lung damage readily progresses but is not easily detected by spirometry until quite advanced. A lung function value of >90% is a false assurance that the lungs are not damaged. Waiting until a patient has less than 90% lung function means there will be structural and often irreversible lung damage before treatment can begin and ignores the fact that the best treatment for CF is preventing the disease from progressing and improving overall health outcomes, including pulmonary exacerbations, quality of life, treatment burden, and mental health. In a study modelling expected outcomes following the introduction of Trikafta in Canada, widespread access to this medication would improve the median age of survival by almost a decade if made available today.   

While the studies in those 12 and above were limited to those with lung function <90%, subsequent studies in children 6-11 years of age demonstrated that those with lung function >90% show substantial improvement despite their more ‘normal’ values. Limiting access to Trikafta to those with lung function <90% unjustly discriminates against children and adolescents with CF, since they are much more likely to have lung function above the 90% threshold.  Thus, many adolescents may not be able to benefit from this therapy until their lung damage is severe enough to drop below this arbitrary 90% threshold.  Denying access to Trikafta to these young patients is particularly problematic given that it is a crucial period of development, both physically and otherwise, for young people with chronic disease. This is also a period of increased risk in the decline in lung function, particularly in young women with CF, and of development of other important complications of cystic fibrosis including CF related diabetes. It is also a period during which young people are making decisions about education, career, and relationships, all of which are affected by the state of their health will have repercussions on their adult lives.  

INESSS was informed by their clinical advisors that there will be inequalities between children and adolescents covered by RAMQ, compared to children and adolescents with the same level of disease but covered by private insurance as these companies cover these medications much more readily than the public coverage. This will lead to a situation where patients with private insurance will be much better covered earlier in their disease process and will have better outcomes. 

Our advice on behalf of the clinicians responsible for the medical care of CF patients, and as experts on the treatment of CF, is that Trikfata be made available to all those patients that would benefit from it.  To do otherwise would create real and significant moral and ethical challenges for physicians, lead to unjust and unintended consequences for patients, and leave many CF patients without the treatment needed to improve or extend their lives.


Larry C. Lands, MD, PhD

Director, Pediatric Cystic Fibrosis Clinic, McGill University Health Centre

Consultant Respirologist, Rouyn-Noranda Cystic Fibrosis Clinic

On behalf of the Cystic Fibrosis Clinics Directors in Quebec


Dr. John Wallenburg

Chief Scientific Officer

Cystic Fibrosis Canada

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