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CF Clinicians Speak Out Against Government Recommendations That Could Restrict Access to Miracle Drug Trikafta
August 12, 2021

CF Canada and two prominent CF clinicians share an open letter directed at all provincial health ministers and premiers, voicing concerns with the overly restrictive criteria recommended for public coverage of Trikafta.


Dear Premiers and Ministers of Health,

We are writing on behalf of physicians that specialize in treating cystic fibrosis and who conduct research in cystic fibrosis, including clinical trials.

Cystic fibrosis (CF) is the most common fatal genetic disease affecting Canadian children and young adults. There is no cure. CF has various effects on the body, but mainly affects the digestive system and lungs. The degree of CF severity differs from person to person, however, because of the destruction of lungs and loss of lung function, CF is a fatal disease for most patients.

We are writing to you today because we believe you have received incorrect and misleading advice with respect to a new treatment for CF. A recent draft recommendation from CADTH, an agency responsible for advising provincial governments about which drugs should be paid for by our public health care system, suggests that some CF patients should not have access to a life-changing new drug. Trikafta is a break-through medicine that could transform the lives of 90% of Canadians living with CF and dramatically reduce deaths and hospitalizations according to recent studies and real-world evidence.

Our advice as the clinicians responsible for the medical care of CF patients, and as experts on the treatment of CF, is that this drug must 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 across Canada, lead to unjust and unintended consequences for patients, and leave many CF patients without the treatment needed to improve or extend their lives.

More specifically, we wanted to provide you with the information and context that is missing for several elements of the CADTH draft recommendation regarding the threshold for eligibility and the requirements for ongoing treatment.

Threshold for initial eligibility

In its most simplified form, the CADTH draft recommendation specifies that patients would only be eligible for Trikafta if their lung function is less than 90% of predicted. There are several problems with this approach.

This threshold for lung function implies that the only impact of CF is on the lungs. This is simply incorrect. CF is a disease that affects many organs in the body, and as such, lung function is not the only test for determining lung health nor overall health in persons with CF. To narrow eligibility by assessing impact on only one organ is simply clinically inappropriate.

There is also evidence showing that Trikafta improves lung function for patients with greater than 90% lung function, making this an arbitrary threshold. What’s more, 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.

It should also be noted that this threshold, which was not dictated by any clinical rationale, also 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 the 90% threshold. Denying access to this therapy 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 where young people are making decisions about education, career, and relationships, all of which are affected by having a chronic disease and will have repercussions on their adult lives.

Finally, the stipulation that patients should be stable, not be hospitalized or receiving antibiotic treatment before receiving Trikafta may be unnecessarily restrictive for patients with severe lung disease. While this draft recommendation may not be a problem for initiating therapy in patients with mild and moderate lung disease, some persons with CF with severe lung disease may require very frequent antibiotic treatments or may have prolonged hospitalizations. Trying to find a window to start treatment with Trikafta may unnecessarily delay initiating therapy, thus delaying the potential clinical benefit of the drug, and ultimately may cost some patients their lives. It is not clinically reasonable or potentially ethical to withhold treatment from these patients.

Clinical benefit requirement for ongoing eligibility

The CADTH draft recommendation also requires an annual assessment of the patient and a sustained improvement of lung function of 5% in order to continue receiving the drug. This threshold for clinical improvement in lung function is arbitrary and not based on clinical evidence, or an understanding of how this disease progresses or evolves over time. It also overlooks other clinical benefits that are as important to disease management and quality of life for CF patients.

As stated above, lung function is only one measure of disease severity and response to therapy in CF. The proposed lung function improvement threshold does not take into account other CF related outcomes such as pulmonary exacerbation that can have a more important effect on health status and quality of life in persons with CF and ignores evidence that shows other factors, like aging, will impact lung function and therefore eligibility.

There is also no guidance for when a patient who has had their treatment withdrawn for failing to maintain a 5% improvement could be reconsidered for restarting therapy after a further decline in lung function or health. Nor do we know the potential adverse effects of stopping therapy with Trikafta. This creates a dangerous situation where doctors must start and stop treatment of patients without understanding the short- and long-term consequences of doing so.

It should also be pointed out that if we wait to begin treatment until a patient has a lung function of less than 90% predicted, many patients will become ineligible for the drug after several years, because of complications stemming from delayed treatment. That is to say, there is a real possibility that the damage created by delaying treatment will mean it is harder for patients to maintain a long-term 5% lung function improvement required for ongoing eligibility for Trikafta. The harm to the patient created by delaying treatment will be compounded as it jeopardizes the patient’s prospects for ongoing eligibility and benefit from the medication. The first wrong will create the second wrong. This represents a moral and ethical dilemma for physicians who treat CF patients and an unacceptable situation for patients.

Furthermore, the stress and anxiety imposed by each annual renewal visit will be excessive as our patients will feel like their life is on the line. This may lead to several mental health crises in a population with very high rates of anxiety and depression to begin with, particularly if they just fall short of the 5% threshold due to circumstances that may be outside of their control.

Beyond the unintended consequences of discrimination against younger individuals with CF (addressed above), and the impact on people’s mental health, these draft recommendations are likely to put people living with CF at risk for significant harm or self-sabotage so that they can meet eligibility criteria, and will mean clinical deterioration if therapy is withdrawn.

In summary, while we agree with some of the conditions proposed in the CADTH reimbursement draft recommendations, there are several elements that are scientifically flawed and have the potential to cause harm. We strongly encourage you to seek the advice of CF clinicians on eligibility to ensure that CF patients have fair access to this potentially life saving therapy.

Sincerely,

Bradley Quon, MD, FRCPC, MSc, MBA
Medical Lead, CF Canada Accelerating Clinical Trials Network (CF CanACT)
CF Physician, St. Paul’s Hospital Adult CF Clinic
Associate Professor of Medicine, University of British Columbia
Michael Smith Foundation for Health Research Scholar

Martha L. McKinney, MD MPH FRCPC
CF Physician, Edmonton Pediatric Cystic Fibrosis Clinic, Stollery Children’s Hospital
Member of the Executive Committee, CF Canada Accelerating Clinical Trials Network (CF CanACT)
Clinical Lecturer, Department of Pediatrics, University of Alberta


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