CF Canada Urges Adoption of New Care Guidelines, Access to Trikafta
Cystic Fibrosis Canada (CFC) is calling on provinces and territories across the country to adopt the latest standardized care guidelines and speed patient access to Trikafta, a triple-combination and “highly impactful” disease-modifying therapy for almost all with cystic fibrosis (CF).
The guidelines, “Initiation, Monitoring and Discontinuation of CFTR Modulator Therapies for Patients with Cystic Fibrosis,” were an initiative of Cystic Fibrosis Canada’s healthcare advisory council to provide CF clinicians across Canada with key information regarding the best-practice use of modulator therapies.
With their publication, the organization is urging local governments to immediately fund Trikafta and other modulators, which include Orkambi, Kalydeco, and Symdeko, through their respective public health plans and to follow other guideline standards. All these therapies are marketed by Vertex Pharmaceuticals.
According to a CFC press release, the guidelines have been endorsed by CF doctors nationwide.
“The healthcare advisory council identified and appointed a working group of healthcare professionals to develop these guidelines,” said Ian McIntosh, the organization’s director of healthcare. “These clinicians and physicians have been involved in clinical trials or compassionate use of modulators, and as such, these are the medical professionals who have the inside knowledge and experience, specifically with modulators, and who know best.”
If adopted, the new guidelines are expected to help CF patients in Canada gain access to the best modulator for them. They also are aimed at patient monitoring, so that those who benefit from modulators will continue to do so over time.
Trikafta is an oral combination of three CFTR modulators (elexacaftor, tezacaftor, and ivacaftor), approved in June for patients in Canada, ages 12 and up, who have at least one F508del mutation, the most common CF-causing mutation. The favorable Health Canada decision was expected to open access for nearly 1,100 Canadian residents to a first treatment targeting their disease’s underlying mechanisms, Vertex reported at the time.
According to one Canadian data study, CF-related deaths with Trikafta’s availability could fall by 15% in Canada over the next decade.
After the approval announcement, the pan-Canadian Pharmaceutical Alliance, a regulatory body, reached a negotiated price for two other Vertex modulator therapies — Orkambi and Kalydeco — covering people with Health Canada-approved and treatment-relevant CF mutations. The alliance negotiates medication prices for the country’s provinces and territories, which operate their own public health programs.
Still, other steps are necessary before Trikafta is publicly funded and accessible. As with the other modulators, provinces must first fund the treatment.
The Canadian Agency for Drugs and Technology in Health (CADTH) also issued a July recommendation regarding Trikafta that, due to what the CFC calls “overly restrictive criteria,” would cut patient access by more than 25%. CADTH is an independent organization responsible for providing Canada’s healthcare decision-makers with evidence to help make informed decisions about the optimal use of treatments and devices.
Meanwhile, CF Canada has provided CADTH with feedback, and Vertex has asked that CADTH reconsider its draft recommendations, a process that could take several months.
CF Canada and two prominent CF clinicians recently shared an open letter — directed at provinces and and territories — expressing concerns about the criteria. Among them was a limit on Trikafta eligibility to patients whose lung function was under 90% of predicted capacity.
“To narrow eligibility by assessing impact on only one organ is simply clinically inappropriate,” the letter stated, as it overlooks how CF affects other organs.
Decision-makers also not consider evidence that this therapy may improve lung function in people with more than 90% lung function. By waiting for patients to fall below this threshold before starting on Trikafta, the limitation risks lung damage that will be structural and possibly irreversible, its authors wrote.
“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,” they added.
An additional reason for concern is that patients need to show sustained lung function improvement of 5% to continue using Trikafta. “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,” the letter stated.
“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,” the experts continued. “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.”
Use of Trikafta, initially approved in the U.S. for patients 12 and older with one F508del mutation, was recently expanded to cover eligible children starting at 6 years old. It entered the U.S. market in 2019 with an annual list price of $311,000.
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