Public Reimbursement of Trikafta Nears in Canada

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by Steve Bryson, PhD |

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Vertex Pharmaceuticals has signed a letter of intent with the pan-Canadian Pharmaceutical Alliance (pCPA), which is an agreement in principle to extend public reimbursement to Trikafta, a triple-combination therapy for eligible cystic fibrosis (CF) patients.

The extension adds to the letter of intent (LOI) with the pCPA for the cystic fibrosis treatments Orkambi and Kalydeco, also marketed by Vertex.

The pCPA negotiates prices of medications for the country’s provinces and territories, which control and manage their own public health programs. Following successful negotiations, an LOI is created, which lists the terms and conditions for funding the therapies.

“This is a significant milestone for patients with CF in Canada,” Duncan McKechnie, senior vice president, North America Commercial Operations at Vertex, said in a press release. “We would like to thank the pCPA and the participating jurisdictions for their collaborative approach.”

“We share the urgency of the CF community to bring this process to a successful conclusion, and we will continue our work with all the provinces and territories so that eligible people with CF have the opportunity to receive TRIKAFTA, KALYDECO and ORKAMBI,” he added.

The LOI extension comes on the heels of a positive clinical recommendation for Trikafta from the Canadian Agency for Drugs and Technology in Health (CADTH). This independent body relays evidence to Canada’s healthcare decision-makers to help them make informed judgments about treatments and medical devices.

The move also was supported by l’Institut national d’excellence en santé et en services sociaux (INESSS) in Quebec, the province’s own regulatory review agency.

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Trikafta was approved in June for Canadian CF patients 12 and older who carry at least one F508del gene mutation, the most common disease-causing mutation. The oral therapy is a combination of three CFTR modulators (elexacaftor, tezacaftor, and ivacaftor) designed to increase the quantity and enhance the function of the F508del-CFTR protein at the cell surface.

In July, the CADTH provided recommendations for the use of Trikafta, which included restricting the therapy to patients whose lung function was below 90% predicted capacity. Patients need to demonstrate a 5% sustained lung function improvement to continue treatment.

Also, in July Cystic Fibrosis Canada (CFC) published standardized care guidelines to provide CF clinicians in Canada with information regarding the use of modulator therapies. In the guidelines, there were no minimum or maximum lung function restrictions. The organization called on provinces and territories across Canada to adopt the latest guidelines, which were endorsed by CF doctors nationwide, they said.

More recently, INESSS provided recommendations that mirrored the same lung function restrictions as those made by the CADTH, which the CFC called “short-sighted.”

“INESSS has missed the opportunity to lead the country in recommending to fund this breakthrough drug for ALL residents who can benefit, and instead has repeated the same mistakes as CADTH, ignoring a key element of the guidelines developed by Canadian cystic fibrosis clinicians,” said John Wallenburg, PhD, chief scientific officer of CFC, in a separate press release. “Cystic fibrosis is a complex multi organ disease. The best use of healthcare dollars in all instances is in preventing disease.”

“In fact, cystic fibrosis survival has dramatically improved over the years precisely because an aggressive regimen of therapy is initiated immediately on diagnosis, to delay the onset of symptoms,” he added. “If approved by the Minister of Health, the INESSS recommendation will create a cruel situation in which children and young adults will need to become demonstrably sicker to get this medicine.”

The CFC has asked the Health Minister of Quebec to trust the expertise of CF clinicians and prescribe Trikafta in agreement with their professional opinion. Along with two well-known CF clinicians, CFC recently shared an open letter conveying its concerns about the restricted criteria.

“This recommendation is not aligned with the recommendations of Canada’s CF clinicians, who almost universally disagree with the limitation both CADTH and INESSS have placed on access,” said Wallenburg. “We are counting on the Minister to do the right thing and save lives by immediately funding Trikafta for all Health Canada approved indications.”

“Canadians with cystic fibrosis have one of the highest rates of survival in the world, a testament to the quality of Canada’s CF clinicians, the real experts in this rare disease,” he added. “Let’s ensure they are the ones making the important clinical decision of who should access this life-changing drug.”