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CADTH paves way for many Canadian children and adults with cystic fibrosis to access life changing drug - Trikafta
May 19, 2022

TORONTO, May 19, 2022 Today, the Canadian Agency for Drugs and Technologies in Health (CADTH) provided hope for many Canadians living with cystic fibrosis (CF), issuing a positive draft recommendation to fund the transformational cystic fibrosis drug, Trikafta, for Canadians aged 6 and older with at least one F508del gene mutation, without undue restrictions. This follows the recommendation that the review body provided in 2021 for the 12 years of age and older group that included an overly restrictive access criterion that has now been removed. Cystic Fibrosis Canada now calls upon public and private payers to fund Trikafta for all who could benefit following CADTH’s final recommendation.   

This new draft recommendation for those six years of age or older has no restrictive lung start criterion which is in contrast to CADTH’s 2021 recommendation to implement a restrictive 90% FEV1 or less (a measurement of lung function) requirement to start Trikafta. In 2021, four provinces recognized that limiting access based on this clinical criterion was not in the best interest of people diagnosed with cystic fibrosis and funded the drug based on the guidelines produced by Canadian CF physicians. Cystic Fibrosis Canada is calling on all jurisdictions and private payers to remove the absurd lung function criterion for those 12 and up and be prepared to fund the drug for all eligible Canadians upon release of the final CADTH recommendation due in June. 

Today’s news offers hope of a brighter future for many Canadian families. We are relieved that CADTH recognized the true value of this drug to the health of people diagnosed with cystic fibrosis in Canada and aligned their recommendation with the advice of Canadian clinical cystic fibrosis experts. This recommendation puts us on a path to public reimbursement, although there is still a long road before Canadian children and previously excluded adults with cystic fibrosis can access this drug. ,” said Kelly Grover, President and CEO of Cystic Fibrosis Canada. “We are urging public and private funders to list Trikafta for 6-11 year olds and to remove any unnecessary restrictive criteria for those aged 12 and older. Last fall, provinces and territories moved quickly to cover this drug. There is every reason to act just as quickly to make Trikafta available to children and adults now eligible.” 

Trikafta is considered the single greatest innovation in the history of cystic fibrosis. It can treat up to 90% of Canadians with cystic fibrosis and addresses the underlying causes of the disease instead of just managing the symptoms, potentially preventing irreversible damage caused by this progressive disease. The Health Canada approval of Trikafta for 6-11 year olds provided an opportunity for the manufacturer to submit to CADTH additional evidence that Trikafta is effective regardless of lung function. CADTH accepted the opportunity to provide updated guidance for all eligible patients aged 6+. Today's milestone draft recommendation from CADTH aligns more closely to the clinical guidelines developed by Canada's cystic fibrosis specialists and brings Canadian children with cystic fibrosis one step closer to accessing this life changing drug.   

“My daughter Maysa has suffered from cystic fibrosis her entire life,” said Sarah Milligan, BC resident and mother to ten-year-old Maysa, who lives with CF. “Trikafta is our greatest hope for Maysa’s future. We are thrilled to know that this recommendation brings her one step closer to accessing a drug that could change her life – hours spent on treatments could be hours spent with her friends, and enjoying her childhood, something Maysa has not been able to fully do, yet.” 

In October 2021, Trikafta was accepted for a priority review by Health Canada and for an aligned review with health technology assessment (HTA) bodies, CADTH and Quebec's Institut national d'excellence en santé et en services sociaux (INESSS). An aligned review allows the two HTA bodies to complete their respective reviews during Health Canada’s priority review period. Health Canada approved Trikafta for children aged six to 11 with at least one F508del gene mutation on April 20, 2022.  

Unlike CADTH, INESSS’s decision-making process and drug review timelines are not published. As a result of the pandemic, the province has a waiting list for drug reviews. In a recent meeting, INESSS shared with Cystic Fibrosis Canada that the drug is now under review and if all goes well, the recommendation will be provided to the minister by fall. The minister has 30 days to respond to INESSS recommendations. Quebec has a provincial election slated for October 3, which could delay the listing of Trikafta if the minister does not receive and review the recommendation in time. Cystic Fibrosis Canada will continue to monitor the situation in Quebec because Quebecers with CF should not be left behind.   

Patient groups, clinicians and researchers now have the opportunity to provide feedback to CADTH on the draft recommendation until June 3, 2022. Cystic Fibrosis Canada will take the opportunity to provide feedback to CADTH, informed by the Canadian cystic fibrosis community. CADTH will issue a final recommendation on Trikafta after all revision requests and feedback have been considered. 

Join our Facebook Live event 

Members of the community and supporters are invited to join a Facebook Live for #FurtherTogether on Thursday, May 19th at 7:30 p.m. ET on the Cystic Fibrosis Canada Facebook page. Hosted in English, the event will bring the community together to discuss the draft recommendation from CADTH, and next steps to gaining access to life-changing cystic fibrosis medications. 

Go Further this CF Awareness month 

The disease in Canada is on the cusp of incredible change for many Canadians with cystic fibrosis, but the CF community must act now to ensure all Canadians impacted by the disease have access to the drugs they need. You can help by joining Cystic Fibrosis Canada’s  ‘#FurtherTogether’ campaign. Take one or all four actions to help make an impact for people living with cystic fibrosis. Walk. Write. Meet. Donate. There is an action for everyone, and people can push further by taking multiple actions. 

No matter how you choose to get involved, you can help protect Canadian children from the cumulative and irreversible damage that happens when growing up with cystic fibrosis. And help to lessen the decline in health of so many others living with cystic fibrosis. 

About cystic fibrosis 

Cystic fibrosis is the most common fatal genetic disease affecting 4,332 Canadian children and young adults. There is no cure. Of the Canadians with cystic fibrosis who died in the past five years, half were under the age of 37. Cystic fibrosis is a progressive, degenerative multi-system disease that affects mainly the lungs and digestive system. In the lungs, where the effects are most devastating, a build-up of thick mucus causes severe respiratory problems. Mucus and protein also build up in the digestive tract, making it difficult to digest and absorb nutrients from food. In addition to the physical effects of the disease, mental health concerns are emerging; anxiety and depression are common among this population. Double lung transplants are the final option for patients with end-stage disease; most fatalities of people with CF are due to lung disease. 

About Cystic Fibrosis Canada 

Cystic Fibrosis Canada has dramatically changed the cystic fibrosis story. We have advanced research and care that has more than doubled life expectancy. Since being founded by parents in 1960, Cystic Fibrosis Canada has grown into a leading organization with a central role engaging people living with cystic fibrosis, parents and caregivers, volunteers, researchers and healthcare professionals, government and donors. We work together to change lives for the 4,332 Canadian children and adults living with cystic fibrosis through treatments, research, information and support. Despite our remarkable progress together, we are not yet done. Not when half of the Canadians with cystic fibrosis who died in the past three years were under the age of 34. We will keep pushing, keep going further until all people with cystic fibrosis can and do experience everything life has to offer —and enjoy everything life has to offer. Learn more at cysticfibrosis.ca. 

For more information:

Nicole Young, Director, Marketing and Communications
Tel: (905) 317-5529
Email:nyoung@cysticfibrosis.ca


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