Health Canada approval of transformational cystic fibrosis medication brings hope to CF community
pan-Canadian Pharmaceutical Alliance (pCPA) says yes for Kalydeco and Orkambi
TORONTO, June 18th, 2021 – The cystic fibrosis community is closer to its goal today as Health Canada announced the approval of Trikafta for sale in Canada, issuing a Notice of Compliance (NOC). Considered the single greatest innovation in the history of cystic fibrosis, Trikafta is a transformational drug that can change the lives of up to 90% of Canadians with cystic fibrosis.
Today’s announcement means that doctors can now prescribe Trikafta for people aged 12 and up with cystic fibrosis and at least one F508del mutation. However, before the drug is accessible to those who need it, public or private insurers need to agree to pay for it.
This news was quickly followed up with an announcement by the pan-Canadian Pharmaceutical Alliance (pCPA), a regulatory body that negotiates drug prices on behalf of the provinces, that they have agreed on a negotiated price for the CF drugs Orkambi and Kalydeco for all Health Canada approved mutations. The pCPA noted that Trikafta may be added to these agreements, pending a positive regulatory and Health Technology Assessment recommendation.
“This is a special day for our community. Together we have been fighting hard for access to these medications. Thousands of letters and signatures and hundreds of meetings later, we are finally seeing the progress our community deserves,” said Kelly Grover, President and CEO of Cystic Fibrosis Canada. “We turn to the provinces next. They must immediately fund Kalydeco and Orkambi, which have been in negotiations for more than a year, and fund Trikafta as soon as possible. Provinces, end the wait and save lives.”
A “Notice of Compliance” by Health Canada is one step in Canada’s complex drug approval and reimbursement process. There will be several other reviews and approvals before Trikafta is publicly funded and accessible; some of these are already underway. Its cost effectiveness is still being considered by Canada’s health technology assessment bodies, Canadian Agency for Drugs and Technologies in Health (CADTH), the Institut national d'excellence en santé et services sociaux (INESSS) and BC Pharmacare. They will make non-binding recommendations on whether their respective governments should pay for the drug. Finally, Canada’s public drug programs must negotiate the price of the drug through the pan-Canadian Pharmaceutical Alliance (pCPA), after which the provinces will decide if they will fund the medication.
Canadian research published in the Journal of Cystic Fibrosis demonstrated that access to Trikafta in 2021 would result in profound health benefits for Canadians living with cystic fibrosis. By 2030, Trikafta could reduce the number of people living with severe lung disease by 60% and reduce the number of deaths by 15%. In addition, the study estimated that the median age of survival for a child born with cystic fibrosis would increase by 9.2 years.
Given these dramatically positive impacts, all public healthcare systems should cover the cost of Trikafta for every person eligible to receive it. The drug has already received regulatory approval in almost 40 countries. It was designated a breakthrough medication and approved by the U.S. Federal Drug Agency in October 2019, a year and a half before Health Canada approval. Recognizing Trikafta as a true game-changer, England’s National Health Service agreed to fund it even before regulatory approval.
“People living with cystic fibrosis need these drugs, and they need them now,” said Grover. “No Canadian should have to fight this long and this hard for the medications they need. People with CF can’t wait. It’s literally a matter of life and death.”
The cystic fibrosis community has fought tenaciously for this moment, but our work is not done. Members of the community and supporters are invited to join a Facebook Live Rally for Trikafta on June 21 at 6pm EST on Cystic Fibrosis Canada’s Facebook page. The event, hosted in English, will bring the community together to discuss Health Canada’s decision, pCPA’s announcement and hear about next steps to gaining access to life-changing cystic fibrosis medications.
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About cystic fibrosis
Cystic fibrosis is the most common fatal genetic disease affecting 4,344 Canadian children and young adults. There is no cure. Of the Canadians with cystic fibrosis who died in the past three years, half were under the age of 34. 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 4,344 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, please contact:
Jennifer Ouellette, Manager
Nicole Young, Director
Tel: (905) 317-5529