A drug that is being hailed as a ‘miracle’ and ‘life-changing’ by cystic fibrosis (CF) patients, advocates and doctors has yet to be funded in Manitoba and the national organization that informs how provinces fund medications is recommending limitations on who would be eligible.
Every morning, Nick McDonald wakes up early for CF treatment. The disease has impacted his lungs, his breathing and his digestive system.
“Every meal that I eat, I have to take five pills in order to properly digest my food,” McDonald said.
That’s just a small portion of what he does to try to keep his cystic fibrosis under control on a daily basis.
But the 26-year-old is desperate to get his hands on Trikafta, a newly approved drug in Canada that he said would alter his life.
“It would mean a lot,” he said.
“It would probably extend my life by several years.”
“It would decrease the amount of treatment I have to do, most likely I would be able to stop taking so many courses of antibiotics. I would just feel healthy,” he said.
But for many, like McDonald, the cost of the drug is prohibitive.
The list price for Trikafta in the U.S. is $310,000, meaning paying to fund the drug for one patient in Canada could be more than $400,000 a year.
That price is significantly reduced when it goes through a provincial program, as they negotiate prices down. But without those programs adding the drug to an approved list, individuals would be left to pay the six-figure sticker price themselves.
Manitoba has yet to add Trikafta to its coverage.
“Really we need it to be funded by the province so that everyone can have access to it without going deeply into debt,” McDonald said.
Health Canada approved Trikafta for people with cystic fibrosis aged 12 and up with at least one F508del mutation.
“The drug works on one particular mutation, in Canada that’s about 90 per cent of the cystic fibrosis population. So it’s pretty significant,” Cystic Fibrosis Canada Chief Scientific Officer Dr. John Wallenburg said.
How it works
Cystic fibrosis is caused by mutations in a specific gene, which create a faulty protein. It impacts people’s lungs, digestive systems and can cause uncontrollable coughing.
“It’s not necessarily the defective protein itself (that’s the problem). It’s the downstream consequences that are so harmful. It’s the symptoms that this leads to,” Wallenburg said.
Rather than just treating symptoms, Trikafta targets the basic defect from one specific genetic mutation that causes CF.
“What’s different about this drug is that (it) goes in and it fixes that broken protein.”
“And so what it’s doing, it’s correcting the biological basis of the problem before the symptoms that are downstream from the problem,” Wallenburg said.
There is no cure for CF, but advocates said Trifakta is as close to one as possible, for now.
“Trikafta is actually a miracle drug,” lead provincial advocate of Cystic Fibrosis Canada Patti Tweed said.
On Thursday, the Canadian Agency for Drugs and Technologies in Health, the body that reviews the cost-effectiveness of drugs on behalf of the public payers, recommended listing this drug.
However, it also recommended restricting coverage to only those with a lung function of 90 per cent or less.
“The way it stands right now with the restrictions, it would eliminate 27 per cent of the population in Canada from actually benefiting,” Tweed said. “And that’s just not acceptable.”
This means CF patients with healthier lungs will be forced to wait for their health to decline before being able to access a drug that could help prevent further disease progression.
“So what that does essentially is eliminate mostly children and young adults who have been doing their very best, by all means possible, to keep their lung function up and to not deteriorate,” Tweed said. ” So we certainly don’t want to have our people become sicker in order to be able to get access to this drug.”
The province says the pan-Canadian Pharmaceutical Alliance (pCPA) reached a mutual agreement on terms (pricing and conditions of coverage) earlier this week.
While the role of the pCPA is to conduct joint provincial, territorial and federal drug plan negotiations for brand name and generic drugs, any final drug funding decision remains under the authority of individual jurisdictional public drug plans.
“Manitoba will now work through our respective processes to make the decision to list Trikafta on the Pharmacare public drug plan,” a provincial spokesperson said.
However, the province did not say when that decision would be made.
“Manitoba realizes the COVID-19 pandemic has presented challenging times for cystic fibrosis patients and their caregivers and will be working as quickly as possible on this file.”