Patients with cystic fibrosis, supporters push Manitoba to fund new “miracle” drugs-Winnipeg

Drugs that are welcomed as “miracles” and “life-changing” Cystic fibrosis (CF) Patients, advocates, and doctors have not yet been funded in Manitoba, and a national organization that informs how the state funds medicines recommends limiting qualified individuals.

Every morning, Nick McDonald wakes up early for CF treatment. The disease affects his lungs, breathing and digestive system.

“Every time you eat, you need to take 5 tablets to properly digest your food,” McDonald’s said.

This is just part of what he does to manage cystic fibrosis on a daily basis.

But the 26-year-old was desperate to get Trikafta, a newly approved drug in Canada, and said he would change his life.

“That would mean a lot,” he said.

“It will probably prolong my life for a few years.”

“It will reduce the amount of treatment I have to do, maybe I can stop taking so many courses of antibiotics. I will just feel healthy,” he says. rice field.

But for many, like McDonald’s, the cost of medicine is exorbitant.

The list price of Trikafta in the US is $ 310,000. This means that you can pay more than $ 400,000 a year to provide a drug to a single patient in Canada.

Once you pass the state program, you will negotiate a price, which will be significantly reduced. However, if these programs do not add the drug to the approved list, individuals will have to pay the 6-digit sticker price themselves.

Manitoba has not yet added Trikafta to its coverage.

“Really we need to be funded by the state so that everyone can access it without deep debt,” McDonald’s said.

Health Canada has approved Trikafta for patients with cystic fibrosis aged 12 years and older with at least one F508del mutation.

“This drug acts on a specific mutation in Canada, which accounts for about 90% of the cystic fibrosis population, so it’s pretty important,” said John Warrenberg, Chief Scientific Officer, Cystic Fibrosis Canada. The doctor says.

How to use

Cystic fibrosis is caused by mutations in certain genes and produces defective proteins. It affects people’s lungs, digestive system and can cause an uncontrolled cough.

“It’s not necessarily the defective protein itself (that’s the problem). It’s the downstream consequences that are very harmful. This is the symptom it causes,” Warenberg said.

Trikafta not only treats symptoms, but also targets basic defects from one particular genetic mutation that causes CF.

“The difference with this drug is that it goes in and fixes broken proteins.”

“And what it does, it modifies the biological basis of the problem before the symptoms downstream of the problem,” Warenberg said.

There is no cure for CF, but supporters have said that Trifakta is as close to one as possible so far.

“Trikafta is actually a miracle drug,” said Patti Tweed, a defender of Canada’s major states of cystic fibrosis.

Coverage issues

On Thursday, the Canadian Institute of Health and Pharmaceutical Technology, the agency that reviews the cost-effectiveness of drugs on behalf of public payers, recommended listing the drug.

However, we also recommended limiting the scope to those with lung function of 90% or less.

“Under the current restrictions, 27% of Canada’s population will not actually be able to make a profit,” Tweed said. “And that’s just unacceptable.”

This means that CF patients with healthy lungs are forced to wait for their health to decline before they have access to drugs that may help prevent further disease progression. increase.

“In essence, it’s about eliminating most of the children and young adults who have done their best to maintain lung function and prevent it from deteriorating,” Tweed said. “So we certainly don’t want our people to get sick in order to have access to this drug.”

Correspondence of Manitoba

The state says the Pan-Canada Pharmaceutical Alliance (pCPA) reached a mutual agreement earlier this week on terms (pricing and coverage terms).

The role of pCPA is to conduct joint drug planning negotiations for brand names and generic drug states, territories, and federals, but the final drug financing decision is the authority of the public drug planning of the individual jurisdiction. It’s below.

“Manitoba goes through each process and decides to include Trikafta in the Pharmacare public medicine program,” said a state spokesman.

However, the state did not say when that decision would be made.

“Manitoba recognizes that the COVID-19 pandemic has created a difficult time for patients with cystic fibrosis and their caregivers, and will work on this file as quickly as possible.”