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Cystic Fibrosis community is one step closer to accessing treatments

July 3, 2020   ·   0 Comments

A better quality of life could be coming to thousands of Canadians with Cystic Fibrosis as negotiations to include gene modulators in public drug plans are now underway.

The Pan Canadian Pharmaceutical Alliance (pCPA) is currently in talks with Vertex, the pharmaceutical company behind breakthrough gene therapy drugs for Cystic Fibrosis such as Orkambi, Kalydeco, and Trikafta.

Vertex’s drugs are covered through public health plans in several other developed countries with socialized medicine, while Canada has fallen behind, largely due its lengthy process for getting new drugs publicly funded.

“Access to these lifesaving drugs make a huge difference in the quality and length of life for thousands of Ontarians and indeed Canadian children and young adults,” said Simcoe–Grey MPP Jim Wilson, at Queen’s Park last month.

“Obviously…many parents across Ontario are very, very worried during this time of COVID-19, which is also a disease or a virus that effects breathing.”

Tottenham mother Sasha Larocque has two boys with Cystic Fibrosis. Her older son, Andre, has been able to access Orkambi through a clinical trial and it has nearly stopped all his symptoms, while her younger son Joshua has to face the illness head-on.

People with Cystic Fibrosis are more prone to getting sick and when infected, and it is exponentially more difficult for them to recover, sometimes leading to multiple hospitalizations each year.

Larocque told the Times her primary concern with the pCPA negotiations is its unclear timeline. She said it’s her hope that the drugs become available before Joshua goes back to school this fall due to the risk of COVID-19 and other viruses that may be circulating.

MPP Wilson, who has been pushing for increased access to the gene modulators for three years, brought up Larocque’s story when pressing Ontario’s Minister of Health Christine Elliot for a timeline on the negotiations at Queen’s Park last month.

Elliot noted that there are other provinces and territories negotiating at the table, so Ontario alone cannot control their speed of the negotiations.

“This is very important to many members here that have constituents that have Cystic Fibrosis,” she said in response to Wilson. 

“There’s nothing more that any of us would like than to make sure that the family that you mentioned as well as many other families across Ontario have a happy summer with their family members being able to breath easily,” Elliot continued.

“We will do everything that we can to move this process forward expeditiously.”

Wilson said because Ontario will be the largest purchaser of Orkambi, Kalydeco, and Trikafta, it should have the most say in getting these drugs to get the market.

The pCPA told the Times their targeted timelines would conclude the negotiations within 130 business days after they begun. However, due to the negotiations’ complexity, it could be drawn out even further.

Kim Steele, spokesperson for Cystic Fibrosis Canada said her organization is thrilled to see the negotiations moving forward now after years of lobbying to improve Canadians access to gene therapy.

“We’re very pleased by this announcement, but it is tempered with the reality that there’s still work to do,” she noted. “There’s no timeline on these negotiations and once they are complete the provinces still have to agree to cover the drugs…but certainly things are moving in the right direction.”

In the fall of 2019, the new gene modulating drug Trikafta was designated a breakthrough medication in the U.S and fast tracked for approval in Canada, as it works on roughly 90 per cent of people with Cystic Fibrosis.

Currently, certain people with Cystic Fibrosis can access Kalydeco, which helps treat roughly four per cent of people with the disease, while Orkambi is much harder to access but has a success rate around 50 per cent.

Under Ontario’s current criteria, a person has to lose 20 per cent of their lung function for six weeks or longer within a six-month period to be prescribed Orkambi,

To date, zero Ontarians have qualified.

Access to gene modulators is largely dependent on location, according to Steele.

“Some mutations are covered in certain provinces and some aren’t, it’s a real mishmash, it’s a reflection of this health system that we live under in Canada, where access depends on where you live,” Steele said.

She said if the current pCPA negotiations go well the criteria and restrictions on accessing Okrambi or Kalydeco could expand.

However, Trikafta appears to have a stronger likelihood of helping even more people with Cystic Fibrosis, but likely won’t be available for everyone anytime soon.

Studies have shown Trikafta has the potential to help around 90 percent of gene mutations found in people with Cystic Fibrosis. This drug is currently not indicated for children 12 and under in Canada, despite trials in the U.S showing it can be more effective on young patients.

“We have just seen so much tremendous responses to these medicines that it doesn’t make sense to not start these medicines earlier and help prevent the progression of the disease, rather than what we’re doing now which is waiting for people to get very sick before they can access any of these medicines,” Steele remarked.

She said how Canada applies these drugs to the Cystic Fibrosis community is an issue as it takes sick people and tries to make them better instead of using a preventative approach. New studies show the gene modulators can slow the progression of the disease.

Due to Trikafta’s lack of clinical trials for children in Canada it could be another few years before it’s made accessible, while Orkambi may act as a buffer in the interim, Steele noted.

She added that the issue is time sensitive, particularly due to the COVID-19 pandemic, which placed pressure on the pCPA to make something happen.

“We certainly felt at this time that it should be managed for humanitarian reasons that our government had to act, and that we had to get something in place for many of the people who may need access at this time,” she said.

“That is definitely the sense of urgency that we placed on government during our ramp up to this.”

By Sam Odrowski
Local Journalism Initiative Reporter

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