Manitoulin family battling CF hopeful about eventual drug coverage, encouraged by support

Ben Buzzell needs to keep this bottle of pills and a sweet drink such as orange juice on-hand every day and take the medication with any bit of food or drink he consumes. This is just one small portion of the full scope of drugs and breathing devices the 11-year-old has to use on a regular basis to manage his cystic fibrosis. His dad Zak Nicholls, left, has been helping to campaign the government on Ben’s behalf for years. photo by Warren Schlote

LITTLE CURRENT – In the ongoing quest to secure government funding to cover new life-altering cystic fibrosis (CF) medication, Little Current’s Ben Buzzell and his family have recently scored a few small wins.

“I get the feeling that (public coverage) is going to happen eventually, but now it’s just a matter of sooner is better,” said Zak Nicholls, Ben’s dad.

CF is a genetic disease that causes the lungs to produce thick mucus that is difficult to remove. These clog the airway and accumulate over time, steadily decreasing lung function. It also causes the loss of key digestive enzymes, meaning the body cannot absorb nutrients without taking special pills to encourage their intake, which leads to rapid weight loss. CF can also lead to complications such as diabetes and lung infections.

A recent win for the family was the Northeast Town’s response to their request that Friday, May 17 be designated as Cystic Fibrosis Awareness Day in the township. Council agreed to the motion and added an extra measure, that they would write to the Ontario Minister of Health and Long-Term Care and request that 11-year-old Ben’s drugs be covered publicly.

“I go to a lot of council meetings and I had a good feeling they’d approve the day, but I was really happy and surprised when they said that extra part,” said Mr. Nicholls.

The Expositor first reported on Ben’s situation in September, when the family was fighting a campaign alongside many other individuals with CF to get the federal government to cover life-altering drugs which help treat the condition. At the time, they were pushing for public access to Orkambi, a relatively new drug by pharmaceutical company Vertex that treats Ben’s specific mutation of CF. The federal government has already approved Vertex’s first-generation drug, Kalydeco, though it does not treat Ben’s mutation.

A third CF drug made by Vertex also exists, called Symdeko. It’s the newest of the three and was only approved by Health Canada in June 2018. Symdeko goes beyond the other two drugs that merely prevent CF from worsening; in some trials it has been shown to reverse the health impacts of CF, including decreased lung function.

Due to the nature of CF, drugs can only effectively treat a specific mutation of the disorder. There are more than 1,700 mutations that can cause CF, a condition found in more than 4,300 Canadians.

Ben’s mutation, called F508del, is the most common cause of CF and makes up about half of the CF cases in Ontario (about 700). Both Orkambi and Symdeko treat F508del.

Late last year, the Canadian Agency for Drugs and Technologies in Health (CADTH) underwent a review period to determine whether Orkambi should be included within public drug coverage. To the chagrin of Ben and his family, along with Cystic Fibrosis Canada and the many individuals impacted by CF across the country, the board recommended against publicly funding the drug.

“Ben and I and the CF community kind of knew that was going to come, so there wasn’t a great deal of disappointment. It just increased our push to come up with other plans,” said Mr. Nicholls.

New hope for CF patients emerged this past February, despite the October decision by CADTH. Along with Alberta and Saskatchewan, Ontario outlined criteria for exceptional cases of CF that would be eligible to receive public coverage of Orkambi, a drug that can cost more than $250,000 per year, per person.

However, the criteria to have these drugs funded is extremely strict. So strict, in fact, that Mr. Nicholls expects few people—if any—to qualify for the treatment.

“A CF patient has to lose 20 percent of their lung function in six months, and it has to be sustained for six weeks. That’s not how CF works; it’s not a rapid decline,” said Mr. Nicholls. 

He added that CF Canada was studying its registry of patients to determine if anyone in its history would have qualified for the funding, which is only available for patients between ages six and 17. CF Canada was unable to provide a response to that question by press time on Monday. 

The CF community was expecting the three provinces to cover Orkambi, so the news came as a disappointment. Ben and his family were all set to celebrate before they learned of the outcome.

“I was just annoyed. They said they were going to do it, and it turned out they were not going to cover anyone,” Ben said.

Despite the setback, Mr. Nicholls said the news might bring an improvement in its own way.

“On the positive side, they did acknowledge the drug, so I think it’s a step in the right direction,” said Mr. Nicholls.

Mr. Nicholls is hoping that the criteria for eligibility for these drugs will be reduced, and that a newer drug called Symdeko will eventually replace Orkambi. He added that a fourth generation of the drug is also in development that promises to be even more effective than Symdeko and carry fewer side effects. The current drugs cause patients to lose a lot of weight and they have a higher risk of developing glaucoma in later years.

There has been growing public support for Ben’s condition recently. Since they have been campaigning for his drug coverage, more than 300 people have signed and sent letters to Christine Elliot, Minister of Health and Long-Term Care, on Ben’s behalf. Ben also wrote a letter and, for the first time in the campaign, the ministry wrote a response acknowledging that Ms. Elliott had read the letter.

On Friday, May 17, Little Current Public School will be hosting Cystic Fibrosis Day, a chance for students and staff to learn about CF, participate in activities that aim to illustrate what living with the condition is like and to wear purple in support of Ben’s condition.

The federal government has also begun to create a national pharmacare program that funds rare disease drugs, beginning in 2022. Although there is a risk that, if the Liberals lose this fall’s election, the plan may be cancelled, Mr. Nicholls said he had hope for the Conservatives’ current shadow health minister Marilyn Gladu.

“Her personally, she’s really in favour of rare disease drugs that go to the right people. And that’s more than just lip service, in my opinion,” said Mr. Nicholls.

He said he explained that the cost for a million-dollar single lung transplant far exceeds the cost of covering a drug like Orkambi or Symdeko for years at a time—drugs that can negate the need for a transplant altogether.

Mr. Nicholls said the present government will set aside between $500 million and $1 billion for rare drug coverage through this program, something he believed has not happened in the past.

“It’s on their radar for some time in the future. I just hope it’s sooner,” said Mr. Nicholls.

For the time being, the family will continue pushing all levels of government to support their fight to get Ben’s life-changing drugs covered.