This 7-year-old is finally on a 'life-changing' cystic fibrosis drug. For others in Ontario, access is tricky

Kelly Marchand of London, Ont., is thankful her seven-year-old daughter Charlotte, who has cystic fibrosis, is finally taking a prescription medication that some doctors describe as life changing.

"It brought me to tears," Marchand told CBC News. "It means so much for my daughter to be able to have this medication, to hopefully slow down her disease progression and cause fewer issues."

CF is a hereditary disease that affects the cells that produce mucus, and can lead to a buildup in the lungs, digestive tract and other organs. It can make the body prone to lung infections and often, lung transplants are required.

In July, Ontario made Trikafta available to children six years old and up. The triple combination therapy works to correct the defect of the gene that causes CF.

According to Cystic Fibrosis Canada, an estimated one in every 3,600 children born in this country has CF. More than 4,300 children, adolescents, and adults with cystic fibrosis attend specialized CF clinics, according to the non-profit organization.

"This is the most promising drug for the underlying treatment of CF," said Marchand, who was able to use private insurance coverage from her job to secure Trikafta. 

Charlotte started Trikafta last week and is still getting used to having the drug as part of her daily routine. 

But many families in Ontario struggle to navigate insurance coverage to afford the drug, which costs about $300,000 a year for a single patient.

Trikafta can be covered by the Ontario Drug Benefit (ODB), available through the Exceptional Access Program (EAP).

It's really hitting a lot of average middle-class families and putting them in awkward positions.- Kim Steele, Cystic Fibrosis Canada

Individuals 24 and younger are eligible to get the medication through OHIP+ as long as they don't have private insurance. People between 25 and 64 can enrol in the Trillium Drug Program if they have a high drug cost relative to their income — there's also a deductible and co-payment.

However, Kim Steele of Cystic Fibrosis Canada said this system doesn't give Ontarians the safety net to secure full coverage because if their private insurance doesn't pay, they may have to be responsible for a deductible for both sectors. 

"If you have private insurance, you're excluded from that program [OHIP+] altogether, even if your insurance won't cover the full cost of the drug," Steele said.

"It's really hitting a lot of average middle-class families and putting them in awkward positions where they or their children might not be able to get the drug unless they remove themselves from their plans, losing benefits like dental and others." 

Drug is a 'game changer,' says doctor

Each province has its own health-care model, which prevents a co-ordinated, Canada-wide approach to accessing medicine like Trikafta, Steele said. 

Dr. April Price, director of the pediatric cystic fibrosis clinic at the London Health Sciences Centre, describes Trikafta as a "game changer" in her patients' lives.

"What this drug has been able to do is really something from a science fiction movie brought to life," she said. "Things start to move which had been stuck for a long time, so cough and increased mucus production are some of the known results of starting the drug." 

But Price acknowledged its high cost prohibits many patients from accessing it, especially when they don't fit criteria of provincial eligibility, she said. 

"As soon as you stop taking this drug, all its effects wear off, so there's going to be an ongoing need for every patient in Canada, which there are over 4,000 per year," Price said.

Canada needs a rare disease strategy to allow provinces and territories to work together to create a united framework in order to reduce barriers in accessibility, Steele said. In November, Cystic Fibrosis Canada will meet with MPPs at Queen's Park in Toronto, along with a team of community advocates, to talk about better access to the drug.

"Something has to change because it's not fair that families have to struggle so much to get this drug for their children in Ontario," Steele said.