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 fewerissues."

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

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

According to Cystic Fibrosis Canada, anestimated one in every 3,600 children born in this countryhas 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.

Charlottestarted Trikaftalast week and is still getting used to having the drug as part of her daily routine.

Butmany families in Ontariostruggle to navigate insurance coverage to affordthe drug, which costs about$300,000 a year for a single patient.

Trikaftacan be covered by the Ontario Drug Benefit(ODB),available through theExceptional 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 areeligible to get the medication through OHIP+ as long as they don't have private insurance. People between 25 and 64canenrol in the Trillium Drug Program if they have a high drug cost relative to their incomethere's alsoa deductible and co-payment.

However,Kim Steele of Cystic Fibrosis Canada saidthis system doesn't give Ontarians the safety net to secure full coverage because if theirprivate 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 yourinsurance 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 provincehas 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, describesTrikaftaas a "game changer" in her patients' lives.

"What this drug has been able to dois really something from a science fiction moviebrought to life," she said. "Things start to move which had been stuckfor a long time, so cough andincreased mucus production are some of the known results of starting the drug."

But Price acknowledgedits 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 allowprovinces and territories to work together to create a united framework in order to reduce barriers in accessibility, Steele said. In November,Cystic FibrosisCanada will meet with MPPs atQueen's Park in Toronto, along with a team of community advocates, to talk about better accessto the drug.

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