The news was a blow to 16-year-old Liam Simpson who is living a normal life thanks to the drug. “My lung function has skyrocketed,” Simpson, who used to get teased in gym class for not being able to run, told CBC News
. “It makes me feel more normal.”
According to experts, Orkambi is one of a handful of cutting-edge drugs that targets the defect at the root of CF, a genetic disorder that affects some 4,100 Canadians. A particular gene mutation causes hyperactivity in the secretory glands, causing lungs to clog up with mucus, which affects the pancreas and gut. Orkambi is used to treat patients over 12 with a specific mutation that manifests in 80% of CF sufferers.
Taking four pills a day has changed Simpson’s life. Before, even a cold could leave him struggling for breath and require a trip to the hospital. But after getting access to the Orkambi as a participant in drug trials during the approval process, his lung function has doubled, and he’s enjoyed three hospital-free years.
However, the treatment is expensive, costing each patient $250,000 a year. With Health Canada giving Orkambi its blessing to be sold in the market, some private insurers have it covered, but others do not cover it or place a limit on the number of claims or the time during which patients can file claims for it.
In spite of Health Canada’s approval, BC will not fund the treatment for any of the estimated 150 people who suffer from the disease in the province. “We understand that quality of life for people with cystic fibrosis can be a significant challenge and it is important that these patients get the treatment they need,” the provincial ministry of health said in an emailed statement. However, it has decided against covering the drug partly because of the Canadian Agency for Drugs and Technologies in Health (CADTH).
In October 2016, the CADTH gave a negative recommendation for Orkambi, saying there is “insufficient evidence” from drug trials to prove its efficacy. The arms-length group urged provinces not to include the drug in their coverage. Brent Fraser, a representative of the CADTH, said the organization reviewed the drug more extensively than most private insurance companies, and that cost was not the only factor.
Dr. John Wallenburg, Cystic Fibrosis Canada’s chief scientific officer, said trials have shown increased lung function due to the drug, which has benefitted more people with CF. According to him, the decision to not cover the drug was “largely motivated by price,” and will be a death sentence for some.
Saskatchewan saddled with hundreds of millions in unnecessary healthcare
Terminal cancer patients’ access to immunity booster Neupogen cut off
Health Canada approved cystic fibrosis (CF) medication Orkambi for its safety and efficacy in January last year. However, the BC Ministry of Health has decided “along with the other provinces and territories, not to list Orkambi as a PharmaCare benefit.”