Medication to support boy with cystic fibrosis can cost nearly $250,000
LITTLE CURRENT—Ben Buzzell can only breathe at two-thirds the capacity of his other Grade 5 classmates. He carries around a slate of pills, inhalers and what is called a PEP machine, a tool he uses to clear mucus from his lungs. He has been living with this condition for his whole 10-year-old life, though he only received a diagnosis at the age of three.
Ben has cystic fibrosis.
Cystic fibrosis is a condition that affects over 4,200 Canadians, with a majority of patients being adults. However, one baby per week is diagnosed with the disease. Unfortunately, mandatory screenings for cystic fibrosis were implemented just two months after he was born, so he went three years before being diagnosed with the condition with which he was living.
“It’s pretty annoying when people tease me about being so short and skinny,” says Ben. He adds that the condition impedes his ability to participate in cross country running and other activities he would otherwise enjoy.
“When I do sports, I usually end up coughing a lot,” he says. “I get headaches easily, and when I go swimming I can get cold pretty easy.”
“It’s stressful, it interrupts one’s lifestyle, and it’s just in the way,” says his dad Zak Nicholls, adding that Ben has to attend four blood clinics per year to undergo tests.
There are drugs available to help manage the condition, but they come at a cost: about $249,000 per year for each person who uses them. That’s the price for a drug called Orkambi, produced by Boston-based Vertex Pharmaceuticals.
According to an information flyer by Cystic Fibrosis Canada and provided to The Expositor, a blind drug test of Orkambi resulted in improved lung function, fewer lung infections, fewer hospitalizations and increases in Body Mass Index, which, it says, increases survival rates.
Orkambi is the second generation of cystic fibrosis drugs by Vertex. The first version, Kalydeco, is already available publicly in Ontario under the exceptional access program (EAP). EAP is a program to which patients can apply in the hopes of getting special consideration to cover their drug costs.
The problem with cystic fibrosis drugs is their limited effectiveness on different strains – drugs only work on the specific mutation of the disease for which they were designed. The problem lies with the fact that cystic fibrosis can be caused by one of over 2,000 genetic mutations. Kalydeco does not treat Ben’s condition, known as “F508del.”
Fortunately for Ben, though, F508del is the most common cystic fibrosis-causing genetic mutation, and the same mutation that Orkambi treats with success. Over half of Canadians with cystic fibrosis—nearly 2,100—have two copies of the F508del gene. Mr. Nicholls says about 700 Ontarians have that particular mutation. However, without the financial support to cover Orkambi, it remains out of reach for the average family.
For the time being, Ben is dealing with the condition as best he can, although it means carrying around a small box of pills and inhalers. One of the pills is an enzyme to help Ben’s body break down food – he has to take one pill for every five grams of fat he eats. Cystic fibrosis not only fills the lungs with mucus but also the pancreas, which normally produces the enzymes needed in digestion. With no way of those reaching the digestive tract, Ben’s body is seriously restricted in what it can digest.
Ben also has a nose spray to reduce polyps in his nose. Those are caused by the excess mucus levels and can be uncomfortable and block the airway. They also interfere with Ben’s sense of smell, something he says he misses. Ben has had to travel to Toronto for surgery in the past to remove the polyps, but they are starting to grow back.
One of his inhalers treats pseudomonas, lung infections that Mr. Nicholls refers to as “similar to mold,” toxins his lungs cannot remove themselves. He also takes heartburn and appetite medications to keep his weight up, as cystic fibrosis patients often have problems maintaining a healthy weight.
“It means I can eat a lot; I like that,” says Ben. “One time I was with my brother at my grandma’s, doing the treadmill. I said, ‘hey, I lost 10 calories!’ and my brother said, ‘Ben, that’s not good!’”
Orkambi could treat a number of these problems for Ben and vastly improve his quality of life.
“It’s corrective gene therapy; it’s pretty incredible,” says Mr. Nicholls. There is a newer drug than Orkambi called Symdeko that was just cleared by Health Canada in June 2018. That drug has the ability to reverse some of the effects of cystic fibrosis, but Mr. Nicholls says there is no push to get that drug covered publicly because of its high price point.
The review process is ongoing, but time is short: The Canadian Drug Expert Committee will make its final recommendation to Vertex and drug plans on September 26. Mr. Nicholls says he has reached out to a number of agencies and governments, and a recent government letter shows the estimated outcome leaning towards declining the drug coverage.
“The only thing that will push it over the top at this point is a large, public outcry,” says Mr. Nicholls. He is trying to get Toronto lawyer Chris MacLeod to come give a talk at Ben’s school in Little Current at an assembly where Ben will also speak. Mr. MacLeod has cystic fibrosis himself and is leading a class-action lawsuit in B.C. over this very drug.
As with all other Canadian provinces, Orkambi is not covered by B.C. but Mr. MacLeod says the high price point has been negotiated down in other countries by using bulk purchasing power to cut a better deal. Mr. Nicholls says provinces can still opt-in to publicly cover Orkambi at this point, but without national support, that purchasing power will be greatly reduced and the costs may become prohibitive.
Mr. Nicholls also says that as a result, Orkambi manufacturer Vertex would likely never reapply for public participation in Canada. This is the third time Orkambi has applied for that process—according to a Vancouver Sun report, the two past applications had been declined, citing insufficient medical benefits. Vertex has allegedly supplied more data with results over a longer period of time.
Orkambi is called a preventative drug, meaning it can slow down the progress of future lung disease, but cannot undo the damage caused by cystic fibrosis. Starting patients on the drug as early as possible is crucial in having the greatest impact—Orkambi is approved for people over the age of 12. But with the high price point, it remains out of reach of most families.
There are four private insurers in Canada that cover Orkambi, but there is no public drug coverage. Mr. Nicholls and others are pushing to have Orkambi covered like Kalydeco under the EAP. This is within the context of Ontario’s new OHIP + program, begun in January 2018, that provides free drugs to people 24 years of age and under. They receive coverage regardless of their income levels or whether they already have private insurance coverage.
That age group will cost $465 million to cover, according to Cystic Fibrosis Canada. Adding in people aged 65 and over at $575 million means a total cost of $1.1 billion per year, which the organization says limits the government’s ability to spend on rare disease medications because their funding priorities are elsewhere.
“I’d want (the minister) to know that it sucks they won’t give me medication,” says Ben.
The Ontario Ministry of Health and Long Term Care could not provide comment by press time.