Thirteen-year-old Larissa Rueckenbach stares quietly at the kitchen table.
“I don’t like being in the hospital,” she says. “It’s a little bit scary.”
She pauses as yet another cough rattles in her throat.
“It’s not very fun, and it takes a few days till I feel better. For the first few days I’m always sick and throwing up,” she says, shyly.
Larissa has cystic fibrosis. Of the more than 1,900 variants of the disease, hers is one of the rarest. Given the ugly moniker G551D, it’s a particularly nasty mutation.
Cystic fibrosis is a genetic disorder that affects the lungs, filling them with mucus and causing repeated bacterial infections. It affects about one in every 3,600 people in Canada, cutting their life expectancy to around 40 years of struggling just to breathe.
Patients like Larissa often have to be hospitalized at least twice a year for treatment that helps clear bacteria out of the lungs.
For most, there is no cure.
Larissa’s variant is extremely uncommon. Only 118 Canadians are affected by it, but it’s special not only for its rarity: it may also have something close to a cure.
Priced out of reach
Health Canada approved a new drug, Kalydeco, for use in 2012, and its clinical trials seem to show that it can halt the damage caused by Larissa’s variant of cystic fibrosis.
“It makes that gene function normally again, so there is no more cystic fibrosis,” says Larissa’s mother, Heike.
“It disappears. The damage in the lungs remains there, but there is no further damage,” she says.
There is one catch, however: it currently costs $349,000 per year and it isn’t covered by any public health-care plans in Canada. The government has been negotiating with Kalydeco’s manufacturer, Vertex, for a year and a half, trying to secure a lower price for the drug, but negotiations are currently stalled.
Larissa’s parents want to see that changed, as fast as possible.
“If it were closer to, say, even $60,000, we could fundraise that, but $349,000,” Heike says, pausing, “it just seems like an endless sum. We need this medication covered for our children.”
The Rueckenbachs have been waiting for years to get their daughter proper treatment. Before moving to Whitehorse this spring, they lived on a trapline outside of Atlin, B.C.
“It was pretty cool,” Larissa says, smiling.
But the lifestyle was also difficult for Larissa. She was sick all the time, and the family tried repeatedly to find out why.
Every time she went to see a doctor, Heike was told that Larissa had asthma, until “Finally, I said no. Something is wrong. It’s not just asthma. We’ve seen five doctors and you’ve got to do something.”
Eventually a chest X-ray led the family to the B.C. Children’s hospital in 2010 and she got tested for CF the next day.
As CF progresses it can bring with it a host of other complications. Larissa also suffers from CF-related arthritis, which sometimes keeps her bedridden for days, unable to move because of the pain.
In order to deal with the symptoms of her illness, Larissa’s current cocktail of medicines includes a long list of drugs like Prednisone, Naproxen, Ventilin, Sulfasalazine and painkillers.
Another common complication is CF-related diabetes, which Larissa also now struggles with.
“She started shooting insulin just last week because her pancreas is at the point where it’s not turning the sugars into energy anymore,” Heike says.
With Kalydeco, the hope is that most of the other drugs could be done away with as her underlying condition improves.
The most pressing reason she needs the drugs, however, is to save her lungs from further harm and prevent what will otherwise be an almost inevitable lung transplant.
“She’s already got holes in her lungs from all the bacterial infections,” her mother says.
If a transplant can be avoided, not only does that mean less risk to Larissa, it also means a life-saving set of new lungs can be saved for someone else who needs them.
‘They are negotiating with my daughter’s life’
Larissa is not alone in this fight. Twelve-year-old Madi Vanstone, from Toronto, has been to that province’s legislature twice now, pleading for the government to cover the cost of her pills.
The Pan-Canadian Pricing Alliance, a government drug-buying consortium led by bureaucrats from Alberta and the Yukon, has been haggling with Vertex for 15 months, trying to negotiate a price that will allow public reimbursement for the costs of the drug.
Frustrated by getting nowhere, Yukon Health Minister Doug Graham and Alberta Health Minister Fred Horne recently called for a face-to-face meeting with Vertex top brass, hoping to break the logjam.
That meeting took place in Toronto on May 13, but still no agreement has been reached. The company says its most recent offer “is as good or better than the agreements in place in 15 countries around the world,” said Stuart Arbuckle, the company’s vice president.
Germans pay one of the highest prices, around $450,000 per year for the drug. Americans, meanwhile, pay one of the lowest – $311,000 per year.
In the meantime, individual provinces and territories are reluctant to cover Kalydeco on their own, for fear of undermining the Pan-Canadian negotiations.
“It’s been 15 months, and they are negotiating my daughter’s life,” Heike says.
Vertex communications manager Zach Barber said the company has invested more than $6 billion into research and development since it launched in the 1990s.
“That’s billion with a ‘B,’” Barber said, adding that two successful drugs have resulted, including Kalydeco and another that treats hepatitis C.
The high cost of Kalydeco is due to three factors, Barber said. First is the expensive development. Second is the fact that it’s a so-called orphan drug. Because of the rarity of people affected by this particular variant of CF, the marketability of Kalydeco is low – around 3,000 people worldwide, Barber said.
Third, the company has to consider how impressive the drug is and what it’s capable of, he said.
“This is the first drug of its kind to treat the cause of CF, not the symptoms of it,” Barber said. The company is currently testing to see if it can be applied to other variants beyond the one Larissa has, he said.
While the negotiations continue, the company offers Kalydeco free-of-charge to patients who are “very sick,” and financial assistance to others, though Barber would not say how sick someone must be to qualify for help from the company directly.
‘Exorbitant’ pricing, says medical association
Last October, the Journal of the American Medical Association slammed Vertex for what it called “exorbitant” pricing. The report pointed out that the drug’s development was aided through the decades by “a highly supportive community built by and around patients and their families,” including $75 million from the Cystic Fibrosis Foundation.
“The vast majority of patients cannot afford this financial burden, and transferring the cost to private or federal insurers does not obviate the underlying problem – an unsustainable pricing structure,” the report says.
“Adding to the unacceptability of the price of this new drug is that the manufacturer did not develop this product alone … Pharmaceutical companies have an implicit obligation to put patient well-being and resource utilization on equal footing with return on investment,” the report says.
The 15-month negotiation with Vertex is nearly double the length of time any country has taken to approve the drug for coverage, according to the company.
Paddy Meade, the Yukon’s deputy minister for health and social services, says that’s because Vertex went to some countries first and cut deals, while other countries got stuck with paying high costs.
“The concern for health ministers is that we’re talking about one drug, one very high cost drug. But there are many more high cost drugs coming down the pipe,” Meade said.
“In fairness to governments, these are fine balancing, tough decisions. It’s not about does the drug work or not,” she said.
“It’s about does it actually do what the manufacturer says and does? It’s been called a miracle drug, but is it a miracle drug for all patients?” Meade said.
While she waits for the haggling to end, Larissa can’t participate in gym class at school, and misses huge amounts of time when her arthritis keeps her at home. This semester alone she missed 160 classes.
“I like all sports. I’d really like to be able to play soccer, volleyball or baseball, but I can’t play any of them,” she says.
One of the few bright spots in Larissa’s day is the time she gets to spend with her horse, Fancy. Riding the eight-year-old horse is one of the only physical activities Larissa can do that doesn’t leave her breathless or aching with arthritic pain.
“I really like riding,” she says, stroking Fancy’s nose. “We go exploring on the trails near here. Sometimes I take her for a drink at the creek.”
Larissa’s connection with Fancy is obvious, but her affinity goes beyond horses.
“I want to be a vet,” she says brightly. “I feel like I have a connection with animals.”
Sitting at the kitchen table talking about doctors and needles and treatment, Larissa is subdued. Coughing occasionally, she talks quietly about her new insulin injections, and how she doesn’t want to keep going back to the hospital anymore.
But outside in the yard, she’s transformed into a bubbly 13-year-old girl with a horse she obviously adores. That’s the girl she deserves to be all the time, her mother says.
“How much is a human life worth? How can you put a price on it?” she asks.
Contact Jesse Winter at