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Last summer, Health Canada approved a hemophilia drug called Hemlibra that has the potential to change Callum Guthrie’s life – but gaining access to it is proving to be an uphill battle

Callum Guthrie cuddles with his cat Stampy at home in Ketch Harbour, N.S. on April 29, 2019 after receiving the first round of lengthy infusions for hemophilia.Darren Calabrese/The Globe and Mail

When it comes to publicly paid prescription-drug bills, Callum Guthrie might be one of the most expensive patients in Canada.

His parents estimate that the wheelbarrow full of intravenous medications they pick up every two weeks to control their 10-year-old son’s severe hemophilia cost as much as $6-million a year, all of it paid for by Canadian Blood Services (CBS).

The drugs don’t even work terribly well: Despite being tethered to an IV pump at his Ketch Harbour, N.S., home for more than two hours every day, Callum still suffers relentless internal bleeds.

If he extends his left arm the wrong way, blood pools in his elbow joint, causing a vise-like pressure that only hospital-grade painkillers can relieve.

On his fourth day of primary school, he stretched in gym class and blood seeped into his quadriceps. He spent the next four months in a wheelchair.

“The pain is just so insidious,” said Michelle Howell, Callum’s mother. “It impacts him in ways you can’t imagine.”

Michelle Howell goes through the cupboards full of medications and medical equipment while preparing her son's first round of infusions for his hemophilia early in the morning.Darren Calabrese/The Globe and Mail

Last summer, Health Canada approved a drug called Hemlibra that has the potential to change Callum’s life, and the lives of about 90 other Canadians who are resistant to infusions of clotting Factor VIII, the conventional treatment for hemophilia A.

In an unusual twist for a breakthrough drug for a rare disease, Hemlibra is significantly cheaper than the medications Callum takes today, despite Hemlibra’s otherwise stratospheric sticker price of $668,685 a year for the average adult patient.

Yet Hemlibra still has not been added to Canadian Blood Services’ roster of drugs. Callum and his family want it desperately, and can’t get it.

The final decision rests with provincial and territorial governments, but PEI, which is currently chairing the Provincial Territorial Blood Liaison Committee, declined to explain the holdup.

However, it could have something to do with the sea change that Hemlibra heralds.

Hemlibra is the first in what is expected to be an explosion of expensive new drugs and gene therapies for the inherited bleeding disorder.

Delivered as a once-a-week injection under the skin, Hemlibra would liberate patients from hours of IV therapy, while controlling bleeding better than the medications available now for patients who are resistant to Factor VIII replacement therapy.

Bags of used medicine bottles rest under the sink as Colin Guthrie washes his hands before preparing Callum's first round of medication.Darren Calabrese/The Globe and Mail

“It’s a dramatic, dramatic change,” said Manuel Carcao, co-director of the hemophilia clinic at the Hospital for Sick Children in Toronto. “I would even call this a revolution in hemophilia management.”

Graham Sher, the chief executive officer of CBS and a hematologist by training, said his organization has already recommended that Hemlibra be covered for patients who are resistant to Factor VIII, the only category for which the drug is currently licensed in Canada.

But the provinces and territories that fund CBS have the final say, and they have to conduct their due diligence, Dr. Sher explained. (The PEI Department of Health and Wellness referred The Globe and Mail’s questions back to CBS.)

Part of that due diligence is evaluating what comes next. If Hemlibra and future drugs like it are eventually made available to the majority of the 3,000 Canadian hemophilia A patients who – unlike Callum – respond well to cheaper Factor VIII, that revolution could break the bank at Canadian Blood Services.

Buying drugs is how CBS spends the bulk of the funding it receives from every province and territory except Quebec. (Quebec has its own blood authority, Héma-Québec.)

In 2018-2019, 57 per cent of CBS’s $1.15-billion budget went to paying for drugs called plasma protein products, most derived from the straw-coloured liquid in human blood.

Colin Guthrie removes a syringe from Callum's implanted central line following the first round of infusions.Darren Calabrese/The Globe and Mail

Patients receive the drugs for free, with none of the co-pays, deductibles or perplexing province-to-province inconsistencies in coverage that plague the rest of Canada’s fractured system for paying for prescription drugs.

As Mark Belletrutti, a pediatric hematologist at Stollery Children’s Hospital in Edmonton, explained: “CBS is almost like national pharmacare for hemophilia patients and bleeding patients in general."

CBS has played that role since it was founded in 1998 in the wake of the tainted blood tragedy, a scandal that could just as easily be called the tainted plasma-product tragedy because of the thousands exposed to HIV and hepatitis C through contaminated plasma-derived drugs.

Michelle Howell, Callum’s mother, remembers how the panicky early days of the AIDS epidemic distressed her late older brother, Russell Howell, a hemophilia patient who was socially ostracized, despite never acquiring HIV himself.

“My brother received death threats, like, ‘You’ve got to move out of this building or we’re going to kill you,' ” Ms. Howell said. “People were really, really frightened.”

Ms. Howell’s brother, who died at the age of 31 of an aneurysm unrelated to his hemophilia, managed to elude the HIV virus because he had an “inhibitor” – in hemophilia parlance, that’s resistance to Factor VIII replacement therapy. He received drugs derived from pig blood instead.

The implanted central line needed for Callum Guthrie's replacement therapy.Darren Calabrese/The Globe and Mail

Like his late uncle, Callum Guthrie has severe hemophilia with an inhibitor.

Hemophilia is an inherited, genetic disease that primarily afflicts men. About 80 per cent have hemophilia A, which is characterized by insufficient amounts of Factor VIII, one of the factors that work in a cascade to make blood clot.

Because of her family history and tests that revealed she was a carrier, Ms. Howell and her husband, Colin Guthrie, knew that if they had a son, he would have a 50-per-cent chance of having hemophilia.

Yet neither anticipated their second child − a little brother for daughter Freya − would have such a severe incarnation of the disease.

Between the ages of 2 and 4, Callum landed in the IWK Health Centre in Halifax more than 300 times as doctors struggled to keep his bleeding in check.

Callum is now attending Grade 4 classes when his health permits, but Ms. Howell home-schooled him for most of his childhood.

His parents filled their home on the ocean with Lego, wooden model boats and other enticing toys to draw neighbouring children into Callum’s circumscribed world.

But Callum dreams of spending more time outdoors, in his friends’ world.

“My neighbour, he actually opens a little skating rink in his backyard every winter and I can’t [skate with them]," Callum said. "I want to be able to do that. I want to be able to play soccer at school.”

Michelle Howell sews pieces of neoprene from donated wetsuits into the knees of her son Callum's jeans for padding.Darren Calabrese/The Globe and Mail

In an effort to prevent internal bleeds, Callum receives twice daily infusions of FEIBA, a bypassing agent that is supposed to do the work of the Factor VIII protein that his antibodies would otherwise destroy.

He also receives a brand of Factor VIII called wilate, prescribed in large doses in the hopes that Callum will one day develop a tolerance for Factor VIII.

All the drugs are free to the Guthrie family. Based on the scant amount of public information available about unit prices, Ms. Howell and Mr. Guthrie estimate taxpayers spend about $6-million a year on Callum’s medications.

The Canadian Agency for Drugs and Technologies in Health (CADTH,) an independent agency that advises the provinces and territories on whether they should pay for new drugs, concluded in a draft value-for-money report obtained by The Globe that covering Hemlibra for patients with inhibitors would save the system more than $32-million in drug costs alone in the first year of use.

“If you ask yourself, why aren’t they approving this? There’s no obvious answer because it’s such a no-brainer,” said David Page, national director of health policy at the Canadian Hemophilia Society, a patient advocacy group.

“There’s got to be something else behind it … if they say ‘yes’ to this drug for this small group of inhibitor patients, then they’re afraid that this will open the floodgates."

Callum Guthrie, 10, laughs after spraying his friend Javen (not pictured) with water while playing in his workshop.Darren Calabrese/The Globe and Mail

Indeed, CADTH’s draft analysis found that if CBS extended coverage of Hemlibra to all hemophilia A patients, it would add $213-million in drug costs alone in the first year, rising to nearly $325-million by Year 3.

Mr. Page called those figures “ludicrous,” because they presume even patients with mild cases of hemophilia would take the new drug. CADTH declined to comment on the draft.

Dr. Sher of CBS said that if Health Canada were to broaden the approval criteria − something for which Hoffman-LaRoche has already applied − CBS would not automatically expand coverage.

There is another quandary at play, too.

Hemlibra is a monoclonal antibody that contains no human blood products whatsoever.

It’s not the first non-blood product that CBS has carried, but it is the first treatment for hemophilia that is neither derived from blood nor a recombinant drug – a lab-engineered copy of a clotting factor protein.

At what point do drugs for hemophilia look so different from traditional blood products that it stops making sense for Canadian Blood Services to provide them at all? Should they be treated like other new drugs, with some of their costs shifted to patients with robust private insurance?

“I will be very honest with you and say these are ongoing conversations with the provinces and territories as our funders,” Dr. Sher acknowledged.

Callum Guthrie, right, 10, and his friend Javen ride their bicycles in Ketch Harbour.Darren Calabrese/The Globe and Mail

Dr. Sher said it was always clear that recombinant copies of clotting factor proteins fit CBS’s mandate to provide blood, blood products and their “respective artificial and substitute products.”

“Now, as these new monoclonals are coming along, it’s a fair discussion point to say, ‘Do they fit the same definition?’ ” Dr. Sher said. “We would contend that they do.”

As provincial and territorial officials continue to deliberate, Hoffman-LaRoche has twice rejected the Guthrie family’s request to access Hemlibra for free on compassionate grounds.

A spokeswoman for Hoffman-LaRoche said by e-mail that she could not comment on individual cases, but that a panel of third-party physicians, not the company itself, decides which patients receive free Hemlibra.

Hoffman-LaRoche offered to defer charging Callum’s parents for Hemlibra if the Nova Scotia government would promise in writing to foot the bill eventually, Ms. Howell and Mr. Guthrie said.

The province declined. A spokesman for Nova Scotia’s Health Minister would not comment on Callum’s case, citing privacy concerns.

“We’re fighting two of the biggest and most challenging forces known to man: A pharmaceutical company and the government," Mr. Guthrie said. "How the hell are you supposed to penetrate that?”

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