By cold mathematics, spending $1.3 billion to transform the lives of 3,700 Canadians might seem a poor return.
But Ron Jobbagy, father of two cystic fibrosis patients, is urging the federal government to look beyond the numbers when it comes to negotiating access to Trikafta, a life-changing treatment that has been available in the U.S. for a year but has yet to enter the approval process in Canada.
The 52-year old Calgary area real estate agent is father to Alyssa, 22, and Dylan, 20, both of whom have the genetic condition that affects the lungs, pancreas, liver, kidneys and intestine, making breathing difficult. It is particularly cruel on parents, who often feel guilt for having passed on the defective gene.
“This monster slowly but surely consumes families,” said Jobbagy. “My kids, my wife and me – it has absolutely consumed us.”
“We struggle so hard to make sense of it all,” he said, becoming emotional. “Nobody is asking for something that can’t happen or that is a pipedream. It’s there – it’s three hours down to the U.S. – and I can’t get it.”
Alyssa has spent six weeks this year in hospital and has seen her lung capacity drop to around one third of its normal function. She is no longer able to ride her horse, “the love of her life,” according to her father.
Jobbagy said Dylan is not nearly as sick but is increasingly angry as he watches his sibling’s condition deteriorate, knowing that his time is coming. He wants to move to Vancouver Island but has been advised to limit travel on his own.
The toll on all involved is physical and mental but also financial. Jobbagy estimates the family’s annual expenditure on drugs is up to $300,000, much of which is covered by drug plans.
But if Trikafta were available, three daily pills would replace many of the medicines Alyssa and Dylan take every day.
“The simple assumption when Trikafta became available in the U.S. was that we were going to see this medication here. It was the most uplifting point in my life,” he said.
Trikafta has been hailed as a miracle drug that enables people who can barely make it to the bathroom on their own lead relatively normal lives. A Dalhousie University study for Sick Kids and St. Michael’s hospitals estimated the age of survival for a child born with CF would increase by 9.2 years, if Trikafta were made available. The same study suggested 60 per cent fewer people would suffer severe lung disease, there would be 19 per cent few hospitalizations, a reduction in lung transplants and a 15 per cent reduction in deaths by 2030.
Its approval and swift introduction would appear to be a no-brainer.
But nothing in the world of leading edge pharmaceuticals is straightforward.
The list price of Trikafta is $311,000 per person every year.
Nobody pays list price but even if the government of Canada was able to secure a 50 per cent discount, the price tag would still be around $700 million a year to cover the 3,700 recipients.
Trikafta has become the benchmark case in a struggle between pharmaceutical companies determined to maximize profits during the window of patent exclusivity and a Canadian government that is equally determined to squeeze up to $13 billion in savings from prices over the next 10 year and improve this country’s access to drugs.
Canada’s new pricing regime guidelines were unveiled earlier this month by the Patented Medicine Price Review Board, the federal drug pricing regulatory agency, and will come into effect on January 1.
By removing the U.S. and Switzerland, countries that pay top dollar for drugs, from its list of comparator countries, Canada hopes to knock off 20 per cent from the starting price at which it opens negotiations with pharma companies.
Every week that passes, someone crosses the bridge and is too far gone for the drug to help
The Trudeau government has also introduced “pharma-economic” measures to further reduce prices – a cost-effectiveness/cost-benefit analysis of how much of an improvement the drug offers over existing therapies.
Critics contend there are no agreed standards for such measures. They also say the government’s new demand for further price cuts as drug sales rise will discourage pharmaceutical companies from bringing innovative drugs to Canada.
“Ottawa has doubled down on the fantasy that government can legislate lower drug prices without causing unintended consequences,” Brett Skinner and Nigel Rawson of the Canadian Health Policy Institute wrote in the Financial Post earlier this year.
Government sources point out that under the existing regime, Canada pays the third or fourth highest prices in the world, yet typically has to wait two years or more before drugs are introduced here.
“We feel strongly that the data doesn’t support price being the only driving factor or we’d have been getting better access before,” said one official.
(Of the 37 medicines launched globally in 2018, only 16 – or 43 per cent – were launched in Canada.)
Yet nobody really knows what is going to happen when the new pricing guidelines come into effect.
That’s why the Trikafta case is so critical.
Patient groups like Cystic Fibrosis Canada have been leaning on the government to step up the pace on Trikafta’s introduction.
The government says Health Canada encouraged Vertex, the company that makes Trikafta, to file a clinical assessment package to speed up the product review last July.
The matter has been raised in the House of Commons, where Health minister Patty Hajdu said Vertex has not applied to sell Trikafta in Canada. “In fact we reached out to the corporation to ask it to apply. We have assured it that we will expedite the review of Trikafta,” she said.
Not so, said Colin Le Fevre, associate director of public affairs at Vertex Canada. “We have been made aware of the minister’s promises to expedite through public updates by patient groups but we have yet to be contacted by the minister or Health Canada with the offer,” he said.
Vertex is supplying the drug to 151 Canadians through a special access program but that is only available to the sickest patients.
Jobbagy said he knows of people who are actively trying to get sick enough to qualify for the special access program.
Patients and patient groups can only look on in frustration as the only two parties that can facilitate the introduction of a life-changing drug drag their feet.
“We feel that everyone is blaming someone else and patients are caught in the middle,” said Kelly Grover, CEO of Cystic Fibrosis Canada.
Le Fevre said Vertex is reviewing the final guidelines from the pricing review board — the PMPRB — to determine the impact they will have. “We remain committed to achieve access for all eligible patients,” he said.
Government sources suggest the real problem is that pharma companies do not want other countries to follow Canada’s lead in driving down prices so aggressively.
We feel that everyone is blaming someone else and patients are caught in the middle
Vertex has done spectacularly well from Trikafta this year, reporting sales of US$918 million in second quarter of this year, from zero last year. That helped the company’s net income to soar 213 per cent year on year.
Health Canada sources say the company was told that it would be grandfathered from the new PMPRB guidelines if it applied and received a drug identification number in advance of the January coming-into-force date, yet it did not do so.
This is “entirely predictable”, according to the Canadian Health Policy Institute’s Skinner. “Companies want to launch product in places where prices don’t prejudice other markets,” he said, adding Canada is alone in using pharma-economic tests to regulate prices. For pharmaceutical companies operating internationally, widespread adoption of the practice could “crater the market,” he said.
The deals the federal government has struck with potential COVID vaccine suppliers include a provision that those companies will be exempt the new PMPRB guidelines. “That’s a tacit admission the regulations are a potential barrier,” said Skinner.
Ron Jobbagy said he understands the business case and that Vertex wants to make as much as it can before other products come on the market. “But why can’t they and the government get along to save lives?” he said.
“Every week that passes, someone crosses the bridge and is too far gone for the drug to help. We need to get this drug into the hands of people who need it now. My son will be okay for another couple of years but my daughter needs it today. She has one foot on the bridge already.”
He said that getting to a place where his kids only have to take three pills a day, and are not worried about hospitalization, would be a cure for many of the mental health issues his family faces. “If they have time to adjust and enjoy life, these two kids can and will become valuable Canadian citizens,” he said.
• Email: [email protected] | Twitter: IvisonJ
Copyright Postmedia Network Inc., 2020
