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Across Canada, dozens of parents are shaking their heads in disbelief that Health Canada is making them switch their sick children to a drug that costs more than $300,000 a year. Especially when they've been happily using an older version of the same drug that costs a fraction of the price.

"It's a ridiculous spot to put parents in," said Trevor Strauss, of Waterloo, Ont., who was stunned to learn that access to his daughter's life-saving drug was being cut off now that a new, more expensive version of the same drug had been approved by Health Canada.

There's no reason for the drug to be this expensive except for profit margins. - Trevor Strauss

Strauss's 10-year-old daughter Gabbie has cystinosis — a deadly genetic disease that destroys her kidneys. She's been taking a drug called Cystagon (cysteamine) for most of her life, but she won't be able to get the drug anymore.

The new form of the drug licensed in Canada, called Procysbi (cysteamine delayed-release), contains the same active ingredient as Cystagon. The difference is that the new form has a special coating that delays the drug's absorption so the chemical is released more slowly in the body. It means taking the drug every 12 hours instead of every six.

Cystagon cost $10,000 a year for Gabbie and it was covered by the Ontario government. Strauss has been told Procysbi will cost $320,000 a year, every year, for the rest of his daughter's life.

Gabbie's father, Trevor, says she is thriving on Cystagon, which she has been taking for nine years. The family doesn't have private insurance and Trevor isn't sure who's going to pay the $320,000 a year for Procysbi. (Submitted by Trevor Strauss)

The provinces have not yet agreed to cover the cost of Procysbi, and Strauss doesn't have private insurance.

"There's no reason for the drug to be this expensive except for profit margins," he said, adding that the new drug is less convenient for his daughter because her meals must be precisely timed and her schedule for other drugs will be disrupted.

'Angst and concern from families'

Specialists who treat cystinosis patients are caught in the middle. They would like to have the choice to prescribe both drugs; some patients would prefer the new formulation because it means they can take fewer drugs.

"It's created an awful lot of angst and concern from families who are very worried that they'll have a gap in treatment," said Dr. Julian Midgley, a pediatric nephrologist with Calgary Health Region who treats most of the cystinosis patients in Alberta.

"It's also caused a lot of angst because some families have taken the view that it's so ridiculously expensive, how can I possibly be using something that costs as much as my house every year."

​Cystagon, produced by Mylan, was never formally licensed in Canada but was available through a special access program operated by Health Canada.

But if a drug gets approved for market in Canada, other unapproved forms of the drug are no longer allowed to be brought in through that program. The fact that the approved drug costs 3,000 per cent more is irrelevant.

Horizon Pharma told CBC News in an email that the price of Procysbi in Canada follws the rules for "a breakthrough medicine," as set by the Patented Medicine Prices Review Board (PMPBR), a federal agency that limits the price of Canadian prescription drugs to the average price in eight other countries including the U.S., England and Germany.

But CBC News has learned that the PMPRB is actively investigating the price of Procysbi.

"Whether that price complies with the law remains to be determined," Jeff Wright, PMPRB communications officer said in an email.

Horizon disclosed in its 2017 U.S. SEC filing that part of its business strategy for the drug is "to drive conversion of patients from Cystagon to Procysbi."

Now Health Canada is telling doctors and patients they can no longer access Cystagon unless the doctor can provide a valid medical reason, leaving patients and their families caught between the pharmaceutical company's high price and Health Canada's regulations.

Cystagon is being replaced by a new form of the drug called Procysbi, which contains the same active ingredient as Cystagon but has a special coating that delays the drug's absorption so the chemical is released more slowly in the body. (Trevor Strauss)

"I am really angry about the price. I feel like it's obscene, actually, the new price on Procysbi," said Crystal Walker, who has two daughters in Alberta with cystinosis.

Right now, Cystagon costs less than $6,000 a year for both children. Walker says she was told by Horizon that the new drug would cost about $150,000 for her seven-year-old, based on her weight. That price will climb to more than $300,000 every year for life when she's an adult.

"I don't see what reason it is to be that expensive," Walker said.

The families are confused about the high cost in part because both versions of the drug were developed by the same academic scientists at the University of California San Diego, who were funded by the patients themselves, through bake sales and golf tournaments and other fundraising via the Cystinosis Research Foundation.

"From my point of the view, the cost is outrageous," said Midgley.

It's estimated that cystinosis affects fewer than 100 patients in Canada. It's not yet clear who is going to pay for their Procysbi prescriptions.

The provinces through the pan-Canadian Pharmaceutical Alliance are in talks with Horizon Pharma, trying to negotiate a lower price.

Horizon has told some patients it will supply Procysbi at no cost for a limited time while the price negotiations are happening.

Walker said the Horizon executives told her they would cover the cost of Procysbi for three months. "And we said, 'What happens at the end of three months? Does that mean we run out of drugs?' They said the provinces have to do something."

Horizon Pharma invited families to a meeting

Meanwhile, based on questions from CBC News, Health Canada said it is looking into contacts between Horizon company officials and Canadian patient families.

Last November, the company invited Strauss to fly to New Orleans for a meeting about cystinosis treatment, covering the travel expenses.

Erin Little and her husband, of Port Elgin, Ont., were also invited to the meeting. Her daughter has cystinosis and is currently taking Cystagon. Little said Horizon's invitation came "out of the blue."

My blood was boiling. I couldn't believe I was in this conversation. They just wanted to teach us what an advocate is. - Erin Little

Horizon Pharma spokesperson Matt Flesch told CBC News, "Representatives from cystinosis patient organizations were invited to help us better understand their needs, and these invites were extended in response to ongoing requests for information about our efforts to make Procysbi available in Canada."

Little said the meeting seemed to be a waste of her time.

"My blood was boiling," Little said, adding that the group was shown a presentation on advocacy.

"I couldn't believe I was in this conversation. They just wanted to teach us what an advocate is."

Strauss said he was asked to sign a confidentiality agreement preventing him from disclosing details of the meeting. He refused to sign it. "No one signed it," he said.

Back in Canada, Little said she was invited to have weekly phone conversations with Horizon Pharma executives, including vice-president Rob Metz and Canadian general manager John Haslam. Two other Canadian parents were also on the calls. The Horizon executives offered information about the status of Procysbi in Canada and advised them on options to have the drug covered.

Flesch said patient organizations have been contacting the company for updates on Procysbi's availability in Canada.

Health Canada says communication between industry representatives and patients could be subject to regulations governing drug advertising.

"If Health Canada deemed the situation to be advertising for the sale of a drug, the activity would be a promotion prohibited under the Food and Drugs Act," Health Canada told CBC News in an email. The agency said it must clarify the circumstances "to determine the legality of such activity."

"It is Health Canada's position that patients should obtain information about a prescription drug from their healthcare professional."

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