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Orphan Drugs: The obstacles facing families

REGINA – The Creighton family in Saskatoon has three kids with phenylketonuria, PKU, a rare genetic disorder that if left untreated can lead to severe intellectual disability.

“Most people tend to stop having children once one or two are diagnosed with PKU. We didn’t take that option,” said Steven Creighton. “We didn’t want to let PKU run our lives.”

The kids are unable to break down a certain amino acid found in foods with protein.

Four years ago, Health Canada approved a drug called Kuvan, which costs upwards of $100,000 per year.

“Most importantly for us is that Kuvan is sort of like a chemical helmet for them. It protects their brain,” said Steven.

Kuvan is funded in Saskatchewan; however, the Creightons have been told their kids don’t meet the criteria. They need to somehow prove the drug is working, difficult to do since they’ve all been treated since birth.

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However, the manufacturer, BioMarin, has been providing the drug free of charge.

In a statement BioMarin said, “Patient eligibility for compassionate use is determined on a per patient basis, and as of September 2013, PKU patients who require KUVAN therapy have to access public funding or private insurance for reimbursement.”

“They’ve committed to provide it for as long as necessary, until they’re covered, but I don’t like that kind of assurance,” said Steven.

Since Kuvan was added to Saskatchewan’s public drug program last September, no one in the province has actually met the criteria. In fact, of the ten provincially funded drugs for rare diseases, three have never been paid for by the province.

“Just because today we may not have a patient doesn’t mean we wouldn’t necessary still ensure that if that day were to come, that we would fund that drug,” said health minister Dustin Duncan.

Industry experts predict budgets for orphan drugs will go from the current one per cent to 20 in the next ten years.

“I think in the future we’re going to see that tried and true mechanisms of looking at drugs and evaluating them are going to come to the fore and we may not be treating these drugs as special as we are today,” said Don Husereau, a health policy consultant and associate with the Institute of Health Economics.

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Orphan drug funding is also on the federal government’s radar. Two years ago, it announced it wanted to create a rare disease framework to help patients get better access to drugs.

Health Canada tells Global News the framework is close to being implemented and is planned to be the first set of proposed regulations coming out of a bill titled Vanessa’s Law.

THREE-PART ORPHAN DRUG FEATURE:

Orphan Drugs: The high cost of rare diseases

Orphan Drugs: The politics of prescription funding

Orphan Drugs: The obstacles facing families

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