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Orphan Drugs: The high cost of rare diseases

REGINA – When Liam McIntyre, 5, was four weeks old, his parents learned he had a rare form of cystic fibrosis.

Liam has difficulty digesting and swallows twelve enzyme pills a day to help with vitamin deficiencies. He has trouble gaining weight and is at risk of lung infection.

“Keeping him healthy and safe are different for us than for most parents,” said Kasey McIntyre, Liam’s mom, from their home near Estevan. “We have the same concerns but he’s more prone to infections.”

In August, Saskatchewan announced it would be the third province to fund Kalydeco, a highly expensive drug hailed as a miracle for CF patients.

“This is pretty big deal to help him breathe better and be more of a normal kid,” said Ken McIntyre, Liam’s dad.

Kalydeco costs an estimated $300,000 per person, per year. However the drug is only beneficial for a small subset of patients, likely only a handful in Saskatchewan, and hopefully Liam when he turns six.

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“We knew it was rare, but we didn’t realize that it was that rare,” said Kasey.

Expensive drugs for rare diseases are becoming more common as genetic breakthroughs are leading to an increase in therapies.

In the next four years, the growth rate for orphan drugs, as they’re called, is expected to double the growth in the overall prescription drug market. Saskatchewan funds ten orphan drugs, benefiting fewer than twenty people in the province.

“These not easy decisions. We recognize that not everything can be funded, within the system,” said Health Minister Dustin Duncan. “There will always be more demands than we have the ability to fund.”

Most of the drugs are in the six-figure price range, representing one per cent of Saskatchewan’s drug budget, or about $3-million, per year.

“The reasons the prices are so high is because the folks that are selling the drugs believe that’s the price that they can command for them. That’s what people are willing to pay,” said Don Husereau, a health policy consultant and associate with the Institute of Health Economics.

THREE-PART ORPHAN DRUG FEATURE:

Orphan Drugs: The high cost of rare diseases

Orphan Drugs: The politics of prescription funding

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Orphan Drugs: The obstacles facing families