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When Canada’s health ministers sit down together on Thursday and Friday in Winnipeg, talks are expected to revolve around Ottawa’s development of a national pharmacare plan. B.C. Health Minister Adrian Dix wants that plan to include a mechanism to grapple with expensive drugs for rare diseases – a fast-growing cost pressure on the public purse.

Conveniently, Health Canada has just provided him with fresh ammunition. Mr. Dix will present the case of Procysbi, one such costly drug for a rare disease approved by Health Canada. It costs 60 times more than a similar, though older, treatment.

Selling treatments for very rare diseases is a growing share of the pharmaceutical industry, with vast profits to be had in catering to so-called orphan diseases. As a result, it’s a growing challenge for those who pick up the tab – patients, the provinces or private medical plans.

For the past two decades in Canada, patients with nephropathic cystinosis, a potentially fatal kidney disorder, have been treated with a relatively low-cost drug called Cystagon, which costs about $5,000 a year, per patient.

But Health Canada last year approved Procysbi – which has the same chemical base as Cystagon, but in a longer-acting formula. That decision effectively prevents doctors from prescribing the old drug.

The new drug will cost $300,000 a year, per patient, for a drug that must be taken for life.

In B.C., decisions on public funding of costly drugs for rare diseases are reviewed by the Drug Benefit Council. The province paid for Cystagon and the new replacement therapy is still under review.

“This puts everyone – especially the parents with children with cystinosis – in a terrible position,” Mr. Dix said in an interview.

“We are paying huge amounts more to an American pharmaceutical company out of public health funds, with no real material advantage – and that is pretty shocking.”

There is a patchwork of prescription drug coverage across the country, with each province determining its own set of rules on what is covered. Private insurance programs pay for some drugs. And sometimes, patients fall between the cracks and have to pay for their own prescriptions.

Cystagon was available to patients through Health Canada’s Special Access Program, which considers requests for access to drugs that are unavailable for sale in Canada from practitioners treating patients with serious or life-threatening conditions, when conventional treatments have failed, are unsuitable or unavailable.

With the approval of Procysbi in Canada, there is now a drug treatment available for nephropathic cystinosis, so patients can no longer access Cystagon unless they have a medical reason that would preclude the use of Procysbi.

Mr. Dix says the decision grants a virtual monopoly to the manufacturers of Procysbi and undermines efforts across the country to rein in unfair drug pricing.

“Procysbi is under investigation for excessive pricing. We feel Health Canada needs to take some steps here.“

There are only four patients in British Columbia with cystinosis, so the annual cost is not, by itself, a major issue. But the manufacturers of Procysbi are not alone in pushing high-cost treatments.

Currently, B.C.’s PharmaCare program is reviewing 13 different drug treatments that are being requested for a total of 243 patients.

The cost for these treatments, on average, would be $628,000 a year for each patient.

By contrast, PharmaCare currently costs the average patient about $1,250 per year. And those in poverty underutilize prescriptions they need because the deductibles are too high. (B.C. is reducing or eliminating deductibles for low-income families starting next year.)

With the current trends, Mr. Dix expects payments for expensive drugs for rare diseases to rise in the next five years by hundreds of millions of dollars. It is, as Mr. Dix has learned in his first year on the job as health minister, a tough thing to say ‘no’ to new drug treatments for those patients struggling with rare diseases.

The B.C. government is working with other provinces to negotiate lower prices for some of these rare disease treatments, but on their own, the provinces have little clout. Under a national pharmacare program, Canada would have a stronger bargaining hand.

Ottawa is studying what form a national pharmacare program may take, with an advisory council now travelling the country to consult with the provinces who pay the vast majority of prescription-drug costs.

Mr. Dix says he wants this national initiative to succeed, but Health Canada can’t work at cross-purposes when it makes decisions on drug approvals for rare diseases.

“Let’s face it: There is a greater interest by the pharmaceutical companies in expensive drugs for rare diseases, they have a system for getting their rate of return, which is by setting extreme prices for those drugs and putting the pressure on to get their prices,” he said.

“These tactics have to be dealt with and not protected.”

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