Health Canada gives 'kiss of death' to planned policy for rare-disease drugs


OTTAWA — Health Canada has quietly deleted from its website all references to a planned framework for rare-disease drugs that dates back to 2012 and was intended to improve the availability of such drugs in Canada.

Canada is one of the only developed countries without a regulatory framework for rare-disease drugs, also known as orphan drugs.

Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders, said the decision “certainly seems to be the kiss of death” for the orphan drug framework.

“I am totally, totally devastated that they have taken such a big move to totally eliminate it,” she said.

Until Oct. 6, a Health Canada webpage claimed the department was “developing an orphan drug regulatory framework that seeks to encourage the development of orphan drugs and increase the availability of these products on the Canadian market.” It also promised consultations that were “expected to take place before the end of 2017.” The webpage has since been removed.

Orphan drug regulations had been listed as one of the proposals that Health Canada expected to bring forward between 2017 and 2019, but the initiative has now disappeared from the department’s regulatory plan.

Conservative MP Tom Kmiec, whose three children have a rare disease called Alport syndrome, said the change is “surprising.”

“Obviously it’s not a priority if it’s not in Health Canada’s forward regulatory plan,” he said.

I am totally, totally devastated that they have taken such a big move

Canada’s orphan drug framework was first announced by the Harper government in 2012, but has sat on the back burner for several years since then. Elsewhere, such frameworks provide incentives for pharmaceutical companies to develop drugs for small numbers of patients suffering from rare diseases, which are defined in Europe as those affecting fewer than one in 2,000 people. Incentives can include tax credits for clinical trial expenses, research grants, expedited approvals and waived approval fees, and market exclusivity for a number of years.

Without a framework in Canada, Wong-Rieger said, pharmaceutical companies may not bother to apply for marketing approval for their orphan drugs in this country. “Companies don’t know to come and they don’t know to come early and they don’t know to do clinical trials here,” she said.

Wong-Rieger said she was “quite dismayed” to learn that Health Canada has pulled orphan drugs from its regulatory plan. “Now it seems that without even letting us know, they’ve decided basically to discontinue it.”

Health Canada says it has launched a new regulatory review of drugs and devices focused on improving access to drugs. “Many elements initially proposed as part of an orphan drug regulatory framework are now being considered more broadly for all drugs as part of this initiative,” a spokesperson told the Post by email.

But the department offered no timeline for that new review, Kmiec pointed out. “That worries me, because patients don’t have time for that.” He believes much of the previous government’s work on the issue has been “undone.”

Wong-Rieger said it’s not enough to treat orphan drugs the same as other pharmaceuticals, when other countries provide incentives for their development. “The problem is that the rest of the world does not get the message that we actually are trying to approve those drugs,” she said.

Internationally, the production of orphan drugs has ballooned in recent years, due in part to orphan drug policies. In the U.S., more than 500 orphan drugs have been approved for marketing since the passage of the Orphan Drug Act in 1983, compared to fewer than 10 in the preceding decade.

But fewer of the drugs have been approved in Canada. A document tabled recently in the House of Commons in answer to a question from Kmiec shows that Health Canada granted marketing approval to 85 orphan drugs between May 2013 and June 2017. In the U.S., the Food and Drug Administration approved between 30 and 45 orphan drugs each year between 2013 and 2016.

In some cases, Canadian patients can access orphan drugs not approved in Canada through Health Canada’s Special Access Programme, but may have to pay for them out-of-pocket. Because they target small populations, orphan drugs are often hugely expensive.

Still, Matthew Herder, director of the Health Law Institute at Dalhousie University, said the change in plans for an orphan drug framework is “a nice example of Health Canada maybe paying attention to the evidence.”

He believes it’s “far from clear” that existing policies in other countries actually work as they’re supposed to. Many of the orphan drugs that are approved are for rare cancers, he said, because pharmaceutical companies know that physicians will often prescribe those medications for cancers other than the ones they’ve targeted, meaning they can be very profitable.

“So the vast majority of rare diseases still don’t have drugs available,” he said. “If the purpose of an orphan drug law is really to encourage research into diseases that the market is neglecting, I’m not sure we’re doing that.”

Drug companies can also target rare types of more common diseases and take advantage of orphan drug incentives, all the while knowing their drugs can actually be used to treat the more common disease, he said — a practice known as “salami-slicing.”

“They’re using that knowledge to game the regulatory structure,” he explained.

Herder’s research has shown that nearly 75 per cent of orphan drugs approved for marketing in the U.S. had also been approved in Canada in 2012, though it does take longer for new drugs to be approved here.

He said Canada could use an orphan drug policy, but added that it should look “nothing like” those in the U.S. or Europe.

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