Health Canada Kills Planned Orphan Drug Framework for Broader Review

October 27, 2017

Daniel S. Levine

Durhane Wong-Rieger has long been working to get regulators in Canada to create an orphan drug designation similar to what is available in the United States and Europe. Wong-Reiger, president and CEO of the Canadian Organization for Rare Disorders and the mother of two children with rare diseases, thought by the end of this year, her long-long fought effort would be brought to a successful conclusion. It won’t.

Earlier this month Wong-Reiger had sent Maura Forrest, a reporter for the National Post, to the Health Canada website for information about the orphan drug framework that has been in the works since 2012. Forrest, who was working on a story about approvals of orphan drugs in Canada, discovered Health Canada had deleted all of those documents.

In a story about Health Canada unexpected action , Forrest noted that until October 6, the Health Canada website said that it was developing an orphan drug regulatory framework to “encourage the development of orphan drugs and increase the availability of these products” in Canada. It stated the agency expected to take action by the end of 2017.

Among the things the orphan drug framework was expected to do was harmonize the process between Canada and both the United States and the European Union. That would allow drug developers seeking a marketing approval for an orphan drug across jurisdictions to do so with a single application.

Health Canada says that it has launched a regulatory review 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,” according to the agency.

“The orphan drug framework was not set aside, and work on improving access to orphan drugs has not stopped,” Health Canada said in an email to questions from Rare Daily about the decision. “The Regulatory Review of Drugs and Devices Initiative advances and integrates work done for the orphan drug framework and offers several improvements.”

As an example of those improvements, it points to expanding the priority review process to decrease review time for products needed by the health care system, which was not in the orphan drug framework. That, the agency said, will improve access to these products.

While Health Cananda notes that 13 of the 38 new drugs approved in 2016 were for orphan indication, Wong-Reiger notes that only about half of the orphan drugs approved in the United States and Europe get approved in Canada. It says it continues to work with Stakeholders such as the Canadian Organization for Rare Diseases to help make orphan drugs available in Canada.

Wong-Reiger, though, met the Health Canada’s action with skepticism. She says she’s never been notified by the agency about its change in approach and that existing rules are inadequate to address the needs of patients with rare disease.

“Companies don’t know there’s a pathway so they don’t bother to come,” she said. “We’ve been working for ten years now and we thought we had it, and we look up and it disappears. And no one says to us we’re going to pull it and here’s how else we might do it. It’s unbelieve. There is no rationale.”

Durhane speculates that what may be behind the sudden action is pressure from the provincial governments, which don’t want to see an orphan designation because they believe it will give license to drugmakers to charge more for drugs that have it. That means absent the designation, drugmaker will likely be slower to pursue an orphan drug approval in Canada.

“It may delay entry with that,” she said. “They are okay with that. Access does not seem to be their primary concern.”

October 27, 2017

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