Earlier this month, Health Canada did some substantial editing to its website, removing all references to a planned regulatory framework for orphan drugs.

Orphan drugs are medications that treat rare diseases. The framework was an effort to help increase their availability for patients and foster their development by companies and researchers.

Currently, Canada is one of the only developed countries in the world without such a framework.

Read: Plan sponsors bracing for onset of new orphan drugs

“The challenge we have in Canada is actually attracting research and development, clinical trials on orphan drugs for rare diseases, and getting researchers and companies who want to come to Canada early, who want to invest in clinical studies in Canada, who actually help support the development of these drugs, . . .” says Durhane Wong-Rieger, president and chief executive officer of the Canadian Organization for Rare Disorders.

The now-deleted framework had been in the works for more than five years, according to Wong-Rieger.

“We kept getting promises that it would be implemented, they needed some additional legislation to actually be able to fully implement certain aspects about it, especially the post-marketing, but the Conservative government never actually submitted it before the election,” she says. “And we had full hopes that when the Liberal government came in, they would be able to pick it up and actually be able then to move it forward.”

While there had been some wavering and delay from Health Canada and some signs that it could put the framework on hold, Wong-Rieger notes it was, in fact, ready.

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“We’ve got a fully developed framework, everybody who looks at it says it’s good, it’s been talked about in conferences internationally . . . and everybody has really indicated, ‘Yes, this is exactly what Canada needs if, in fact, you can bring this in.’”

As for a replacement, Health Canada said in a statement that it’s putting together a regulatory review of both medications and medical devices. It noted it would develop a plan to “work together with our health partners at home and abroad, provide more timely access to drugs and devices, including drugs for rare diseases, enhance the uses of real-world evidence to support regulatory decisions for drugs and devices.”

Speaking specifically to the orphan drug framework, Health Canada stated: “Improving access to necessary prescription drugs is a priority for the minister of health. It is in her mandate and funded as part of budget 2017. The orphan drug framework was not set aside, and work on improving access to orphan drugs has not stopped.

“The regulatory review of drugs and devices initiative advances and integrates work done for the orphan drug framework and offers several improvements. For instance, 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, will improve access to these products.

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“Since 2012, Health Canada has been working with key stakeholders, such as the Canadian Organization for Rare Diseases, to improve access to orphan drugs by encouraging the development and availability of these products. That work continues.”

Wong-Rieger agrees the work will continue.

“We have to. We have thousands of families that are dependent on us, so we’re continuing to, we will continue to work obviously with Health Canada to see how we can get the drugs in,” she says.

“We think that on an individual basis that the members of Parliament are very supportive. We think that just from a government point of view, they’re hesitant to actually make the action. And I think that’s too bad. I don’t want to say it’s all the provinces that are pushing back and [are] making this happen, but honestly, I cannot understand why we just don’t have the regulatory framework that every other developed country has. There’s no good reason for it.”

Read: Drug Plan Trends Report: Alarm about costs sparks ‘monumental shift’

Copyright © 2018 Transcontinental Media G.P. Originally published on benefitscanada.com
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ric ellens:

Prometic Life Sciences is doing clinical studies on Orphan diseases

Saturday, November 25 at 8:05 pm | Reply

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