Canadians Face Hurdles Obtaining Drugs for Rare Diseases

By: Sam Garnett

February 25, 2017

Canadians grappling with rare disease can have major difficulties accessing medication they need, Dave Chan reports in the Globe and Mail. Drugs that treat rare disease can be extremely costly or difficult to obtain for patients in Canada, even when the drug is approved by the US FDA. Canadian clinical trials can be nearly impossible to undertake, given the extremely small populations with these diseases, yet no formal structure is in place to assess drugs when clinical trials are done elsewhere. Canadians who need medication to treat rare diseases, such as Duchenne Muscular Dystrophy or Prader Willi Syndrome, can undertake significant financial burden and debt in order to get medication that can treat their illnesses, as well as facing bureaucratic hurdles in obtaining the drug or having it approved by their insurance.

”"We're the only developed country that doesn't have an orphan-drug framework," said Dr. Durhane Wong-Rieger [to Dave Chan], president and chief executive officer of the Canadian Organization for Rare Disorders. "This is really a challenge."”

Such a policy could potentially reduce red-tape in approving drugs, while giving patients who suffer from extremely rare diseases more resources to find and fund potential treatments. Not only could these measures improve the quality of life for Canadians with rare disease, but could also prevent future complications requiring additional costly medical intervention.

Dave Chan writes:

“Health Canada's website says the department is in the process of 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," but no other information is available on timing. Federal Health Minister Jane Philpott's office did not provide information about the status of a rare-disease framework in Canada.”

Read the full report in the Globe and Mail.