Health Canada plants ‘kiss of death’ on rare disease drug policy

Website drops references to framework plan

OTTAWA • Health Canada has quietly deleted from its website all reference to a planned framework for raredisease 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 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.

Canada’s orphan drug framework was first announced by the Harper government in 2012.

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.

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 noted.