Regulatory Focus™ > News Articles > In Landmark Reversal of Policy, Health Canada Announces Launch of Orphan Drug Policy

In Landmark Reversal of Policy, Health Canada Announces Launch of Orphan Drug Policy

Posted 04 October 2012 | By Alexander Gaffney, RAC 

Canadian Health regulators have announced the launch of a new orphan drug regulatory approach aimed at getting more treatments to patients with rare diseases.

In a 3 October 2012 announcement, Leona Aglukkaq, Canada's minister of health, said the Canadian government was committing to a, "New approach that will better support the development and authorization of drugs for rare diseases and [the] launch of a new Web portal to assist patients in finding the information and services they need."

Rare diseases, defined in Canada as any disease affecting fewer than five in 10,000 patients, have historically been an unprofitable and difficult area in which to promote the development of new therapies. Because of the costs associated with drug development and regulatory approval, companies have usually focused on commercializing therapies for diseases with larger pools of patients.

This paradigm began to dramatically shift in 1983 in the US under the Orphan Drug Act, which provided additional market exclusivity and monetary incentives for companies that agreed to develop new therapies. Over time, legislators have used a similar model to promote other products, such as medical devices and, as of July 2012, antibiotics.

Reversal of Long-Established Policy

Until now, Canada has eschewed this approach, instead relying on Special Access Programs (SAPs) applied to on a case-by-case basis, through which drugs to treat rare diseases may be obtained. One of the primary considerations for Health Canada was apparently cost. The price for many rare treatments can often be prohibitive due to the small patient population relative to total development costs. Because of the country's public healthcare system, all costs are ultimately borne among consumers.

Regulators conceded that this approach was not working for Canadian patients, and was often creating time-consuming delays.

"A regulatory framework designed and used specifically to approve drugs to treat small, vulnerable populations will more effectively address this need," Health Canada said in a statement. "A key focus of this new approach will be on international information-sharing and collaboration for the development and regulation of orphan drugs."

The new regulatory framework is still in the works, said Health Canada, but will come to affect the development, evaluation and approval of drug products in Canada.

Regulators said the new approach will also allow them to gather and share information on the registration of clinical trials, help patients participate in rare disease trials and promote a consistent policy for rare disease treatment coverage.

'Dressed and Ready for Action'

In addition, Health Canada said it plans to join Orphanet, a rare disease database that will allow patients to find information on where to find treatments to help manage their conditions. The system has until now been used exclusively in the EU, Africa and Asia.

In a statement, the Canadian Organization for Rare Disorders (CORD), a rare disease advocacy organization, praised the move, noting that the initiative overturns a 16-year-old policy against the formation of orphan drug regulations, and 30 years after the US affirmed the need for the same.

The group also said the new Orphan Drug Policy would pave the way for guidelines for newborn screening, diagnosis and the creation of centers of excellence for treatment.

"We hope this announcement will stimulate the provinces to work together to develop a national program to review and fund drugs for rare diseases," said Maureen Smith, a patient with a rare disorder and secretary of CORD.

"Canada may be a bit late to the party, but we are dressed and ready for action," CORD quipped.

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