On October 3, 2012, Canada's Minister of Health, the Honourable Leona Aglukkaq, announced that Health Canada will be modernizing its framework for the designation, authorization and monitoring of orphan drugs.1

The Present System

Unlike in the United States, Canada does not have a separate regulation to address access to orphan drugs that have not gone through the usual new drug review and approval process.

Under the present Canadian system, in order for a patient to have access to an unapproved drug, his or her physician must make an application under the Special Access Program (known as the "SAP"). In each SAP application, a Canadian physician makes a request in respect of a single patient for up to a 6 months supply of an unapproved drug for emergency treatment purposes. 

The New Framework

The exact structure of the proposed framework is in the final design stages and not yet publicly available. Health Canada's announcement indicates, however, that the new approach will provide better access to both drugs and information for patients with rare conditions. 

As with the present SAP system, the new framework will maintain clinical trial-based evidence requirements and the safety and effectiveness of orphan drugs will be closely monitored.  

Health Canada advises that the new approach will focus on international information-sharing and collaboration with respect to orphan drugs. In this regard, the Orphanet portal is being launched in Canada. Orphanet is an online rare disease reference portal for both patients and healthcare practitioners, led by a consortium of over 35 countries. The Orphanet-Canada project will create a Canadian homepage, compile Canadian resources and engage Canadian stakeholders.

It is important to note that the new framework will apply only to unapproved orphan drugs. There is no word on whether the SAP system will remain intact and unchanged in respect of emergency access to unapproved drugs for non-rare conditions. 

Although the announcement is encouraging, implementation of a Canadian orphan drug program will take some time. In terms of immediate next steps, once Health Canada releases its framework proposal there will be a consultation process, during which the comments and feedback of stakeholders will be sought. We will continue to monitor this developing issue and report on the proposed framework once it is released.

Footnotes

1 Orphan drugs are loosely defined by Health Canada as drugs used to treat life-threatening, seriously debilitating, or serious chronic conditions that affect a very small number of patients (typically fewer than 5 in 10,000 persons).

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