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
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
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
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
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.
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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