Proposed Orphan Drug Framework For Canada

James Radke | RDR: Rare Disease Report | April 15, 2014

The gold standards for Orphan Drug Policies are those in the United States and the European Union. Canada is one of the few developed countries without a regulatory framework for orphan drugs. Hopefully, that will soon change.  Health Canada are currently developing a National Orphan Drug Framework. Two members of Health Canada recently published an overview of the framework being developed.

Health Canada's David Lee and Barbara Wong [1] stated Canada is working towards a new regulatory framework for orphan drugs that would span the lifecycle of the drug with the key objectives of the framework being to provide Canadians with better, timelier access to orphan drugs and to encourage and facilitate clinical research in the area of rare diseases.

Lee and Wong note 6 key features of the framework being developed.

Designation. The regulations being developed will list the criteria that a rare disease and an orphan drug must meet. Under the new framework being developed, Health Canada will review an application for designation and render a decision to designate the candidate drug as an orphan drug...