Health Canada
Symbol of the Government of Canada

Common menu bar links

About Health Canada

An Orphan Drug Framework for Canada

News Release: Harper Government Takes Action to Help Canadians with Rare Diseases

What are Orphan Drugs?

Orphan drugs are those drugs used to treat rare diseases. A rare disease is a life-threatening, seriously debilitating, or serious chronic condition that only affects a very small number of patients (typically less than 5 in 10,000 persons). Various international health organizations estimate the number of rare diseases at over 7,000, with the vast majority of these being linked to genetic factors.

New rules and approaches to the development, evaluation and approval of these drugs are needed to address the fact that the small size of the patient population makes it scientifically difficult and most often commercially impractical for drug companies to develop and market orphan drugs.

The New Framework

Health Canada is developing a modern framework for the designation, authorization and monitoring of orphan drugs that will provide a significant benefit to Canadians with rare diseases and spur research and innovation in Canada.

A key focus of this new approach will be on international information-sharing and collaboration for the development and regulation of orphan drugs. Enabling Canadian scientists and regulators to participate with trusted global counterparts will make better use of scarce resources and benefit Canadian patients.

The new framework will maintain evidence requirements based on clinical trials and will be supported by greater information sharing amongst international partners who are committed to pooling scarce resources for maximum benefit. Once authorized, drugs will continue to be closely monitored for effectiveness and safety while in use.


The proposed framework is in the final design stages and will soon be targeted for public consultation. Comments and feedback gathered during the consultation will be incorporated into a final version of the proposal.