What is a rare disease?

A “rare disease” (also called an "orphan disease") is an illness that affects a small percentage of the population, thereby limiting scientific research, clinical expertise and patient access to effective treatment options.

What is an orphan drug?

An "orphan drug" is a medication or other medicinal product used to treat a rare disease.

They are called “orphan drugs” because due to their limited market, they are rarely adopted by pharmaceutical companies.

The Drug Approval Process in Canada

Before any drug can be sold on the Canadian market, it must go through Health Canada’s stringent drug review process. That's why even when an "orphan drug" exists or is developed elsewhere to treat a "rare disease", bringing it to the Canadian market can be a long and expensive process.