The primary predicament regarding patient access to pharmaceutical treatments for rare disease, known as orphan drugs, is their prohibitive cost, upwards of $300,000 (about €213,000) a year per patient in Canada. Therefore, many orphan drugs do not get recommended for provincial reimbursement. Further complicating the issue is that some orphan drugs, available in other countries, are not approved for sale by Health Canada because the research sample size is too small to pass Health Canada’s safety and effectiveness requirements using common evaluation methods.
On 3 October 3 2012, Health Canada (the federal ministry of health) announced two initiatives aimed at improving patient information about, and access to, treatments for rare diseases. Canada joined Orphanet, an online information site about rare diseases and their treatments, designed for patients and healthcare providers. On access, Health Canada promised to develop a national framework to address the designation, authorization and monitoring of treatments for rare diseases after years of refusing to do so.
These initiatives came as the first federal announcement concerning orphan drugs since the 1997 Drugs Directorate (DD) decision that Canada had no need for an orphan drug policy given the relatively small number of individuals who contracted these rare diseases. The DD justified its position on the basis that the cost of an orphan drug policy was too high relative to the benefits for the very few who would gain from a change in policy.
It was not until 2000, when the Canadian Organization for Rare Disorders (CORD) received media attention for partnering with various orphan disease associations to lobby for change. By 2000, CORD had 150 different rare disease organizations as members. This collective action was articulated in a statement that was reiterated in an extensive number of articles throughout the mid 2000s and leading up to the 2012 decision; "There are about 6,000 rare disorders. Each disorder may only have an impact on a small number of people but, taken together, they affect about three million Canadians", representing approximately one in ten Canadians.
CORD’s momentum carried through the mid 2000s. Health Canada, the provincial governments and the pharmaceutical industry all received criticism for their inaction on orphan drugs. Canada was unfavourably compared to other developed countries. Beginning in 2007, CORD began to present policy options and solution, giving the organization direct access to political actors. They presented arguments to both Parliament and Senate and were recognized as contributing to the federal government’s change in position and the 2012 initiatives.
In summary, primarily through collective mobilization and policy access through the promotion of concrete policy options, CORD pushed the need to address the orphan drug problem onto the federal decision agenda.
For an analysis of this reform, see: https://escarpmentpress.org/hro-ors/article/view/1186