In the last federal budget, the Canadian government committed one billion dollars over two years, starting in 2022, to cover the costs of health care related to rare diseases. Canada’s relative lack of a rare disease policy opens conversations on what should be included in an effective strategy to provide Canadians better access to drugs for these ‘orphan diseases.’

Dr. Joel Lexchin, an associate professor at U of T’s Department of Family and Community Medicine, and Nicholas Moroz, a recent Master of Public Health graduate at U of T, published a paper on the subject. It analyzed whether a rare disease policy similar to Australia’s could be effectively implemented in Canada to broaden the availability of treatment options for patients with rare diseases in the country.

The state of orphan diseases in Canada

Orphan diseases are rare diseases that affect approximately five people in 10,000. These diseases have been the subject of policy discourse in Canada for more than two decades, yet no policy has been set, according to the authors. 

Now, as legislators have a go-ahead from the government for investing in orphan drug treatments, they must also consider the best policy for regulating them.

Health Canada currently has two regulatory pathways in place that facilitate the speedy delivery of orphan drugs to the market. These provide patients access to orphan drugs in the interim of passing a formal policy, according to the authors. 

The first is the priority review, which takes 180 days, instead of the standard 300-day review. The second is a 200-day conditional approval of drugs based on limited clinical data. 

Would an orphan drug policy be beneficial?

To determine if a drug policy such as Australia’s would be beneficial to improving access to drugs, Lexchin and Moroz looked at the frequency and speed drugs assigned an ‘orphan’ status by the US Food and Drug Administration (FDA) were approved by Australia and Canada. 

The paper does not suggest that an orphan drug policy like Australia’s would help improve access to medicine in terms of speed or variety. 

The conclusions showed that both Health Canada and the Australian Therapeutic Goods Administration (TGA) approved 74 of the 119 drugs designated for orphan diseases by the FDA. Health Canada shows more propensity for approving drugs, as it approved an additional 11 drugs, while the TGA only approved four other drugs. 

There was no significant difference between the approval rate by Canada and Australia. 

Why do we even need an orphan drug policy?

Australia’s orphan drug policy is focused on getting drugs to the market rapidly. However, the authors noted that Canadian lawmakers could help patients by considering other objectives, including incentives for drug development and economic accessibility. 

While Australia implemented fee waivers for applications for orphan drug status and annual registration, its policy does not guarantee market exclusivity, grants, or tax credits targeted at orphan drug research. Lexchin and Moroz think that such a framework would help provide developmental incentives for new drugs, which is one of the objectives that they argue Canada should include in orphan drug framework. 

When it comes to economic accessibility of orphan drugs, the study recommends that the government include public subsidies and reduce the price charged by companies.  

There is no evidence that having an orphan drug framework similar to Australia’s would make Canada a more attractive market for orphan drugs, wrote the authors. They concluded that in order for Canada to provide quicker access to orphan drugs, it is essential for the policy framework to be much different than the one in place in Australia.