Pitfalls in Canada’s Approach to Orphan Drugs

Pitfalls in Canada’s Approach to Orphan Drugs

“While each disease on its own may be rare, together they represent countless individuals who desperately require better access to medication.”

A country’s approach to healthcare is a defining characteristic that alters the day-to-day lives of its citizens. This is especially evident in the United States where, in recent years, healthcare has become a hot topic worthy of extensive media coverage. In Canada, however, the healthcare system has remained largely static, much to the dismay of rare disease patients and advocates alike. Since 2005, many Canadian families have been rallying for the implementation of a rare disease drug plan seemingly to no avail. In 2015, the Canadian Organization for Rare Disorders (CORD) announced their strategy for rare diseases, calling those at Parliament Hill to action.1 This seemed like an important step forward but, as of 2017, such a plan has still not been enacted.2 Although orphan drugs have been included in Canada’s Forward Regulatory Plan for 2017-2019, little additional information is available on what this entails.3 It appears that Canada’s healthcare falls behind the United States and the European Union in that they do not have a federal level rare disease drug plan. It is time for Canada to get serious about a rare disease strategy, using plans from both within and outside of its borders for inspiration.

“Orphan drugs” is the term often given to rare disease medication. These drugs are incredibly difficult and expensive to produce, given the small numbers of patients who need them. Many countries, including the United States and parts of the European Union, have established orphan drug policies to incentivize the research and development of these drugs.4,5 Canada, however, is not one of these countries. Currently, Canada does not have a standardized national plan, meaning it is up to the individual provinces and territories to handle this problem.2 The absence of a plan means that there may be discrepancies between what drugs are publicly insured between regions, a facet that could be the difference between health and pain for many families with rare disease patients.

Provinces within Canada have developed their own systems for rare disease drugs, with varying degrees of success. In Ontario, a Drugs for Rare Diseases Framework has been established to review potential rare disease drugs, including those used to treat MPS-I, Hunter’s Syndrome, Cryopyrin-Associated Periodic Syndrome, Pompe disease, MPS VI, and Niemann Pick Type C.6 The government of Ontario provides assistance with funding these drugs for rare disease patients. A similar system is used in New Brunswick, where drugs that have been approved by Ontario’s system are funded by the New Brunswick Drugs for Rare Diseases Plan.6 In Alberta, individuals who have been registered through the Alberta Health Care Insurance Plan for at least five years are eligible for the Rare Disease Drug Coverage Program.7 As with the Ontario and New Brunswick plans, the number of drugs covered is extremely limited. The Alberta Human Services Drug Benefit Supplement 2017 only lists those drugs used to treat Gaucher’s disease, Fabry disease, MPS-I, Hunter disease, and Pompe disease.7 Though this plan is certainly impactful for individuals with these diseases, there are some clear drawbacks. Firstly, individuals that had previously been living outside of Alberta must be moving from a province or territory in which they had coverage.7 They must then go through the process of registering under the Alberta Health Care Insurance Plan. Individuals moving to Canada, or individuals who were not able to receive coverage in their home province, will, therefore, be denied funding. Additionally, the plan will only provide a one-month supply of the prescription at any time7, thereby putting restrictions on the patients’ ability to travel. Although these provinces do have their own systems, the creation of a federal plan is still necessary to allow individuals with rare diseases the comfort of knowing they will be granted the funding they need, regardless of what province or territory they live in.

In contrast to Canada, the United States Food and Drug Administration (FDA) implemented an Orphan Drug Act in 1983.8 The Act, still in place today, grants a special status to drugs that will be used by fewer than 200,000 individuals in the United States,5 resulting in a variety of incentives including an exemption from prescription drug fees for patients.9 The Act promotes research and development of rare disease drugs and brings patients closer to the medication they desperately need. The American system is not without faults though, and over time the system has accumulated a massive backlog of requests for drugs to be granted orphan status. The FDA is aware of this and has included it in their plans for modernization, stating that they will create a “team of senior, experienced, proficient…reviewers to focus solely on reviewing orphan drug designation requests, starting with the oldest ones first.”10 The backlog emphasizes the demand for rare disease treatment, showing that while each disease on its own may be rare, together they represent countless individuals who desperately require better access to medication. By assembling a similar system in Canada in which a large number of officials are appointed to review cases for the entire country, Canada will present a more united front in aiding rare disease research.


Rare diseases affect one in twelve Canadians – a sizable portion of the population – and yet few medications are available for these patients. Even when medications are available, it often seems like individuals must jump through hoops to get to them.

Rare diseases affect one in twelve Canadians1 – a sizable portion of the population – and yet few medications are available for these patients. Even when medications are available, it often seems like individuals must jump through hoops to get to them. These problems do exist in other countries, but Canada’s lack of a unified national plan for orphan drugs identifies it as a country falling behind in rare disease care. The creation of such a program would finally answer the prayers of so many individuals who have rallied in favour of this, unifying provinces in their approach, and putting the force of the entire country behind the movement for better rare disease care.


Works Cited:

1. Canadian Organization for Rare Disorders. Canada’s Rare Disease Strategy. https://www.raredisorders.ca/canadas-rare-disease-strategy/.

2. Menon D, Clark D, Stafinski T. Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now? Healthc Policy. 2015;11(1):15-32.

3. Canada H, Canada H. Regulatory Initiative: Regulations Amending the Food and Drug Regulations - Orphan Drugs - Forward Regulatory Plan 2017-2019. aem. https://www.canada.ca/en/health-canada/corporate/about-health-canada/legislation-guidelines/acts-regulations/forward-regulatory-plan/2016-2018/regulatory-initiative-regulations-amending-food-drug-regulations-orphan-drugs.html. February 26, 2013.

4. European Medicines Agency - Overview - Orphan designation. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp.

5. E-CFR: Title 21: Food and Drugs PART 316—ORPHAN DRUGS. Vol Title 21: Food and Drugs PART 316—ORPHAN DRUGS. https://www.ecfr.gov/cgi-bin/retrieveECFR?gp=&SID=718f6fcbc20f2755bd1f5a980eb5eecd&mc=true&n=sp21.5.316.c&r=SUBPART&ty=HTML#se21.5.316_120.

6. Government of New Brunswick C. New Brunswick Drugs for Rare Diseases Plan. http://www2.gnb.ca/content/gnb/en/departments/health/services/services_renderer.201352.New_Brunswick_Drugs_for_Rare_Diseases_Plan.html. Published 08:44:39.0.

7. Alberta Government. Alberta rare diseases drug program : fact sheet. https://open.alberta.ca/publications/alberta-rare-diseases-drug-program-fact-sheet.

8. Orphan Drug Act. Vol 19.; 1983:2049-2066. https://www.gpo.gov/fdsys/pkg/STATUTE-96/pdf/STATUTE-96-Pg2049.pdf.

9. US Food and Drug Administration. Designating an Orphan Product: Drugs and Biological Products. https://www.fda.gov/forindustry/developingproductsforrarediseasesconditions/howtoapplyfororphanproductdesignation/default.htm. Updated September 14, 2017.

10. US Federal Drug Administration. FDA’s Orphan Drug Modernization Plan. June 29, 2017.


Cite This Article:

Coles V., Chan G., Palczewski K., Lewis K., Ho J. Pitfalls in Canada’s Approach to Orphan Drugs. Illustrated by C. Scavuzzo. Rare Disease Review. November 2017. DOI:10.13140/RG.2.2.26088.78089.

The Diabetes Mimic: a Primer on Addison’s Disease

The Diabetes Mimic: a Primer on Addison’s Disease

Drug Repurposing: The New R&D

Drug Repurposing: The New R&D