For decades Canada has stood as an outlier country standing without a defined national rare disease policy, despite Canadian patients, advocacy groups, clinicians, researchers and industry desiring a national strategy to connect the provision of drugs for rare diseases across thirteen Canadian provinces and territories. With a growing number of Canadians being affected by rare diseases, Canadians need improved understanding, expertise, and access throughout the country. Each rare disease affects such a small number of individuals, as such, accessing adequate diagnosis and care is incredibly challenging.
In 2019, with the most recent election of the Trudeau-led Liberal government, the subject of rare diseases received renewed national attention. The 2019 Federal Budget has proposed that the Canadian government will invest up to one billion dollars over two years, starting in 2022-2023, with up to five hundred million dollars invested in drugs for rare diseases per year ongoing. The government has indicated that this budget would include "the creation of a national strategy for high-cost drugs for rare diseases, to gather and evaluate evidence on high-cost drugs for rare diseases, improve the consistency of decision-making and access across the country, negotiate prices with drug manufactures, and ensure that effective treatments reach the patients who need them" 1. Unfortunately, a national rare disease policy has been subject to political football and manoeuvring with drafts of previous plans that were developed under the Conservative Stephen Harper government in 2012 yet "sat on the back burner since then…[with] Health Canada recently deleted all references from its website" 2.
Since the initial publication of the 2019 budget, Canadians have not yet been provided with any update on the development of a national strategy for rare diseases. Not only is it unclear what the national policy will look like, but it isn't clear how core issues will be addressed or even if a national policy will be a magic bullet to resolve access and availability challenges related to drugs for rare diseases.
Constructing effective Canadian policy will be critical to ensure patient access to safe and effective treatment and avoid some of the pitfalls currently being faced by other national policies such as the American Orphan Drug Act. That policy was created in 1983 to incentivize research and development in a space that offered little market interest due to small patient (read consumer) populations. Incentives ranging from tax credits of up to 50% of R&D costs, R&D grants, waived FDA fees, protocol assistance and prospective clinical trial tax incentives, are now be gamed with one "orphan" drug such as adalimumab being designated for use for numerous rare and also non-rare conditions, and thereby offsetting industries R&D cost and circumventing the spirit of the policy.
Furthermore, it isn't evident if there will be a definition of what counts as a rare disease -as the ratios of cases per 1000s varies dramatically across provinces- or what it may be…but that challenge is the topic of another blog.
On June 23rd, the Government of Canada hosted a research webinar designed to give the Patented Medicines Prices Review Board "insight into the spending on expensive drugs for rare diseases" 3. The cost of these products is undoubtedly an issue, but it certainly isn't the only one. Groups such as the Canadian Organization for Rare Diseases (CORD) have long been calling for a five-point action plan that would help address unnecessary delays in testing, prevent incorrect diagnosis, and avert missed opportunities to treat 4. The five-point action plan proposed by CORD calls for:
Improving early detection and prevention
Providing timely, equitable, and evidence-informed care
Enhancing community support
Providing sustainable access to promising therapies and
Promoting innovative research
To be sure, the dedication of one billion dollars to a Canadian national rare disease policy can be a positive development…if lessons can be learned from older policies from other national constituencies. However, in the meantime, as a Canadian national policy fumbles along, there is a continued urgent need for social innovation in the field of rare diseases. Hopefully a national policy will help address challenges of access and availability of safe and effective drugs for rare disease but it will not resolve them. As individuals with rare diseases continue to go without treatment, it continues to be essential for social innovation in this space to move to the forefront. The creation, adoption, development, and integration of new and renewed concepts, systems, and practices that put individuals with rare diseases first, in cooperation with drug developers, for not necessarily market motivations may be the best hope for Canadians and others worldwide affected by rare diseases to receive the medicines they need.
1. Government of Canada. (2019, March 19). Moving forward on implementing national pharmacare. Retrieved July 1, 2020 from Budget2019: https://www.budget.gc.ca/2019/docs/themes/pharmacare-assurance-medicaments-en.html
2. Forrest, M. (2017, October 17). Health Canada gives 'kiss of death' to planned policy for rare-disease drugs. National Post , 2020 (July): https://nationalpost.com/news/politics/health-canada-gives-kiss-of-death-to-planned-policy-for-rare-disease-drugs
3. Government of Canada. (2020, August 19). Patented Medicine Prices Review Board Draft Guidelines Consultation. Retrieved August 24, 2020 from Government of Canada: https://www.canada.ca/en/patented-medicine-prices-review/services/consultations/draft-guidelines.html
4.Canadian Organization for Rare Disorders. (2015, September 14). Canada's rare disease strategy. Retrieved August 20, 2020 from Canadian Organization for Rare Disorders: https://www.raredisorders.ca/canadas-rare-disease-strategy/
Written by: Conor Douglas (PhD) and Shir Grunebaum (MA)
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