This article is the final part of an 8-part series from the authors. The series, Waiting for new drugs for rare disorders in Canada, is an in-depth look at the disadvantages faced by Canadians with rare disorders in accessing needed, innovative drugs.
By Nigel Rawson and John Adams, August 30, 2023
In the previous two articles in our series, we discussed proposals for national pharmacare and a Canadian Drug Agency. Neither appear to include plans for accelerating access to drugs for rare disorders for Canadians who need them. Huge unmet needs exist for Canadians afflicted with any of the 11,000 or so rare disorders. Only 500 of those 11,000 rare disorders (less than five percent) have any treatments, other than symptom relief and palliative care.
Unlike almost all other developed countries, Canada doesn’t have a rare disorder strategy that includes incentivizing research, development and commercialization of medicines in this country. A strategy must be more comprehensive than a focus just on medicines. It should comprise early detection and prevention and the provision of timely, equitable, evidence-based and coordinated care, in addition to sustainable access to potentially beneficial treatments.
Early detection should include fair and comprehensive screening of newborns for rare disorders. Canada currently has no national standards or guidelines or federal leadership for newborn screening, resulting in screening being inadequate and inconsistent across the country.
Diagnosing rare disorders can take months, even years, with patients being referred from specialist to specialist, often with misdiagnoses, in the hope that one will identify their condition and be able to help them. During this diagnostic odyssey, patients frequently receive treatments that may or may not be beneficial but can waste the health care system a lot of time and money. Procedures must be implemented to shorten the diagnostic process in Canada.
Many patients with rare disorders require services and treatments that are commonly not recognized as essential in Canada. Because such specialized services are lacking in much of the country, repeated travel from home to a distant centre of highly specialized care, which may be out of province or even out of country, is often necessary. This places a heavy burden on individual patients and their families.
A rare disorder strategy must include affordable access to drugs for these disorders uninhibited by burdensome and restrictive coverage rules. The creation of new genetic and cell therapies following increased understanding of the human genome has led to treatments for diseases that 20 years ago were untreatable. However, their development is a time-consuming and costly enterprise with the potential for limited sales if the medicine is one of the few that makes it through all the testing to being accessible by patients. Consequently, drugs are priced to provide returns justifying the pre-marketing investment and inevitable failures, as well as produce a profit once successfully launched.
All these components – early detection and diagnosing, coordinated and evidence-based care, and sustainable access to potentially beneficial treatments – were elaborated in the Canadian Rare Disease Strategy managed and launched by Canadian Organization for Rare Disorders in 2015 and revived in 2022. The federal government should have used this strategy as a starting point to improve the lives of Canadians with rare disorders.
Instead, in their 2019 federal budget, the Liberals said they would introduce their own strategy and, in their 2021 election platform promised $500 million per year starting in 2022-23 to help fund drugs for rare disorders. A comprehensive strategy for rare disorders is significantly different from a plan focused only on limiting the cost of these medicines. Nevertheless, in March 2023, the federal government re-committed $1.5 billion over three years to “increase access to, and affordability of, effective drugs for rare diseases to improve the health of patients across Canada,” with over 93 percent earmarked for bilateral agreements to be negotiated between Health Canada and provinces and territories.
People living with rare disorders are like other Canadians – they want affordable access to medicines for their unmet health needs. No one with a rare disorder should go broke paying for their treatment and no one should be left behind. But Canadians with rare disorders don’t want access denied by the federal government’s plans to force down drug prices to levels that are unsustainable for developers, who could easily decide the Canadian market isn’t worth entering.
Canadians living with rare disorders need many things: a comprehensive and actionable policy that includes incentives (such as time-limited market exclusivity provisions and support for data protection of new uses for old medicines) and funding to encourage developers to launch drugs in Canada; a mandatory requirement that all government drug plans list all rare disorder drugs with a successful price negotiation within a short period of time; and less restrictive and burdensome criteria that patients must satisfy before being able to obtain coverage. Without these actions, Canadians with rare disorders will not have timely access to the many innovative treatments on the research horizon that can reduce suffering, improve and even save lives.
Nigel Rawson is a Senior Fellow with the Macdonald-Laurier Institute and an Affiliate Scholar with the Canadian Health Policy Institute. John Adams is cofounder and CEO of Canadian PKU and Allied Disorders Inc., a Senior Fellow with the Macdonald-Laurier Institute and volunteer board chair of Best Medicines Coalition.
The views expressed are the authors’ own and do not necessarily represent those of organizations with which they collaborate.