Changes are needed to Canada’s national rare disease strategy to improve timely access to innovative medicines.
Over the past two years, Canada has made some progress in addressing the needs of the 1 in 12 Canadians living with a rare disease. The announcement of the National Strategy for Drugs for Rare Diseases in March 2023, coupled with a $1.5-billion investment, marked a significant first step, although it has taken nearly two years for British Columbia, Alberta, Saskatchewan, Ontario, Newfoundland and Labrador, and New Brunswick to sign bilateral agreements with the federal government. Encouragingly, organizations funded by the federal government, such as Canada’s Drug Agency, have initiated projects to address data gaps, update screening protocols, and explore uses of real-world evidence.
Ongoing access challenges
While these developments are promising, they have yet to translate into improvements in access to treatments for patients with rare diseases. The time from drug launch to public listing still takes too long, averaging two years for rare disease drugs to become available on a public plan following Health Canada approval. Additionally, the number of treatments launched in Canada continues to lag behind peer countries.
Transparency has been an issue as well. Fifty per cent of the funding under the agreements is for “common list drugs,” which were chosen to help provinces and the federal government develop a process for using real-world evidence to evaluate rare disease drugs. Unfortunately, the list of drugs was created without input from those most affected and knowledgeable: patients, caregivers, healthcare providers, and researchers. There is no transparency in the drug selection process either, and not even drug manufacturers are informed of whether a drug is on the list until the pan-Canadian Pharmaceutical Alliance (pCPA) negotiations are complete.
Encouraging collaboration and innovation
Money alone cannot address the challenges faced by patients and their families. A more comprehensive, collaborative, and expedient approach is needed to reform Canada’s regulatory and reimbursement processes. The federal government should create an accelerated pathway for medicines that meet a prevalence definition of a rare disease (e.g. no more than 1 in 2,000 people). This approach, already implemented in several international jurisdictions, would establish a more predictable, efficient process.
Under such a pathway, medicines would undergo a streamlined Health Canada regulatory review. Simultaneously, value assessments would be conducted by a committee comprised of rare disease experts, including those with lived experience. This committee would directly engage with patients, caregivers, healthcare providers, researchers, and manufacturers, emphasizing real-world impact over traditional pharmaco-economics, which often fail to account for the unique nature of rare disease treatments.
Addressing gaps and challenges
The federal government should work with provinces to develop a dedicated negotiation stream within the pCPA specifically for rare disease treatments. This should include agreements linked to evidence developments and innovative reimbursement models. Provinces could further accelerate access by implementing interim access mechanisms akin to models in Germany or France while pCPA negotiations are underway. Innovative Medicines Canada members have proposed a Canadian version of such a model to provincial governments.
These additional elements would help ensure that patients and families can access the care they need. They would also position Canada as a more attractive place for launching innovative treatments, benefiting patients and strengthening the country’s healthcare and life sciences ecosystems.
Canada’s innovative pharmaceutical industry is ready to collaborate with governments, patients, providers, and researchers to improve access to medicines for rare diseases. By working together, we can achieve meaningful change and deliver tangible benefits for patients with rare diseases and their loved ones. The time to act is now.
To learn more, visit innovativemedicines.ca.
