The Government of Canada has made an announcement which will benefit the 1 in 12 people (many of whom are children) living with a rare disease in the country.
The Government of Canada will support the National Strategy for Drugs for Rare Diseases, which is the first of its kind. The purpose of this strategy is to “help increase access to, and affordability of, effective drugs for rare diseases to improve the health of patients across Canada, including children”. The investment will include up to $1.5 billion over three years. The Non-Insured Health Benefits Program (NIHB) at Indigenous Services Canada will also receive $33 million in new investments.
Despite there being thousands of such rare diseases affecting many individuals and their support systems, only a small percentage of conditions have treatments available. Of those who may benefit from the effectiveness of these treatments in their quality of living, many patients are unfortunately still restricted from utilizing the medications due to significant financial barriers and limited access.
This strategy is promising, as it shows a national commitment towards providing Canadians with rare diseases with the drugs they need in both a timely manner and at a reasonable price. In addition, the early diagnosis and screening for rare diseases will improve.
The announcement can be found here in full with associated links (for example: Regulatory innovation for health products). It also includes the following quick facts:
- Innovative treatments for rare disease can cost anywhere from $100,000 to more than $2 million per year. Budget 2019 provided funding up to $1 billion over two years, with up to $500 million per year ongoing, to help Canadians with rare diseases access the drugs they need.
- In July 2021, Health Canada released a What We Heard Report from national engagement on the National Strategy. The engagement garnered diverse perspectives from over 650 individuals and organizations, including patients with lived experience, family members, and caregivers.
- In December 2022, Health Canada pre-published proposed amendments to the Food and Drug Regulations that would introduce regulatory flexibilities such as the option of a rolling review, which may facilitate earlier market access for certain drugs, including drugs for rare diseases. The proposal would also allow for terms and conditions for all drugs, enabling Health Canada to include obligations on the manufacturer to optimize the benefits and manage any risks and uncertainties associated with the drug. This would be particularly important in the cases of drugs for rare diseases where patient populations are small.
- Additionally, Health Canada’s Pediatric Drug Action Plan aims to improve access to safe and effective medicine for children in Canada, including drugs for rare diseases, which tend to be more prevalent in the pediatric population.