Rare diseases, by definition, affect fewer than 1 in 2000 persons.

Key points:
  1. Canada is the ONLY country in the developed world WITHOUT a proper Federal Healthcare policy for the rare disease community.
  2. 1 in 12 Canadians (approximately 3 million people) suffer from one of approximately 7 000 rare diseases. These patients have poor access to diagnosis, care and treatment.
  3. More Canadians suffer from rare diseases than HIV/AIDS, Heart Disease, and Cancer combined.
  4. Canada does not have a definition for a rare disease. Rare diseases, by definition, affect fewer than 1 in 2,000 persons.
  5. Most rare diseases are life-threatening or debilitating; about 80% are genetic, and 75% affect infants and children.
  6. Without a Canadian Orphan Drug Policy, pharmaceutical companies have no incentive to research & develop therapies because individual patient populations are so small.
  7. When therapies DO exist, they can face delayed approval by the Federal government. Also, such therapies are so prohibitively expensive that no patient or family could possibly afford them.
  8. It means that even when rare therapies ARE approved by the Healthcare system; it is left to individual provinces to decide whether they will foot the bill – leaving patients prisoners in their own provinces.
  9. No rare disease therapies have been funded in Canada without patient advocacy. A heavy burden on the patient (and their families). Patients battling for funding while living with a debilitating, and often deadly, disease.
  10. Funding rare disease therapies will provide patients better quality of life and in the long term help decrease costs on the Canadian medical system.
  11. In 1984, the United States passed the Orphan Drug Act, resulting in more than 350 effective therapies for unmet needs.  In 2009, the European Council, based on the principle that those with rare diseases have the same right to care as those with more common ones, recommended all member states establish national plans for rare disorders, with a target of 2013.
  12. What is needed in a National Plan:
    • Canada to adopt a rare disease definition. Rare diseases, by definition, affect fewer than 1 in 2,000 persons.
    • A national commitment to fund current rare disease therapies & treatments.
    • A revamped process for the approval of these rare disease therapies & treatments
    • Support research for other individual rare disease therapies & treatment
What does not having a proper Canadian Rare Disease Plan mean for 1 in 12 Canadians?
  • Pharmaceutical companies have no incentive to develop therapies because individual patient populations are so small.
  • It means that even when therapies DO exist, they can face delayed approval by the Federal government. Also, such therapies are so prohibitively expensive that no patient or family could possibly afford them.
  • It means that even when rare therapies ARE approved by the Healthcare system; it is left to individual provinces to decide whether they will foot the bill – leaving patients prisoners in their own provinces.
What is needed is a National Healthcare Plan that encourages:
  • New Drug Discovery for individual rare diseases
  • A revamped process for the approval of these therapies
  • A national commitment to fund their costs for individual families

It is vital we continue to raise awareness of this important issue in Canada (and worldwide) with both policymakers and the general population. We further ask that our government continue to explore ways to support this not so rare segment of the Canadian population as it seeks equality in the eyes of our healthcare system.


book

Learn more at the Canadian Organization for Rare Disorders.

Simon’s book, ‘Rare Diseases in the Age of Health 2.0’ is available for purchase.
All book proceeds go to support the iBellieve Foundation.