SUMMARY:

Up to ten per cent of the Canadian population has a disease classified as ‘rare’, also commonly referred to as an 'orphan' disease. The resulting social costs and burden of care implications make it imperative that a Canadian Orphan Drug Policy be developed immediately to ensure timely, affordable access to necessary therapies.

POSITION STATEMENTS:

Canadians with rare or ‘orphan’ disorders that are progressively debilitating or life threatening need a made-in-Canada Orphan Drug Policy that will ensure they have timely, affordable access to the best therapies when needed.

Competitive incentives should be provided for Canadian-based companies engaging in research and development to bring new therapies to market. In turn, this incentive should be tied to a commitment by the companies to drug pricing relief to reflect these incentives.

The approach proposed in the section entitled “Expensive Drugs for Rare Diseases” as part of the National Pharmaceuticals Strategy (NPS) is worthwhile and warrants further development. The NPS progress report stresses the development and implementation of “consistent national processes and standards to ensure that Canadians with rare, severe and progressive life-threatening diseases have access to appropriate and affordable treatments.” These processes or standards should take the form of a Canadian Orphan Drug Policy, and be named as such.

Canada, as one of the last industrialized countries to institute an Orphan Drug Policy, should learn from other jurisdictions and create a policy based on best practices. The policy should reflect the entire pharmaceutical life cycle, including research, development and regulatory approval, affordable access and funding for drug coverage. Importantly, while based on international best practices, the policy must provide a made-in-Canada solution, addressing the roles of federal, provincial, and territorial governments and including other uniquely Canadian issues.

To read the full Position Statement on Orphan Drugs, click here (PDF file).