A recent article in the magazine Managed Care discussed the management of drug costs for rare genetic diseases. According to the authors, most private health insurers pay for ultra-rare genetic disorders (such as Fabry or Gaucher’s Disease) in part because very little of total health or drug plan spending goes to treat rare conditions. In fact, most (75 percent) of the spending on specialty drug products such as biologic and injectable agents goes to treating just five categories: oncology, HIV/AIDS, renal disease, transplant medications, and hemophilia.” Spending on ultra rare diseases does not drive pharmacy costs: “On average the number of patients with rare diseases in any one plan is simply too low …to increase the typical payment per member per month by more than a few pennies per month.”

The Canada Health Act clearly states “that continued access to quality health care without financial or other barriers will be critical to maintaining and improving the health and well-being of Canadians.” Arguably, the primary public policy imperative that led to the Canada Health Act is the principle that no one should suffer financial devastation or be denied access to available treatments for a life-threatening illness because of cost. There are a total of 200 patients in Canada with Fabry Disease. Ensuring that they and others who suffer from rare disorders have access to available treatments is a fundamental test of whether provincial health plans are driven by the principles of the Canada Health Act or whether they are driven by containing costs by pre-selecting illnesses and conditions for which treatments will be covered.