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October 3, 2012

The Canadian Association of PNH Patients applauds the rare diseases framework announced today by Health Canada as an important first step in addressing the critical need for improved access to information and new treatments.  We are appreciative of this greatly needed investment in our community, and look forward to working with the federal government to continue expanding support through this framework for patients living with rare diseases across the country.

We know that there is work still to be done with respect to access to funding for treatment, as patients battling rare diseases continue to fall through the cracks.  Federally, the Common Drug Review simply cannot be effectively applied in the drugs for rare diseases paradigm.  In some provinces, patients are still not able to access catastrophic drug funding, and in others special policies are needed to ensure funding for drugs for rare diseases is made available to patients in a timely manner.  We are hopeful that through this framework, Health Canada will continue to work with stakeholders and the provinces to address these issues around access to funding for treatments that impact life and death.

Our very small group of Canadians living with PNH (paroxysmal nocturnal hemoglobinuria) and their families had to advocate at the most difficult time in our lives for improved access to treatment funding from the provinces.  Despite the establishment by the provinces of the pan-Canadian purchasing alliance over a year ago, there are still PNH patients who are not receiving funding for a life-saving treatment. When patients with a rare disease are extremely ill, it is inhumane that their only course of action is to plead louder to government than those patients living with more common diseases.

We are encouraged by this announcement today and join the rare diseases community in our hope that the new rare diseases framework will be enacted immediately to support patients living with rare conditions, and expanded to address the issue of access to funding for treatment.  There is no more time to waste when Canadians’ lives are at stake.