President & CEO, Canadian Organization for Rare Disorders
Following years of advocacy led by the Canadian Organization for Rare Disorders, the Canadian rare diseases community finally has something to cheer about. In December 2018, the provincial and territorial governments announced a “supplemental process” to address barriers to access of complex, specialty, and rare disease drugs.
In March 2019, the federal government committed $1 billion for a Rare Disease Drug Program and Strategy starting in 2022. And in June 2019, the National Pharmacare report proposed a distinct pathway for “expensive drugs for rare diseases,” and a national expert panel to work with patients and their clinicians to determine which rare disease drugs should be funded for which patients.
Therapies that we hardly dared dream about are here today, with dozens and dozens more just around the corner. The biggest impact is seen in treatments for rare blood disorders and rare cancers that replace missing or defective proteins, counteract or stop the reproduction of faulty genes, and can stop disease progression or even prevent symptoms from occurring.
With so many innovative therapies, some say we cannot afford to make them all available to every patient who could possibly benefit from them. Or can we? “Rare access to all” is the explicit goal of the Canadian Organization for Rare Disorders.