National Gaucher Foundation of Canada | The power of individual stories to spark widespread advocacy
Christine White, President of the National Gaucher Foundation of Canada, discusses how publishing personal Gaucher stories acted as a catalyst for widespread media campaigns that gained political attention resulting in access to a new life-changing therapy for the Gaucher community.
Q. Can you tell us about your work with the National Gaucher Foundation for Canada and how your organisation supports your community?
The National Gaucher Foundation of Canada was founded by—and continues to comprise—individuals living with Gaucher and family members of those individuals. We support our community through education, advocacy, peer support and much more. We provide support to those affected by Gaucher disease every step of the way, up to and including helping to facilitate a diagnosis.
Q. What do you feel are your community’s biggest needs/challenges and how does your organisation work towards trying to resolve and raise the agenda for these?
Currently, our biggest challenge is access to new medications; for example, we do not have a first-line oral therapy listed on our provincial formulary—drugs for Gaucher in Canada are paid for by the government, and only those Canadians who have private insurance have access to therapies that are not listed with provincial plans.
We will have the opportunity very shortly to submit evidence to the government that this is an impactful and necessary therapy for patients; we have been successful in the past in gaining access and are hopeful that we can demonstrate a strong need for oral therapy.
This very unfair policy often results in patients being “prisoners in their own province”.
Q. I read on your website how instrumental you were in making the treatment Ceredase available in Canada, how challenging is the topic of treatment equity, and what advice would you give to patient organisations who face a similar challenge?
Treatment equity is still a big challenge here in Canada, quite often where you reside will determine which therapy you receive. This very unfair policy often results in patients being “prisoners in their own province”. This means that even if a patient has access in their province, moving elsewhere in the country could result in a change in drug therapy.
As we work very closely with the Canadian Organisation for Rare Disease (CORD), we recently had the opportunity to make a submission to Health Canada regarding a federal rare disease strategy. I would advise patient organisations that have similar challenges to reach out to CORD and other patient organisations for support and advice.
I find the rare disease community is very collegial and greatly supportive of one another here in Canada.
Q. With some treatments already in existence for Gaucher disease, why is there a need to keep researching new treatments?
Currently, the available therapies do not address the needs of all individuals living with Gaucher. At present, there is no cure for type 2 Gaucher disease, and sadly, many infants and very young children die. Also, there is not an effective treatment available for type 3 Gaucher disease as current therapies do not cross the blood-brain barrier. This leaves little hope for individuals afflicted with the more severe forms of Gaucher.
Q. What can people with Gaucher disease do to get involved with advocacy and why is it important that they do so?
One of the key strategies in advocacy is awareness—it is important for individuals living with Gaucher to tell their stories. For example, we gained access to a life-changing therapy once we began an awareness campaign through the media. Before our media exposure, every request we made to the government was denied, but once people heard the stories we promoted, they began to pressure the government to change their position which resulted in a successful outcome.
From a personal perspective, I feel advocacy provides patients with a sense of empowerment: as a young mother of two seriously ill children, advocating on their behalf made me feel less helpless.
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