A day for action for the Rare Disease community

Today, February 28, marks Rare Disease Day. A day that provides the ALS Society of Canada (ALS Canada) a chance to bring awareness to the disease alongside other potentially lesser-known diseases to work together towards equity in health care, and access to diagnosis and therapies. Rare Disease Day helps to shed light on the lack of resources and challenges faced by people living with a rare disease. Health Canada’s draft definition of a rare disease is one that affects fewer than five in 10,000 persons in Canada. 

Amyotrophic lateral sclerosis (also known as ALS, Lou Gehrig’s disease, or motor neuron disease), a fatal neurodegenerative disease, results in progressive paralysis and, eventually, the loss of the ability to walk, talk, eat, swallow, and eventually breathe. Approximately 3,000 Canadians live with ALS, many of whom die within two to five years of diagnosis. To change this reality, ALS Canada funds research across the country and advocates for equitable, affordable, and timely access to proven therapies. Although ALS is technically a rare disease, it doesn’t feel that way for people who are affected including the person living with ALS, their loved ones, caregivers and friends and families – the impact is significant and can cause ripples for generations due to the devastation it inflicts.  

“Today marks a time to recognize the rare disease community, and at ALS Canada, it is a day to continue to raise awareness of the need to make crucial changes to our government’s health policies and investments,” says Tammy Moore, CEO of ALS Canada.   

Each year approximately 1,000 Canadians will learn that they have ALS, and a similar number will die. As ALS progresses so quickly, swift access to treatment is needed, yet the community continues to face significant challenges in how long it takes for provinces across Canada to reimburse ALS therapies. Long drug approval processes and reimbursement decisions – some taking more than three years – unnecessarily delay access in Canada.  

“There is no time to waste. For people living with ALS and their families, timely and equitable access to therapies is everything. We must continue to work together to advocate for changes to the current drug approval and reimbursement processes in Canada to respond to the urgent and unique needs of a person living with ALS,” continues Moore.    

It is vital that people living with ALS have their voices heard at a government level. Bringing forward the lived experiences of this community shows decision-makers the human impact of this devastating disease and why it is necessary to make changes to the drug access processes in Canada.   

“I realized, having gone through the Canadian ALS Learning Institute (CALI), just how important sharing my personal story can be to bring to light issues faced by people like me,” says Ray Freebury, who was diagnosed with ALS in 2018. “I used the training I received to approach my MPP. Like many of her colleagues, she had little knowledge of ALS and the problems of getting treatment approved and paid for,” Ray says. “I wanted her to know just how crucial access to existing therapies is. They can delay progression of the disease, improve the quality of life for people with ALS, and make life easier for personal carers who are often family members. Access to therapies also provides hope that more effective treatments are on the way.”  

Rare Disease Day is just one opportunity to bring these issues to the forefront however, our advocacy efforts don’t stop here. ALS Canada’s  Time is Now position paper, written in consultation with the broader Canadian ALS community, offers two concrete solutions to getting Health Canada-approved therapies to Canadians living with ALS in a timeframe that more accurately meets the urgent needs of the community.   

Alongside community members like Ray, ALS Canada continues to engage with officials in the federal and provincial governments to advocate for policy changes that will have a meaningful impact on people living with ALS today and in the future.    

If you’d like to get involved, download our Advocacy-in-a-Box Toolkit – together we can work toward change.