Defeat Duchenne Canada would like to share that the Institut national d’excellence en santé et en services sociaux (INESSS) has issued a final negative recommendation for vamorolone (AGAMREE).
INESSS is responsible for evaluating new treatments and making funding recommendations to the Québec government. In its final recommendation, INESSS does not support reimbursement of vamorolone for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older.
It appears that public coverage is not recommended due to concerns with the cost of the drug; however, we are pleased to see INESSS acknowledge the clinical value of vamorolone and its significance for people affected by DMD. An important next step will be working with Québec’s public drug plan to encourage their participation in pricing negotiations to ensure access to treatment.
“While INESSS recognized the clinical value, we are disappointed that INESSS issued a negative final recommendation based on the economic value. This is not the outcome the DMD community was hoping for. Defeat Duchenne Canada remains committed to working with all partners to explore next steps and to continue advocating for fair and timely access to treatments for individuals living with DMD, both in Québec and across Canada.”
– Nicola Worsfold, Executive Director of Defeat Duchenne Canada and Duchenne Mom
“The INESSS final recommendation does not reflect the urgent needs of Québec’s DMD community. For families living with DMD, timely access to treatment directly affects quality of life and time we cannot afford to lose. La Force DMD, alongside Defeat Duchenne Canada, remains committed to working with Québec partners to advance equitable and timely access to therapies for those affected in our province.”
– Marie-Catherine Du Berger, Executive Director of La Force DMD and Duchenne Mom
Read INESSS’s final recommendation below:
Update on Canada’s Drug Agency (CDA)
Defeat Duchenne Canada submitted feedback on CDA’s draft recommendation for vamorolone, highlighting areas that could require revision to ensure equitable access for all people living with DMD who can benefit from this treatment. While the recommendation was positive overall, the manufacturer made a request, which CDA accepted, to reconsider certain aspects of the submitted feedback. The CDA expert committee will be meeting on September 23 on the reconsideration.
We had expected to see the drug move through the reconsideration in a timelier manner; however, Defeat Duchenne Canada appreciates CDA’s willingness to undertake this process. We hope that the outcomes of the reconsideration will ultimately lead to broader access for the DMD community, who have already been waiting far too long to access the first approved therapy for DMD in Canada.
We will continue to update the community as we learn more.
Your Voice Matters, Now More Than Ever!
We encourage families and individuals living with DMD to:
- Check out our webinar resources to understand how you can continue to advocate as we move towards reimbursement across Canada.
- Learn how to add your voice through our advocacy toolkit.
This is advocacy in action. Together, we can continue moving forward toward equitable and affordable access for Canadians living with Duchenne.