Cure SMA Canada is recognized for our contribution to the Canadian government’s understanding of the impact of SMA and played a major role in the associated changes to access to treatment brought by this contribution.
Thank you to all of those who participated and supported Cure SMA Canada initiatives! Together we truly do make a difference for us all!
January 31, 2020,
Susi Vander Wyk, Executive Director
Cure SMA Canada
Dear Susi,
Thank you for providing joint patient input to us in August 2018, on Spinraza for 5q spinal
muscular atrophy across all types.
Your joint input helped CADTH develop the review protocol by identifying important outcomes, including muscle strength or mobility, ability to eat independently, ability to breathe independently, and quality of life.
In our clinical report, your input was used to describe the different types of spinal muscular atrophy and the hopelessness and despair parents feel, watching their children decline. It was also used to describe the impact of spinal muscular atrophy on children, families, and caregivers in the economic report.
A member of the Canadian Drug Expert Committee presented your input to provide patients’ perspectives and to underscore treatment gaps. Committee members reflected on how the evidence under review addressed the issues identified by your group.
CADTH reviewers and expert committee members found your input useful, particularly with regards to:
• Involving youth, adults, and parents of children living with spinal muscular atrophy (types 1,2, 3, and 4). Committee members heard direct quotations from these individuals.
• Highlighting the importance of maintaining mobility, including upper body mobility, and the value of not losing further mobility and independence.
CADTH reviewers and the Canadian Drug Expert Committee members thank you for your effort in providing patients’ perspectives. Patient input is an integral part of our process and we appreciate your contribution.
Sincerely,
Sarah Berglas
Manager, Patient Engagement, CADTH
