The Neuromuscular Disease Network for Canada

On September 9th and 10th in Ottawa, Ontario, 14 adult SMA experts including, clinicians, allied health care providers, patient organization representatives, and the Canadian Neuromuscular Disease Registry (CNDR) representatives gathered to reach an updated national consensus on outcome measures for adults with SMA. With increasing experience following adults on novel disease-modifying therapy alongside advances in standardized outcome measures, it was essential to revisit the prior Canadian adult SMA toolkit (2020), with a renewed emphasis on meaningful outcomes for patients. The consensus work will help to support discussions within individual provinces to understand response to therapy, advocate for appropriate access to high-cost therapeutics, and enable national comparisons. 

During this multi-day meeting, the group considered a broad spectrum of outcome measures that may be applicable to adult SMA to determine a core set of recommended, suggested, and exploratory measures, based on the individual’s current functional status. To support measure selection, patient preferences and perspectives, practitioner experiences, feasibility (including time and equipment needed), and provincial payer requirements were discussed.  

The meeting concluded with a vote to determine which outcome measures should be completed across Canada for adults with SMA, and consensus was reached. Based on the new consensus results, the CNDR will update its SMA dataset accordingly.  Official results from this national consensus-building meeting will be released soon.