Adults living with SMA are a heterogeneous population, with a wide range of functional abilities, severity of symptoms and complications. Currently, access criteria and scientific research place a disproportionate weight on gross motor function assessments, whilst gaps exist in other meaningful aspects of fine motor function, respiratory function, bulbar function, and fatigue. Current assessments of treatment response and observations of improvement are not well measured. Important questions about when a treatment is effective and how to assess treatment efficacy remain unanswered, which can negatively impact patient access to and quality of care.

A modified Delphi study will be conducted, consisting of two online survey rounds and a final consensus meeting. A steering committee of six medical experts and three patient advocates will provide scientific oversight. Panelists will be double blinded to the study sponsor, study investigators and other participants for the entirety of the study. Eligible participants will include 25 neurologists, 25 allied health professionals and six patient advocacy group representatives from the USA, UK, France, Germany, Italy and Spain.

Across both survey rounds, panelists will be asked to provide detailed insights on the key outcomes that should be assessed to determine what constitutes a clinically meaningful treatment outcome in adults living with SMA. Panelists will then participate in a consensus meeting, to discuss and consolidate their responses and reach their final conclusions. Throughout the study, definitions of a meaningful treatment response will also be explored.

The Delphi study design will be presented.

Outputs from the study will be used to educate stakeholders on the true value of defining and utilizing meaningful treatment outcomes personalized to adults living with SMA, helping to support improvements in and access to adult SMA care.