Clinical outcome measures are tools used to assess the impact of healthcare interventions on patients’ health and well-being. These measures help healthcare professionals to quantify changes in patients’ symptoms, function, and quality of life over time. It provides metrics to identify healthcare providers who provide good care for a specific rare disease and provide others with a set of goals to strive for.

Our Action Plan includes the development of outcome measures through the work package on outcome measures and clinical recommendations/expert consensus statement.

Outcome Measures