Roundtable Discussion: Redefining Clinical Success Metrics to Ensure Meaningful Functional Recovery in Peripheral Neuropathies Disease
- Evaluating score-based severity reduction, such as reducing a scale from 1 to 0.5, and questioning whether statistical improvement translates into tangible muscle function recovery like improved grip strength, brushing teeth, or walking stability
- Comparing existing neurological assessment tools (e.g., grip strength, MRC scores and other scales), analysing their variability and lack of a clear gold standard to determine which tools best capture meaningful neuromuscular improvement in Phase III trials
- Defining what “good enough” improvement means, acknowledging that partial improvement may not reflect meaningful daily function restoration to align trial endpoints with patient-relevant outcomes rather than abstract metrics