☆ 4.8 Letter

First regulatory qualification of a digital primary endpoint to measure treatment efficacy in DMD

NATURE MEDICINE (2023)

Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls

Francesco Muntoni et al.

Summary: Using external controls based on real-world or natural history data (RWD/NHD) for drug evaluations in Duchenne muscular dystrophy (DMD) is appealing given the challenges of enrolling placebo-controlled trials, especially for multi-year trials. This study compared the changes in North Star Ambulatory Assessment (NSAA) total score between trial placebo arms and RWD/NHD sources, and found that the differences were small and not statistically significant after adjustment for baseline prognostic factors. The results support the suitability of RWD/NHD external controls for drug evaluations in ambulatory DMD.

NEUROMUSCULAR DISORDERS (2022)

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Article Clinical Neurology