Francesco Muntoni, James Signorovitch, Gautam Sajeev, Nathalie Goemans, Brenda Wong, Cuixia Tian, Eugenio Mercuri, Nicolae Done, Hallee Wong, Jackson Moss, Zhiwen Yao, Susan J Ward, Adnan Manzur, Laurent Servais, Erik H Niks, Volker Straub, Imelda Jm de Groot, Craig McDonald
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. Comparisons to external controls, however, face risks of bias due to differences in outcomes between trial and RWD/NHD settings. To assess this bias empirically, we conducted a multi-institution study comparing mean 48-week changes in North Star Ambulatory Assessment (NSAA) total score between trial placebo arms and RWD/NHD sources, with and without adjustment for baseline prognostic factors...
April 2022: Neuromuscular Disorders: NMD