A US Food and Drug Administration statistician used placebo arm data from a Sarepta Therapeutics, Inc. clinical trial of Elevidys (delandistrogene moxeparvovec-rokl) to highlight the challenges of trying to use external controls to demonstrate efficacy.
Sarepta’s External Controls Analysis Weakened By Elevidys Placebo Data Comparison
When placebo data from the only randomized trial of the Duchenne muscular dystrophy gene therapy were compared with Sarepta’s proffered external control data, the placebo subjects numerically outperformed the external controls, ‘calling into question the comparability of the external control group,’ an FDA statistical reviewer said.
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