Direct utility of natural history data in analysis of clinical trials: Propensity match-based analysis of Omaveloxolone in Friedreich ataxia using the FA-COMS dataset

David Lynch, Angie Goldsberry, Christian Rummey, Jennifer Farmer, Sylvia Boesch, martin delatycki, Paola Giunti, j chad Hoyle, Caterina Mariotti, Katherine Mathews, Susan Perlman, SH Subramony, George Wilmot, Theresa Zesiwiecz, Lisa Weissfeld, Colin Meyer medRxiv 2022.08.12.22278684; doi: 10.1101/2022.08.12.22278684 

These results suggest a clinically meaningful slowing of FRDA progression with omaveloxolone, and consequently details how propensity-matched analysis contributes to the understanding of the effects of therapeutic agents. This demonstrates the direct value of natural history studies in the evaluation of clinical trials.