Can Machine Learning from Real-World Data Support Drug Treatment Decisions? A Prediction Modeling Case for Direct Oral Anticoagulants.

If prescribers are undecided about the potential benefits of different treatment options, ITEs can support decision making, especially if evidence is inconclusive, risk-benefit profiles of therapeutic alternatives differ significantly, and the patients' complexity deviates from "typical" study populations. In the exemplary case for DOACs and potentially in other situations, the significant impact could also become practically relevant if recommendations were available in an automated way as part of decision making.HighlightsIt was possible to calculate individual treatment effects (ITEs) from routine claims data for rivaroxaban and apixaban, and the characteristics between the groups with recommendation for one or the other option differed significantly.ITEs resulted in recommendations that were significantly superior to usual (observed) treatment allocations in terms of absolute risk reduction, both separately for stroke and in the composite endpoint of stroke and major bleeding.When similar patients from routine data were selected (precision cohorts) for patients with a strong recommendation for one option or the other, those similar patients under the respective recommendation showed a significantly better prognosis compared with the alternative option.Many steps may still be needed on the way to clinical practice, but the principle of decision support developed from routine data may point the way toward future decision-making processes.