Causal inference and digital twins: a roadmap for the future of clinical trials.

Ruhrberg Estévez, Silas; Peck, Richard; McKinney, Eoin; Weatherall, Jim; Bailey, Stuart; Rochon, Justine; Anagnostopoulos, Chris; Marquet, Pierre et al. · NPJ Digit Med · 2026

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Abstract

Clinical trials generate essential evidence on treatment safety and efficacy, but slow timelines, high costs, and limited inclusivity constrain efficiency, generalisability, and clinical impact. Causal inference and digital twins offer complementary tools to define estimands, characterise treatment-effect heterogeneity, assess transportability, and simulate patient trajectories under alternative interventions. Integrated responsibly into trial design, recruitment, monitoring, and post-trial translation, they could support faster, fairer, and more informative clinical trials.