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arXiv · 2511.21266

Treatment effect estimation by comparing observed and predicted outcomes: conditions for valid inference and practical illustration

Lotta M. Meijerink·Artuur M. Leeuwenberg·Jungyeon Choi·Bas B. L. Penning de Vries·Johannes A. Langendijk·Judith G. M. van Loon·Remi A. Nout·Karel G. M. Moons·Ewoud Schuit
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Abstract

Prediction models developed before the introduction of a new treatment may be used to estimate treatment effects of newly introduced treatments. One approach, known as model-based clinical evaluation in radiotherapy, does this by comparing observed outcomes under a new treatment with predicted outcomes had these patients received the standard treatment. This article clarifies the relevant conditions needed for valid average treatment effect estimation using this approach, using the potential outcomes framework and a practical case study.

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Lotta M. Meijerink, Artuur M. Leeuwenberg, Jungyeon Choi, Bas B. L. Penning de Vries, Johannes A. Langendijk, Judith G. M. van Loon, Remi A. Nout, Karel G. M. Moons, Ewoud Schuit. 2025-11-26. Treatment effect estimation by comparing observed and predicted outcomes: conditions for valid inference and practical illustration. https://doi.org/10.1186/s41512-026-00233-y

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