Treatment effect estimation by comparing observed and predicted outcomes: theory and practical illustration in radiotherapy

TL;DR

This paper discusses a method for estimating treatment effects in radiotherapy by comparing observed outcomes with model-predicted outcomes, emphasizing conditions for validity and illustrating with a case study.

Contribution

It clarifies the conditions necessary for valid treatment effect estimation using model-based evaluation in radiotherapy, grounded in the potential outcomes framework.

Findings

Conditions for valid treatment effect estimation are identified.

The approach is illustrated through a practical case study.

Theoretical framework supports practical application.

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.