Evaluation of clinical utility in emulated clinical trials

TL;DR

This paper discusses methods for evaluating the clinical utility of dynamic treatment regimes using observational data, emphasizing detailed procedures for emulated clinical trials and comparing treatment strategies in rheumatoid arthritis.

Contribution

It introduces detailed estimators and practical guidelines for conducting emulated clinical trials to assess treatment strategies' utility.

Findings

Proposed multiple estimators for clinical utility evaluation.

Applied methods to rheumatoid arthritis treatment data.

Highlighted importance of comparing to current medical guidelines.

Abstract

Dynamic treatment regimes have been proposed to personalize treatment decisions by utilizing historical patient data, but they may not always improve on the current standard of care. It is thus meaningful to integrate the standard of care into the evaluation of treatment strategies, and previous works have suggested doing so through the concept of clinical utility. Here we will focus on the comparative component of clinical utility as the average outcome had the full population received treatment based on the proposed dynamic treatment regime in comparison to the full population receiving the ``standard" treatment assignment mechanism, such as a physician's choice. Clinical trials to evaluate clinical utility are rarely conducted, and thus, previous works have proposed an emulated clinical trial framework using observational data. However, only one simple estimator was previously suggested, and the practical details of how one would conduct this emulated trial were not detailed. Here, we illuminate these details and propose several estimators of clinical utility based on estimators proposed in the dynamic treatment regime literature. We illustrate the considerations and the estimators in a real data example investigating treatment rules for rheumatoid arthritis, where we highlight that in addition to the standard of care, the current medical guidelines should also be compared to any estimated ``optimal'' decision rule.