Estimating individualized treatment effects from randomized controlled trials: a simulation study to compare risk-based approaches.

ABSTRACT:

Background

Baseline outcome risk can be an important determinant of absolute treatment benefit and has been used in guidelines for "personalizing" medical decisions. We compared easily applicable risk-based methods for optimal prediction of individualized treatment effects.

Methods

We simulated RCT data using diverse assumptions for the average treatment effect, a baseline prognostic index of risk, the shape of its interaction with treatment (none, linear, quadratic or non-monotonic), and the magnitude of treatment-related harms (none or constant independent of the prognostic index). We predicted absolute benefit using: models with a constant relative treatment effect; stratification in quarters of the prognostic index; models including a linear interaction of treatment with

SUBMITTER: Rekkas A 

PROVIDER: S-EPMC10045909 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature