Going beyond randomised controlled trials to assess treatment effect heterogeneity across target populations.

ABSTRACT: Methods have been developed for transporting evidence from randomised controlled trials (RCTs) to target populations. However, these approaches allow only for differences in characteristics observed in the RCT and real-world data (overt heterogeneity). These approaches do not recognise heterogeneity of treatment effects (HTE) according to unmeasured characteristics (essential heterogeneity). We use a target trial design and apply a local instrumental variable (LIV) approach to electronic health records from the Clinical Practice Research Datalink, and examine both forms of heterogeneity in assessing the comparative effectiveness of two second-line treatments for type 2 diabetes mellitus. We first estimate individualised estimates of HTE across the entire target population defined by applyi

SUBMITTER: Lugo-Palacios DG 

PROVIDER: S-EPMC11631826 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature