Baldwin, FD 
ORCID: 0000-0002-9425-791X, Khalaf, RKS ORCID: 0000-0002-7666-0617, Kolamunnage-Dona, R ORCID: 0000-0003-3886-6208 and Jorgensen, AL ORCID: 0000-0002-6977-9337
(2025)
Methodologies for the Emulation of Biomarker-Guided Trials Using Observational Data: A Systematic Review
Journal of Personalized Medicine, 15 (5).
195-.
ISSN 2075-4426, 2075-4426
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Text
Methodologies for the Emulation of Biomarker-Guided Trials Using Observational Data A Systematic Review.pdf - Open Access published version Available under License Creative Commons Attribution. Download (1MB) | Preview |
Abstract
Background: Target trial emulation involves the application of design principles from randomised controlled trials (RCTs) to observational data, and is particularly useful in situations where an RCT would be unfeasible. Biomarker-guided trials, which incorporate biomarkers within their design to either guide treatment and/or determine eligibility, are often unfeasible in practice due to sample size requirements or ethical concerns. Here, we undertake a systematic review of methodologies used in target trial emulations, comparing treatment effectiveness, critically appraising them, and considering their applicability to the emulation of biomarker-guided trials. Methods: A comprehensive search strategy was developed to identify studies reporting on methods for target trial emulation comparing the effectiveness of treatments using observational data, and applied to the following bibliographic databases: PubMed, Scopus, Web of Science, and Ovid MEDLINE. A narrative description of methods identified in the review was undertaken alongside a critique of their relative strengths and limitations. Results: We identified a total of 59 papers: 47 emulating a target trial (‘application’ studies), and 12 detailing methods to emulate a target trial (‘methods’ studies). A total of 25 papers were identified as emulating a biomarker-guided trial (42%). While all papers reported methods to adjust for baseline confounding, 40% of application papers did not specify methods to adjust for time-varying confounding. Conclusions: This systematic review has identified a range of methods used to control for baseline, time-varying, and residual/unmeasured confounding within target trial emulation and provides a guide for researchers interested in emulation of biomarker-guided trials.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | biomarker-guided trials, personalised medicine, target trial emulation |
| Divisions: | Faculty of Health & Life Sciences Faculty of Health & Life Sciences > Inst. Population Health Faculty of Health & Life Sciences > Inst. Population Health > Inst. Population Health Faculty of Health & Life Sciences > Inst. Population Health > Health Data Science Faculty of Health & Life Sciences > Inst. Population Health > Inst. Population Health (T&R Staff) |
| Depositing User: | Symplectic Admin |
| Date Deposited: | 16 Jan 2026 11:26 |
| Last Modified: | 23 May 2026 10:14 |
| DOI: | 10.3390/jpm15050195 |
| Related Websites: | |
| URI: | https://livrepository.liverpool.ac.uk/id/eprint/3196631 |
| Disclaimer: | The University of Liverpool is not responsible for content contained on other websites from links within repository metadata. Please contact us if you notice anything that appears incorrect or inappropriate. |
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