Are Target Trial Emulations the Gold Standard for Observational Studies?

2023/05/31 by Neil Pearce, Jan P. Vandenbroucke · 2 citations
Decision Sciences · Economics, Econometrics and Finance · Mathematics · #Health Systems, Economic Evaluations, Quality of Life #Meta-analysis and systematic reviews #Statistical Methods in Clinical Trials

paper · doi:10.1097/ede.0000000000001636

openalex publication_date 2023/05/31 · openalex created_date 2025/10/10 · openalex updated_date 2026/08/01

Abstract

There has been considerable debate in epidemiology about whether the randomized controlled trial (RCT) is the gold standard for epidemiologic studies; some of the debate is old, but some is new. Almost all are agreed that a well-conducted RCT, where possible and appropriate, is to be preferred because an experimental set-up enables control of several key features of a study, and randomization ensures that, on the average, and provided that there are no other biases (e.g., in the ascertainment of the outcome), that the groups being compared are comparable (exchangeable) with regards to baseline (i.e., in the absence of exposure) outcome risk. It is thus tempting to apply the RCT paradigm to observational studies, that is, to propose that if an RCT is not possible for a particular issue, then an observational study that closely mimics the RCT approach is the most preferable.

Citations

• Using Big Data to Emulate a Target Trial When a Randomized Trial Is Not Available: Table 1.
• Triangulation in aetiological epidemiology
• Target trial emulation: teaching epidemiology and beyond
• Electronic medical records can be used to emulate target trials of sustained treatment strategies
• Mendel’s laws, Mendelian randomization and causal inference in observational data: substantive and nomenclatural issues

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