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Design and Implementation of Observational Studies Emulating a Target Trial - PubMed

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  • #Target Trial Emulation
  • #Observational Studies
  • #Causal Inference
  • Target trial emulation (TTE) is increasingly used to estimate causal effects with observational data.
  • The study reviewed 237 TTE studies published from 2017 to 2023 in top clinical journals.
  • Over half (54.0%) of the studies evaluated drug interventions, mainly in infectious diseases, cardiology, and oncology.
  • TTE was applied in 8 recurring clinical scenarios, including RCT replication and extending evidence to underrepresented populations.
  • Major methodologic gaps were identified: only 56.5% had a prespecified protocol, and 53.6% did not review existing RCTs.
  • During implementation, 43.5% of studies did not report all 7 methodologic components, and only 30.8% addressed unmeasured confounding.
  • A 5-step framework for target trial design was developed to improve future TTE studies.
  • The study highlights common pitfalls and provides recommendations to enhance observational evidence credibility.