What is a Target Trial Emulation, NIHR RSS Research Methods Academy

Session starts at 7 minutes into the recording. Presented by Ruth Keogh is Professor Biostatistics and Epidemiology in the Medical Statistics Department at the London School of Hygiene & Tropical Medicine (LSHTM), and co-director of the LSHTM Centre for Data and Statistical Science for Health (DASH). Chaired by Dr Will Hulme, statistical epidemiologist at the Bennett Institute for Applied Data Science, Nuffield Department of Primary Care Health Sciences, University of Oxford. The target trial emulation (TTE) framework helps us to study the causal effects of interventions (e.g. treatments, policies) using observational data. Randomised controlled trials are the gold standard for studying the effects of interventions, but not all important questions can be answered using a trial – including questions about effects in certain patient groups, about long-term effects, and complex treatment strategies. Observational data offer opportunities to study questions that would not be possible otherwise. However, this is challenging, not only due to confounding, but because of how information on treatments and patient characteristics are observed over time. We need to think carefully about designing such studies to avoid biases, and the TTE framework helps us to do this. TTE involves two steps: (1) describing the protocol for the “target trial” that would answer the causal question; (2) emulating each protocol component using the observational data. This talk will introduce the TTE framework and discuss good practice and challenges. We will cover examples and recently proposed TTE reporting guidelines. We will not give details of specific statistical analysis methods, but will highlight the need to tailor the analysis to the question and correctly consider challenges arising for different treatment strategies. Key Links: Hernán MA, Dahabreh IJ, Dickerman BA, Swanson SA. The Target Trial Framework for Causal Inference From Observational Data: Why and When Is It Helpful? Ann Intern Med. 2025 Mar;178(3):402-407. doi: 10.7326/ANNALS-24-01871. Cashin, A. G.; Hansford, H. J.; Hernán, M. A.; Swanson, S. A.; Lee, H.; Jones, M. D.; Dahabreh, I. J.; Dickerman, B. A.; Egger, M.; Garcia-Albeniz, X.; Golub, R. M.; Islam, N.; Lodi, S.; Moreno-Betancur, M.; Pearson, S.-A.; Schneeweiss, S.; Sharp, M. K.; Sterne, J. A. C.; Stuart, E. A.; McAuley, J. H. Transparent Reporting of Observational Studies Emulating a Target Trial—The TARGET Statement. JAMA 2025, 334 (12), 1084–1093. https://doi.org/10.1001/jama.2025.13350. BMJ 2025. https://doi.org/10.1136/bmj-2025-087179.