{"about":{"site":"https://codewithpapers.app","non_affiliation":"Code with Papers and Syntology are not affiliated with, endorsed by, or sponsored by Papers with Code, Meta, or the pwc-archive mirror.","licence":"CC BY-SA 4.0","licence_url":"https://creativecommons.org/licenses/by-sa/4.0/legalcode","attribution":"https://codewithpapers.app/attribution","modified":"archive material modified by Syntology; see the attribution page"},"url":"/paper/causal-inference-for-n-of-1-trials","title":"Causal inference for N-of-1 trials","arxiv_id":"2406.10360","date":"2024-06-14","proceeding":null,"authors":["Marco Piccininni","Mats J. Stensrud","Zachary Shahn","Stefan Konigorski"],"abstract":"The aim of personalized medicine is to tailor treatment decisions to individuals' characteristics. N-of-1 trials are within-person crossover trials that hold the promise of targeting individual-specific effects. While the idea behind N-of-1 trials might seem simple, analyzing and interpreting N-of-1 trials is not straightforward. Here we ground N-of-1 trials in a formal causal inference framework and formalize intuitive claims from the N-of-1 trials literature. We focus on causal inference from a single N-of-1 trial and define a conditional average treatment effect (CATE) that represents a target in this setting, which we call the U-CATE. We discuss assumptions sufficient for identification and estimation of the U-CATE under different causal models where the treatment schedule is assigned at baseline. A simple mean difference is an unbiased, asymptotically normal estimator of the U-CATE in simple settings. We also consider settings where carryover effects, trends over time, time-varying common causes of the outcome, and outcome-outcome effects are present. In these more complex settings, we show that a time-varying g-formula identifies the U-CATE under explicit assumptions. Finally, we analyze data from N-of-1 trials about acne symptoms and show how different assumptions about the data generating process can lead to different analytical strategies.","url_abs":"https://arxiv.org/abs/2406.10360v2","url_pdf":"https://arxiv.org/pdf/2406.10360v2.pdf","source":{"archive":"pwc-archive (Hugging Face), CC BY-SA 4.0","snapshot":"2025-07-28","licence_url":"https://creativecommons.org/licenses/by-sa/4.0/legalcode","row_kind":"links_only","authors_date_abstract":"arXiv metadata, CC0 1.0 (https://info.arxiv.org/help/license), from the Kaggle arXiv metadata snapshot of 2026-09-12"},"code_links":[{"paper_slug":"causal-inference-for-n-of-1-trials","repo_url":"https://github.com/MarcoPic-dot/Causal_Inference_Nof1_trial_acne","is_official":1,"mentioned_in_paper":1,"mentioned_in_github":1,"framework":"none","reach":null}],"tasks":[],"methods":[],"datasets_introduced":[],"methods_introduced":[],"results":[],"syntology":{"syntology_url":null,"atlas_url":null,"mcp":null,"developers":"https://syntology.ai/developers"},"arxiv_metadata":null,"syntology_extracted_results":null}