N-of-1 trials: The epitome of personalized medicine?

Joyce P Samuel; Susan H Wootton; Jon E Tyson

doi:10.1017/cts.2023.583

N-of-1 trials: The epitome of personalized medicine?

J Clin Transl Sci. 2023 Jul 3;7(1):e161. doi: 10.1017/cts.2023.583. eCollection 2023.

Authors

Joyce P Samuel¹, Susan H Wootton¹, Jon E Tyson¹

Affiliation

¹ Department of Pediatrics, Center for Clinical Research and Evidence-Based Medicine, McGovern Medical School, The University of Texas Health Science Center at Houston, Houston, TX, USA.

Abstract

Observational studies are notoriously susceptible to bias, and parallel-group randomized trials are important to identify the best overall treatment for eligible patients. Yet, such trials can be expected to be a misleading indicator of the best treatment for some subgroups or individual patients. In selected circumstances, patients can be treated in n-of-1 trials to address the inherent heterogeneity of treatment response in clinical populations. Such trials help to accomplish the ultimate goal of all biomedical research, to optimize the care of individual patients.

Keywords: Personalized medicine; n-of-1 trials; precision medicine; randomized trials.