Individualized therapy trials: navigating patient care, research goals and ethics
- 12 October 2021
- journal article
- research article
- Published by Springer Science and Business Media LLC in Nature Medicine
- Vol. 27 (10), 1679-1686
- https://doi.org/10.1038/s41591-021-01519-y
Abstract
‘Individualized therapy’ trials (sometimes called n-of-1 trials) use patients as their own controls to evaluate treatments. Here we divide such trials into three categories: multi-crossover trials aimed at individual patient management, multi-crossover trial series and pre–post trials. These trials all customize interventions for patients; however, the latter two categories also aim to inform medical practice and thus embody tensions between the goals of care and research that are typical of other types of clinical trials. In this Perspective, we discuss four domains where such tensions play out—clinical equipoise, informed consent, reporting and funding, and we provide recommendations for addressing each.Keywords
This publication has 85 references indexed in Scilit:
- Experimental designs for small randomised clinical trials: an algorithm for choiceOrphanet Journal of Rare Diseases, 2013
- A theoretical framework for early human studies: uncertainty, intervention ensembles, and boundariesTrials, 2012
- N-of-1 Trials in the Medical LiteratureMedical Care, 2011
- The ClinicalTrials.gov Results Database — Update and Key IssuesThe New England Journal of Medicine, 2011
- The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?Personalized Medicine, 2011
- Individual (N-of-1) trials can be combined to give population comparative treatment effect estimates: methodologic considerationsJournal of Clinical Epidemiology, 2010
- The culture of faith and hopeCancer, 2010
- What Ever Happened to N‐of‐1 Trials? Insiders' Perspectives and a Look to the FutureThe Milbank Quarterly, 2008
- Equipoise and the Ethics of Clinical ResearchThe New England Journal of Medicine, 1987
- Determining Optimal Therapy — Randomized Trials in Individual PatientsThe New England Journal of Medicine, 1986