Individualized therapy trials: navigating patient care, research goals and ethics

Nat Med. 2021 Oct;27(10):1679-1686. doi: 10.1038/s41591-021-01519-y. Epub 2021 Oct 12.

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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Clinical Trials as Topic*
  • Humans
  • Informed Consent*
  • Patient Care / trends*
  • Precision Medicine / trends*
  • Research Design

Grants and funding