N-of-1 Trials in Cancer Drug Development

Cancer Discov. 2023 Jun 2;13(6):1301-1309. doi: 10.1158/2159-8290.CD-22-1377.

Abstract

The current approaches for cancer drug development lag behind an accelerated need in the field for a fast and efficient method for evaluating drugs in the personalized medicine era. In that regard, N-of-1 studies emerge as a potential addition to the drug development arsenal, although there are several considerations before its broad application becomes feasible. In essence, N-of-1 trials are a departure from the traditional "drug-centric" model to a "patient-centric" model. Herein, we review the concept of N-of-1 trials and provide real-world examples of their use in the developmental therapeutics field. N-of-1 trials offer an exceptional opportunity for fast-tracking of cancer drug development in the precision oncology era.

Publication types

  • Review
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents* / pharmacology
  • Antineoplastic Agents* / therapeutic use
  • Drug Development
  • Humans
  • Medical Oncology / methods
  • Neoplasms* / drug therapy
  • Neoplasms* / genetics
  • Precision Medicine / methods

Substances

  • Antineoplastic Agents