Basket Studies: Redefining Clinical Trials in the Era of Genome-Driven Oncology

Annu Rev Med. 2018 Jan 29:69:319-331. doi: 10.1146/annurev-med-062016-050343. Epub 2017 Nov 9.

Abstract

Understanding a tumor's detailed molecular profile has become increasingly necessary to deliver the standard of care for patients with advanced cancer. Innovations in both tumor genomic sequencing technology and the development of drugs that target molecular alterations have fueled recent gains in genome-driven oncology care. "Basket studies," or histology-agnostic clinical trials in genomically selected patients, represent one important research tool to continue making progress in this field. We review key aspects of genome-driven oncology care, including the purpose and utility of basket studies, biostatistical considerations in trial design, genomic knowledgebase development, and patient matching and enrollment models, which are critical for translating our genomic knowledge into clinically meaningful outcomes.

Keywords: basket study; basket trial; genome-driven oncology; knowledgebase; precision medicine.

Publication types

  • Review

MeSH terms

  • Biomarkers, Tumor / genetics*
  • Clinical Trials as Topic*
  • Genomics*
  • Humans
  • Medical Oncology*
  • Molecular Targeted Therapy
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Precision Medicine*

Substances

  • Biomarkers, Tumor