Phase II cancer clinical trials for biomarker-guided treatments

J Biopharm Stat. 2018;28(2):256-263. doi: 10.1080/10543406.2017.1372777. Epub 2017 Nov 27.


The design and analysis of cancer clinical trials with biomarker depend on various factors, such as the phase of trials, the type of biomarker, whether the used biomarker is validated or not, and the study objectives. In this article, we demonstrate the design and analysis of two Phase II cancer clinical trials, one with a predictive biomarker and the other with an imaging prognostic biomarker. Statistical testing methods and their sample size calculation methods are presented for each trial. We assume that the primary endpoint of these trials is a time to event variable, but this concept can be used for any type of endpoint.

Keywords: Enrichment trial; interaction; predictive biomarker; prognostic biomarker; progression-free survival; stratified randomized trial.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Biomarkers, Tumor*
  • Clinical Trials, Phase II as Topic / methods*
  • Clinical Trials, Phase II as Topic / statistics & numerical data
  • Endpoint Determination / methods*
  • Humans
  • Medical Oncology / methods*
  • Medical Oncology / statistics & numerical data
  • Molecular Targeted Therapy
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Neoplasms / metabolism
  • Probability
  • Research Design
  • Sample Size


  • Biomarkers, Tumor