A review of statistical designs for improving the efficiency of phase II studies in oncology

Stat Methods Med Res. 2016 Jun;25(3):1010-21. doi: 10.1177/0962280215588247. Epub 2015 May 31.


Phase II oncology trials are carried out to assess whether an experimental anti-cancer treatment shows sufficient signs of effectiveness to justify being tested in a phase III trial. Traditionally such trials are conducted as single-arm studies using a binary response rate as the primary endpoint. In this article, we review and contrast alternative approaches for such studies. Each approach uses only data that are necessary for the traditional analysis. We consider two broad classes of methods: ones that aim to improve the efficiency using novel design ideas, such as multi-stage and multi-arm multi-stage designs; and ones that aim to improve the analysis, by making better use of the richness of the data that is ignored in the traditional analysis. The former class of methods provides considerable gains in efficiency but also increases the administrative and logistical issues in running the trial. The second class consists of viable alternatives to the standard analysis that come with little additional requirements and provide considerable gains in efficiency.

Keywords: RECIST; augmented methods; continuous; multi-stage; oncology; phase II.

Publication types

  • Review

MeSH terms

  • Biomedical Research / methods
  • Clinical Trials, Phase II as Topic / methods*
  • Humans
  • Medical Oncology / methods*
  • Neoplasms / drug therapy*
  • Research Design*