A new statistical decision rule for single-arm phase II oncology trials

Research output: Contribution to journalArticle

3 Citations (Scopus)

Abstract

Most single-arm phase II clinical trials compare the efficacy of a new treatment with historical controls through statistical hypothesis testing. One major problem with such a comparison is that the efficacy of the historical control is treated as a known constant, whereas in reality, it is never precisely known. This partially explains why many "Go" decisions made in single-arm phase II trials are shown to be incorrect in phase III trials. In this paper, we propose a new decision rule for an improved transitional decision for single-arm phase II oncology clinical trials with binary endpoints. This new decision rule is jointly based on the p value and a new statistical index named the testing confidence value. The testing confidence value reflects the uncertainty associated with the null value in the hypothesis testing of single-arm trials. Simulations are used to evaluate the operating characteristics of the new decision rule in comparison with the traditional decision rule and a widely used Bayesian decision rule. The application of the new decision rule is illustrated using a clinical trial on marginally resectable pancreatic cancer. A webpage http://www.yiyichenbiostatistics.com/TCV.HTML is available for readers to interactively compute the testing confidence value and to find the suggested decision based on the new decision rule.

Original languageEnglish (US)
Pages (from-to)118-132
Number of pages15
JournalStatistical Methods in Medical Research
Volume25
Issue number1
DOIs
StatePublished - Feb 1 2016

Fingerprint

Oncology
Phase II Clinical Trials
Decision Rules
Pancreatic Neoplasms
Uncertainty
Clinical Trials
Confidence
Hypothesis Testing
Testing
Efficacy
HTML
Operating Characteristics
p-Value
Null
Cancer
Binary
Evaluate

Keywords

  • "Go/No Go" decision
  • oncology trials
  • phase II clinical trials
  • testing confidence value
  • Transitional decision

ASJC Scopus subject areas

  • Epidemiology
  • Health Information Management
  • Statistics and Probability

Cite this

A new statistical decision rule for single-arm phase II oncology trials. / Chen, Yiyi; Chen, Zunqiu; Mori, Motomi (Tomi).

In: Statistical Methods in Medical Research, Vol. 25, No. 1, 01.02.2016, p. 118-132.

Research output: Contribution to journalArticle

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