A comparison of phase II study strategies

Clin Cancer Res. 2009 Oct 1;15(19):5950-5. doi: 10.1158/1078-0432.CCR-08-3205. Epub 2009 Sep 29.

Abstract

The traditional oncology drug development paradigm of single arm phase II studies followed by a randomized phase III study has limitations for modern oncology drug development. Interpretation of single arm phase II study results is difficult when a new drug is used in combination with other agents or when progression-free survival is used as the endpoint rather than tumor shrinkage. Randomized phase II studies are more informative for these objectives but increase both the number of patients and time required to determine the value of a new experimental agent. In this article, we compare different phase II study strategies to determine the most efficient drug development path in terms of number of patients and length of time to conclusion of drug efficacy on overall survival.

Publication types

  • Comparative Study
  • Evaluation Study
  • Review

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Clinical Trials, Phase II as Topic / methods*
  • Clinical Trials, Phase II as Topic / trends
  • Clinical Trials, Phase III as Topic / methods
  • Clinical Trials, Phase III as Topic / trends
  • Drug Design
  • Humans
  • Neoplasms / drug therapy*
  • Neoplasms / mortality
  • Randomized Controlled Trials as Topic / methods
  • Randomized Controlled Trials as Topic / trends
  • Survival Analysis
  • Time Factors
  • Treatment Outcome