10. Targeted Therapy Design

  1. Guosheng Yin

Published Online: 9 JAN 2012

DOI: 10.1002/9781118183335.ch10

Clinical Trial Design: Bayesian and Frequentist Adaptive Methods

Clinical Trial Design: Bayesian and Frequentist Adaptive Methods

How to Cite

Yin, G. (2011) Targeted Therapy Design, in Clinical Trial Design: Bayesian and Frequentist Adaptive Methods, John Wiley & Sons, Inc., Hoboken, NJ, USA. doi: 10.1002/9781118183335.ch10

Publication History

  1. Published Online: 9 JAN 2012
  2. Published Print: 19 DEC 2011

ISBN Information

Print ISBN: 9780470581711

Online ISBN: 9781118183335



  • adaptive signature design;
  • adaptive threshold design;
  • contingency tables;
  • hypothesis testing;
  • predictive biomarkers;
  • prognostic marker;
  • randomized discontinuation design (RDD);
  • targeted therapy design


Targeted therapy is typically developed along with important biomarkers that regulate cell signaling, malignant transformation, and proliferation. To discriminate prognostic and predictive biomarkers, this chapter creates four 2 x 2 contingency tables, in which each cell represents the response rate of that specific group. It also describes the randomized discontinuation design (RDD), adaptive signature design, and adaptive threshold design. The adaptive signature design can achieve three goals: to identify the subset of patients who are most likely to benefit from the new agent; to maintain a properly powered test of an overall treatment effect at the end of the trial using all randomized patients; and to test the treatment effect for the subset of sensitive patients selected from the patients enrolled in stage 2. Finally, the chapter discusses two different procedures for hypothesis testing.

Controlled Vocabulary Terms

generalized randomized block design; hypothesis testing