Group sequential designs for developing and testing biomarker-guided personalized therapies in comparative effectiveness research

Contemp Clin Trials. 2013 Nov;36(2):651-63. doi: 10.1016/j.cct.2013.08.007. Epub 2013 Aug 29.

Abstract

Biomarker-guided personalized therapies offer great promise to improve drug development and improve patient care, but also pose difficult challenges in designing clinical trials for the development and validation of these therapies. We first give a review of the existing approaches, briefly for clinical trials in new drug development and in more detail for comparative effectiveness trials involving approved treatments. We then introduce new group sequential designs to develop and test personalized treatment strategies involving approved treatments.

Keywords: Adaptive randomization; Biomarker classifiers; Generalized likelihood ratio statistics; Group sequential design; Multiple testing; Targeted therapies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Bayes Theorem
  • Biomarkers / metabolism*
  • Clinical Protocols
  • Comparative Effectiveness Research / methods*
  • Comparative Effectiveness Research / standards
  • Drug Therapy
  • Humans
  • Models, Statistical
  • Precision Medicine / methods*
  • Precision Medicine / standards
  • Randomized Controlled Trials as Topic / methods
  • Randomized Controlled Trials as Topic / standards

Substances

  • Biomarkers