Get 20M+ Full-Text Papers For Less Than $1.50/day. Start a 14-Day Trial for You or Your Team.

Learn More →

The role of statistics in the design and analysis of companion diagnostic (CDx) studies

The role of statistics in the design and analysis of companion diagnostic (CDx) studies Companion diagnostic tests are crucial in the development of precision medicine. These tests provide information that is essential for the safe and effective use of specific therapeutic products. Statistics plays a key role in the design and analysis of studies to demonstrate the safety and effectiveness of the companion diagnostics. This article can serve as an introduction to companion diagnostics for therapeutic statisticians and for diagnostic ones as well as a discussion of some of the statistical challenges. The topics include biomarker development, diagnostic performance, misclassification, prospective-retrospective validation, bridging studies, missing data, follow-on diagnostics and complex signatures. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Biostatistics & Epidemiology Taylor & Francis

The role of statistics in the design and analysis of companion diagnostic (CDx) studies

Campbell, Gregory
Biostatistics & Epidemiology , Volume 5 (2): 14 – Jul 3, 2021
Download PDF

The role of statistics in the design and analysis of companion diagnostic (CDx) studies

Abstract

Companion diagnostic tests are crucial in the development of precision medicine. These tests provide information that is essential for the safe and effective use of specific therapeutic products. Statistics plays a key role in the design and analysis of studies to demonstrate the safety and effectiveness of the companion diagnostics. This article can serve as an introduction to companion diagnostics for therapeutic statisticians and for diagnostic ones as well as a discussion of some of the...
Loading next page...
 
/lp/taylor-francis/the-role-of-statistics-in-the-design-and-analysis-of-companion-zoI300Qegh
Publisher
Taylor & Francis
Copyright
© 2021 International Biometric Society – Chinese Region
ISSN
2470-9379
eISSN
2470-9360
DOI
10.1080/24709360.2021.1913706
Publisher site
See Article on Publisher Site

Abstract

Companion diagnostic tests are crucial in the development of precision medicine. These tests provide information that is essential for the safe and effective use of specific therapeutic products. Statistics plays a key role in the design and analysis of studies to demonstrate the safety and effectiveness of the companion diagnostics. This article can serve as an introduction to companion diagnostics for therapeutic statisticians and for diagnostic ones as well as a discussion of some of the statistical challenges. The topics include biomarker development, diagnostic performance, misclassification, prospective-retrospective validation, bridging studies, missing data, follow-on diagnostics and complex signatures.

Journal

Biostatistics & Epidemiology – Taylor & Francis

Published: Jul 3, 2021

Keywords: Co-development; predictive biomarker; diagnostic performance; bridging studies; complex signatures

References

  • Clinical trial designs of predictive biomarker validation: theoretical considerations and practical challenges
  • Receiver operating characteristic plots: a fundamental evaluation tool in clinical medicine
  • Personalized medicine: Four perspectives of tailored medicine
  • Adaptive randomization of neratinib in early breast cancer
  • Partly nonparametric approach for determining the limit of detection
  • Analytical and clinical evaluation of biomarkers assays: when are biomarkers ready for prime time?
  • Towards complete and accurate reporting of studies of diagnostic accuracy: the STARD initiative
  • Laboratory assessment of the status of Her-2/neu protein and oncogene in breast cancer specimens: comparison of immunohistochemistry assay with fluorescence in situ hybridisation assays
  • HER2 testing: current status and future directions
  • The impact of companion diagnostic device measurement performance on clinical validation of personalized medicine
  • Stratification and partial ascertainment of biomarker-driven clinical trials
  • Liquid biopsy preservation solutions for standardized pre-analytical workflows—venous whole blood and plasma
  • Statistical consideration and challenges in bridging study of personalized medicine
  • Statistical evaluation of drug efficacy for bridging study in companion diagnostic test trials
  • Missing data in the regulation of medical devices
  • Missing data handling methods in medical device clinical trials
  • Identifying a subpopulation for a tailored therapy: bridging clinical efficacy from a laboratory-developed assay to a validated in vitro diagnostic test kit
  • Statistical methods for clinical validation of follow-on companion diagnostic devices via an external concordance study
  • Clinical trials designs for predictive biomarker validation: theoretical considerations and practical challenges
  • An FDA perspective on the regulatory implications of complex signature to predict response to targeted therapies
  • Jones WD, et. al. The microarray quality control (MAQC) II study of common practices for development and validation of microarray based predictive models
  • . Validation and clinical utility of a 70-gene prognostic signature for women with node-negative breast cancer
  • Lessons for tumor biomarker trials: vicious cycles, scientific method & developing guidelines
  • Evolving Japanese regulations on companion diagnostics