Innovative Biomarker-Integrated Clinical Trial Design and Analysis

Led by: Jianwen Cai, PhD

With advances in biomedical technology, biomarkers are playing an increasingly important role in disease prognosis and treatment selection for cancer patients. Innovative clinical trial designs that incorporate biomarker information can improve study efficiency, require fewer patients, and reduce costs, thereby accelerating cancer drug development. The primary objective of this project is to develop new and innovative statistical methodology to address issues in the design and analysis of biomarker-integrated clinical trials. We focus on the following four specific aims:

  1. Develop statistical design and analysis methods for clinical trials with baseline biomarkers.
  2. Develop methods for incorporation of pre-randomization longitudinal biomarkers.
  3. Develop design and analysis methods for biomarker-enrichment trials.
  4. Develop two-stage trials for discovery of predictive biomarkers.