Pattern mixture models for clinical validation of biomarkers in the presence of missing data.

@article{Gao2017PatternMM,
  title={Pattern mixture models for clinical validation of biomarkers in the presence of missing data.},
  author={Fei Gao and Jun Dong and Donglin Zeng and Alan Rong and Joseph G. Ibrahim},
  journal={Statistics in medicine},
  year={2017},
  volume={36 19},
  pages={
          2994-3004
        }
}
Targeted therapies for cancers are sometimes only effective in a subset of patients with a particular biomarker status. In clinical development, the biomarker status is typically determined by an investigational-use-only/laboratory-developed test. A market ready test (MRT) is developed later to meet regulatory requirements and for future commercial use. In the USA, the clinical validation of MRT showing efficacy and safety profile of the targeted therapy in the biomarker subgroups determined by… CONTINUE READING