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

• Published in: Statistics in medicine Vol. 36; no. 19; pp. 2994 - 3004
• Main Authors: Gao, Fei, Dong, Jun, Zeng, Donglin, Rong, Alan, Ibrahim, Joseph G.
• Format: Journal Article
• Language: English
• Published: England Wiley Subscription Services, Inc 30.08.2017
• Subjects: Algorithms; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Biomarkers; Biometry - methods; Cancer therapies; Clinical trials; Clinical Trials, Phase III as Topic; Colorectal cancer; Colorectal Neoplasms - drug therapy; companion diagnostics; Computer Simulation; Humans; Medical statistics; Missing data; Proportional Hazards Models; Proto-Oncogene Proteins p21(ras) - drug effects; Randomized Controlled Trials as Topic; Regression Analysis; United States > US; companion diagnostics; clinical trials; missing data

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...