title: Genomic biomarkers for personalized medicine: development and validation in clinical studies.
abstract: The establishment of high-throughput technologies has brought substantial advances to our understanding of the biology of many diseases at the molecular level and increasing expectations on the development of innovative molecularly targeted treatments and molecular biomarkers or diagnostic tests in the context of clinical studies. In this review article, we position the two critical statistical analyses of high-dimensional genomic data, gene screening and prediction, in the framework of development and validation of genomic biomarkers or signatures, through taking into consideration the possible different strategies for developing genomic signatures. A wide variety of biomarker-based clinical trial designs to assess clinical utility of a biomarker or a new treatment with a companion biomarker are also discussed.
authors.affiliation: Department of Data Science, The Institute of Statistical Mathematics, 10-3 Midori-cho, Tachikawa, Tokyo 190-8562, Japan. smatsui@ism.ac.jp
authors.forename: Shigeyuki
authors.initials: S
authors.lastname: Matsui
mesh_terms: D015415:Biomarkers / Q000032:analysis; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data; D019295:Computational Biology; D005819:Genetic Markers; D005820:Genetic Testing / Q000706:statistics & numerical data; D006801:Humans; D015233:Models, Statistical; D057285:Precision Medicine; D011237:Predictive Value of Tests; D011379:Prognosis; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data
chemical_list: D015415:Biomarkers; D005819:Genetic Markers