Applied Statistics in Biomedicine and Clinical Trials Design: Selected Papers from 2013 ICSA/ISBS Joint Statistical Meetings (ICSA Book Series in Statistics)

This volume is a unique combination of papers that cover critical topics in biostatistics from academic, government, and industry perspectives. The 6 sections cover Bayesian methods in biomedical research; Diagnostic medicine and classification; Innovative Clinical Trials Design; Modelling and Data Analysis; Personalized Medicine; and Statistical Genomics. The real world applications are in clinical trials, diagnostic medicine and genetics. The peer-reviewed contributions were solicited and selected from some 400 presentations at the annual meeting of the International Chinese Statistical Association (ICSA), held with the International Society for Biopharmaceutical Statistics (ISBS). The conference was held in Bethesda in June 2013, and the material has been subsequently edited and expanded to cover the most recent developments. Front Matter....Pages i-xxiv Front Matter....Pages 1-1 An Application of Bayesian Approach for Testing Non-inferiority Case Studies in Vaccine Trials....Pages 3-15 Bayesian Design of Noninferiority Clinical Trials with Co-primary Endpoints and Multiple Dose Comparison....Pages 17-33 Bayesian Functional Mixed Models for Survival Responses with Application to Prostate Cancer....Pages 35-59 Bayesian Predictive Approach to Early Termination for Enriched Enrollment Randomized Withdrawal Trials....Pages 61-71 Front Matter....Pages 73-73 Estimation of ROC Curve with Multiple Types of Missing Gold Standard....Pages 75-87 Group Sequential Methods for Comparing Correlated Receiver Operating Characteristic Curves....Pages 89-108 Nonparametric Covariate Adjustment for the Youden Index....Pages 109-132 Comparative Effectiveness Research Using Meta-Analysis to Evaluate and Summarize Diagnostic Accuracy....Pages 133-145 Front Matter....Pages 147-147 Some Characteristics of the Varying-Stage Adaptive Phase II/III Clinical Trial Design....Pages 149-161 Collective Evidence in Drug Evaluation....Pages 163-184 Applications of Probability of Study Success in Clinical Drug Development....Pages 185-196 Treatment Effect Estimation in Adaptive Clinical Trials: A Review....Pages 197-202 Inferiority Index, Margin Functions, and Hybrid Designs for Noninferiority Trials with Binary Outcomes....Pages 203-234 Group-Sequential Designs When Considering Two Binary Outcomes as Co-Primary Endpoints....Pages 235-262 Issues in the Use of Existing Data: As Controls in Pre-Market Comparative Clinical Studies....Pages 263-271 A Two-Tier Procedure for Designing and Analyzing Medical Device Trials Conducted in US and OUS Regions for Regulatory Decision Making....Pages 273-284 Multiplicity Adjustment in Seamless Phase II/III Adaptive Trials Using Biomarkers for Dose Selection....Pages 285-299 Front Matter....Pages 301-301 Empirical Likelihood for the AFT Model Using Kendall’s Rank Estimating Equation....Pages 303-311 Analysis of a Complex Longitudinal Health-Related Quality of Life Data by a Mixed Logistic Model....Pages 313-328 Goodness-of-Fit Tests for Length-Biased Right-Censored Data with Application to Survival with Dementia....Pages 329-345 Front Matter....Pages 301-301 Assessment of Fit in Longitudinal Data for Joint Models with Applications to Cancer Clinical Trials....Pages 347-365 Assessing the Cumulative Exposure Response in Alzheimer’s Disease Studies....Pages 367-380 Evaluation of a Confidence Interval Approach for Relative Agreement in a Crossed Three-Way Random Effects Model....Pages 381-392 Front Matter....Pages 393-393 Assessment of Methods to Identify Patient Subgroups with Enhanced Treatment Response in Randomized Clinical Trials....Pages 395-410 A Framework of Statistical Methods for Identification of Subgroups with Differential Treatment Effects in Randomized Trials....Pages 411-425 Biomarker Evaluation and Subgroup Identification in a Pneumonia Development Program Using SIDES....Pages 427-466 Front Matter....Pages 467-467 A Stochastic Segmentation Model for the Indentification of Histone Modification and DNase I Hypersensitive Sites in Chromatin....Pages 469-493 Combining p Values for Gene Set Analysis....Pages 495-518 A Simple Method for Testing Global and Individual Hypotheses Involving a Limited Number of Possibly Correlated Outcomes....Pages 519-546 "This volume is a unique combination of papers that cover critical topics in biostatistics from academic, government, and industry perspectives. The six sections cover Bayesian methods in biomedical research; Diagnostic medicine and classification; Innovative clinical trials design; Modelling and data analysis; Personalized medicine; and Statistical genomics. The real world applications are in clinical trials, diagnostic medicine and genetics. The peer-reviewed contributions were solicited and selected from some 400 presentations at the annual meeting of the International Chinese Statistical Association (ICSA), held with the International Society for Biopharmaceutical Statistics (ISBS). The conference was held in Bethesda in June 2013, and the material has been subsequently edited and expanded to cover the most recent developments."-- Provided by publisher