Bayesian Analysis with R for Drug Development : Concepts, Algorithms, and Case Studies (Paperback)

SECTION IBackground1. Bayesian Statistics in Pharmaceutical DevelopmentIntroductionOverview of Drug DevelopmentBasic ResearchDrug DiscoveryFormulationLaboratory Test MethodsPre-Clinical StudiesClinical DevelopmentTranslational ResearchChemical Manufacturing and ControlRegulatory RegistrationStatistics in Drug Research and DevelopmentBayesian StatisticsOpportunities of Bayesian ApproachPre-Clinical DevelopmentCMC DevelopmentClinical TrialsChallenges of Bayesian ApproachObjection to BayesianRegulatory HurdlesConcluding Remarks2. Basics of Bayesian StatisticsIntroductionStatistical InferenceResearch QuestionsProbability DistributionFrequentist MethodsBayesian InferenceSelection of PriorsBayesian ComputationMonte Carlo SimulationExampleMarkov Chain Monte CarloComputation ToolsBUGS and JAGSSAS PROC MCMCUtility of JAGSConcluding Remarks3. Bayesian Estimation of Sample Size and PowerIntroductionSample Size DeterminationFrequentist MethodsBayesian ConsiderationsBayesian ApproachesPower and Sample SizeInterim AnalysisFutility and Sample SizeCase ExampleModelling of Overall SurvivalMaximum Likelihood EstimationFutility AnalysisConcluding RemarksSECION II Pre-Clinical and Clinical Research4. Pre-Clinical Efficacy StudyIntroductionEvaluation of Lab-Based Drugs in CombinationBackgroundStatistical MethodsAntiviral CombinationEvaluation of Fixed Dose CombinationBayesian Survival AnalysisLimitations of Animal DataCurrent MethodsBayesian SolutionCase ExampleConcluding Remarks5. Bayesian Adaptive Design for Phase I Dose-Finding StudiesIntroductionAlgorithm-Based Design3+3 DesignAlternate Algorithm-Based DesignsAdvantages and Disadvantages of Algorithm-Based DesignsModel Based DesignsContinual Reassessment MethodsCRM for Phase I Cancer TrialsEscalation with Overdose ControlEscalation Based on Toxicity IntervalsConcluding Remarks6. Design and Analysis of Phase II Dose-Ranging StudiesIntroductionPhase II Dose-Ranging StudiesCriticisms of Traditional MethodsModel-Based ApproachesEstimating Predictive Precision and Assurance for New TrialCOPD StudyEstimation MethodConcluding Remarks7. Bayesian Multi-Stage Designs for Phase II Clinical TrialsIntroductionPhase II Clinical TrialsMulti-Stage DesignsFrequentist ApproachesBayesian MethodsBayesian Single-Arm TrialsContinuous Monitoring of Single-Arm TrialsComparative Phase II StudiesExamplesOncology TrialMulti-Stage Bayesian DesignConcluding RemarksSECTION III Chemistry, Manufacturing, and Control8. Analytical MethodsIntroductionMethod ValidationBackgroundStudy Design for Validation of Accuracy and PrecisionCurrent Statistical MethodsTotal Error ApproachBayesian SolutionsExampleMethod TransferBackgroundModelLinear ResponseCase ExampleConcluding Remarks9. Process DevelopmentIntroductionQuality by DesignCritical Quality AttributesRisk of OncogenicityBayesian Risk AssessmentModeling Enzyme Cutting EfficiencyBayesian SolutionExampleDesign SpaceDefinitionStatistical Methods for Design SpaceBayesian Design SpaceExampleProcess ValidationRisk-Based Lifecycle ApproachMethod Based on Process CapabilityMethod Based on Predictive PerformanceDetermination of Number of PPQ BatchesConcluding Remarks10. StabilityIntroductionStability StudyShelf-Life EstimationCurrent MethodsBayesian ApproachesExamplesSelection of Stability DesignBayesian CriterionSetting Release LimitsConcluding Remarks11. Process ControlIntroductionQuality Control and ImprovementControl ChartsTypes of Control ChartsShewhart I-MR ChartEWMA Control ChartCUSUM Control ChartJ-ChartMultivariate Control ChartBayesian Control ChartsControl Chart for Data with CensoringControl Chart for Discrete DataControl Limit for Aberrant DataProduct Quality Control Based on Safety Data from SurveillanceConcluding Remarks