Bayesian Adaptive Design for Immunotherapy and Targeted Therapy

Description

This book provides a comprehensive review of novel adaptive trial designs for targeted therapies and immunotherapies. This book covers a wide range of novel statistical designs for various clinical settings, including early phase dose-escalation study, proof-of-concept trials, and confirmatory studies with registrational. The book includes real-life examples and software to facilitate practitioners to learn and use the designs in practice. Preface Contents Part I Phase I Trials 1 Introduction to Phase I Dose-Finding Clinical Trials 1.1 Phase I Dose-Finding Trials 1.1.1 Continual Reassessment Method (CRM) 1.1.2 Modified Toxicity Probability (mTPI) Design 1.1.3 Keyboard Design 1.1.4 Bayesian Optimal Interval (BOIN) Design 1.1.5 Deviation from the Planned Design 1.1.6 Software 1.2 Challenges of Immunotherapies, Targeted Therapies and New Methods References 2 Phase I Designs for Late-Onset Toxicity 2.1 Model-Based Phase I Designs for the Late-Onset Toxicity 2.1.1 Time-to-Event CRM (TITE-CRM) 2.1.2 Fractional CRM (fCRM) 2.1.3 Data Augmentation CRM (DA-CRM) 2.2 Model-Assisted Phase I Design for the Late-Onset Toxicity 2.2.1 Time-to-Event BOIN (TITE-BOIN) 2.3 Software References Part II Phase I/II Trials 3 Optimal Biological Dose and Phase I/II Trials 3.1 Background 3.2 Elements of a Phase I/II Design 3.3 Literature Review References 4 Model-Based Designs for Identification of Optimal Biological Dose 4.1 EffTox Design 4.2 Logistic Models 4.3 A Bayesian Phase I/II Trial Design for Immunotherapy 4.4 Nonparametric Isotonic Design References 5 Model-Assisted Designs for Identifying the Optimal Biological Dose 5.1 BOIN12 5.2 U-BOIN 5.3 Summary References Part III Phase II Trials 6 Single Arm Phase II Clinical Trial 6.1 Simon's Two-Stage Design 6.2 Limitations of Simon's Two-Stage Design 6.3 Approaches Based on Posterior and Predictive Probabilities 6.4 Bayesian Optimal Phase II Design (BOP2) 6.5 Time-to-Event Bayesian Optimal Phase II (TOP) Trial Design 6.6 Conclusion References 7 Randomized Phase II Designs 7.1 Introduction 7.2 Frequentist Randomized Two-Arm Phase II Designs 7.3 Bayesian Randomized Two-Arm Phase II Design 7.3.1 Randomized BOP2 Design with Binary, Ordinal, and Co-primary Endpoints 7.3.2 Randomized BOP2 Design with the Survival Endpoint 7.3.3 Two-Stage Screened Selection Design (SSD) 7.4 Summary References Part IV Master-Protocol Trials 8 Introduction to Basket Trials 8.1 Introduction 8.2 Basket Design Based on the Binomial-Test 8.2.1 Two-Stage Basket Design 8.2.2 Optimal Two-Stage Basket Design 8.3 Bayesian Hierarchical Model (BHM)-Based Basket Design 8.3.1 Bayesian Hierarchical Model (BHM) 8.3.2 Calibrated Bayesian Hierarchical Model (CBHM) 8.3.3 Optimal Bayesian Hierarchical Model (OBHM) 8.4 Basket Trials with the Multisource Exchangeability Model (MEM) 8.5 Bayesian Basket Trial Design with Predictive Sample Size Determination 8.6 Single-Drug Non-randomized and Multiple-Individual-Drug … 8.7 Extensions to Randomized and Confirmatory Basket Designs 8.7.1 Randomized Basket Design 8.7.2 Confirmatory Basket Design 8.8 Summary References 9 Platform Trials 9.1 Introduction to Platform Trials 9.2 Adaptive Platform Design with the Binary Endpoint 9.2.1 Controlled Multi-arm Platform Design 9.2.2 A Bayesian Drug Combination Platform Trial Design 9.3 Adaptive Platform Design with the Survival Endpoint 9.3.1 Adaptive Phase I/II Platform Design for the Immunotherapy Drugs 9.3.2 Adaptive Phase II Platform Design with the Survival Endpoint 9.3.3 A Rolling-Arms Platform Design 9.3.4 A Bayesian Platform Trial Design with Borrowing from Historical Control Data 9.4 More Discussions on Adding New Arms to Ongoing Clinical Trials 9.4.1 Randomization Procedures and Interim Analyses When Adding New Treatment Arms 9.4.2 Impact on Error Rates of Multi-arm One-Stage Platform Trials When Adding New Treatment Arms 9.4.3 Statistical Considerations of Phase III Platform Trials from a Single Institutional Perspective 9.4.4 Planning a 2+2-Experimental Arm Trial that Controls the PWER 9.5 Summary References