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Table of Contents
Intro
Contents
1: History of Drug Development for Children with Cancer
1.1 Introduction
1.2 Initial Progress
1.3 Rise of the Cancer Cooperative Groups
1.4 Impact of Regulation to Improve Safety and Efficacy Federal Laws Providing a Regulatory Framework for Drug Development in Children
1.5 Indications
1.6 Summary
References
2: Targeted Small Molecule Drug Discovery
2.1 Introduction
2.2 Stage Gating
2.3 Phenotypic Drug Discovery
2.4 Target-Based Drug Discovery: Considerations for Target Selection/Identification
2.5 Target-Based Drug Discovery: Identifying Chemical Starting Points
2.6 Hit-to-Lead
2.7 Lead Optimization
2.8 Candidate Nomination
References
3: An FDA Oncology Perspective of Juvenile Toxicity Studies to Support Pediatric Drug Development
3.1 Introduction
3.2 Should a JAS Be Considered?
3.2.1 ICH S11: Nonclinical Safety Testing in Support of Development of Pediatric Medicines
3.2.2 ICH S9: Nonclinical Evaluation for Anticancer Pharmaceuticals Questions and Answers
3.3 Dinutuximab and Naxitamab
3.4 TRK Inhibitors
3.5 Conclusion
References
4: Design and Statistical Considerations for Early Phase Clinical Trials in Pediatric Oncology
4.1 Introduction
4.2 Rule-Based Designs
4.2.1 Traditional 3 + 3 Design
4.2.2 Accelerated Titration Design
4.2.3 Rolling Six Design
4.3 Model-Based Designs
4.3.1 Continual Reassessment Method (CRM)
4.3.2 EWOC and BLRM
4.3.3 TITE-CRM and TITE-EWOC
4.4 Model-Assisted Designs
4.4.1 Modified Toxicity Probability Interval (mTPI) Design
4.4.2 Keyboard Design
4.4.3 Bayesian Optimal Interval (BOIN) Design
4.5 New Designs
4.5.1 Modified 4 + 4 Design
4.6 Conclusions
References
5: Exploratory Clinical Development: From First in Humans to Phase 3 Ready
5.1 Introduction
5.2 The Therapeutic Hypothesis
5.3 Dose and Schedule Determination
5.4 Clinical Proof of Concept (PoC)
5.5 Putting It All Together: Combined Phase 1-2 Studies
5.6 A Role for Pediatric Cancer in Exploratory Drug Development
References
6: Gene and Cell Therapy: How to Build a BioDrug
6.1 Introduction
6.2 BioDrug ToolKit: Cells
6.2.1 Hematopoietic Stem Cells (HSCs)
6.2.2 T Lymphocytes
6.2.3 Natural Killer (NK) Cells
Contents
1: History of Drug Development for Children with Cancer
1.1 Introduction
1.2 Initial Progress
1.3 Rise of the Cancer Cooperative Groups
1.4 Impact of Regulation to Improve Safety and Efficacy Federal Laws Providing a Regulatory Framework for Drug Development in Children
1.5 Indications
1.6 Summary
References
2: Targeted Small Molecule Drug Discovery
2.1 Introduction
2.2 Stage Gating
2.3 Phenotypic Drug Discovery
2.4 Target-Based Drug Discovery: Considerations for Target Selection/Identification
2.5 Target-Based Drug Discovery: Identifying Chemical Starting Points
2.6 Hit-to-Lead
2.7 Lead Optimization
2.8 Candidate Nomination
References
3: An FDA Oncology Perspective of Juvenile Toxicity Studies to Support Pediatric Drug Development
3.1 Introduction
3.2 Should a JAS Be Considered?
3.2.1 ICH S11: Nonclinical Safety Testing in Support of Development of Pediatric Medicines
3.2.2 ICH S9: Nonclinical Evaluation for Anticancer Pharmaceuticals Questions and Answers
3.3 Dinutuximab and Naxitamab
3.4 TRK Inhibitors
3.5 Conclusion
References
4: Design and Statistical Considerations for Early Phase Clinical Trials in Pediatric Oncology
4.1 Introduction
4.2 Rule-Based Designs
4.2.1 Traditional 3 + 3 Design
4.2.2 Accelerated Titration Design
4.2.3 Rolling Six Design
4.3 Model-Based Designs
4.3.1 Continual Reassessment Method (CRM)
4.3.2 EWOC and BLRM
4.3.3 TITE-CRM and TITE-EWOC
4.4 Model-Assisted Designs
4.4.1 Modified Toxicity Probability Interval (mTPI) Design
4.4.2 Keyboard Design
4.4.3 Bayesian Optimal Interval (BOIN) Design
4.5 New Designs
4.5.1 Modified 4 + 4 Design
4.6 Conclusions
References
5: Exploratory Clinical Development: From First in Humans to Phase 3 Ready
5.1 Introduction
5.2 The Therapeutic Hypothesis
5.3 Dose and Schedule Determination
5.4 Clinical Proof of Concept (PoC)
5.5 Putting It All Together: Combined Phase 1-2 Studies
5.6 A Role for Pediatric Cancer in Exploratory Drug Development
References
6: Gene and Cell Therapy: How to Build a BioDrug
6.1 Introduction
6.2 BioDrug ToolKit: Cells
6.2.1 Hematopoietic Stem Cells (HSCs)
6.2.2 T Lymphocytes
6.2.3 Natural Killer (NK) Cells