Development and Approval of Combination Products

A step by step, integrated approach for successful, FDA approved combination drug products Using a proven integrated approach to combination drug development, this book guides you step by step through all the preclinical, clinical, and manufacturing stages.

Informationen zum Autor Evan B. Siegel, PhD, is President and CEO of Ground Zero Pharmaceuticals, Inc., and an Adjunct Professor at the University of Queensland in Australia. Dr. Siegel has held positions as a Toxicology Reviewer at the U.S. Food and Drug Administration and Supervising Toxicologist in the California Department of Health Services. He has also served in regulatory affairs and executive positions in CROs, the pharmaceutical industry, and trade associations. In addition, Dr. Siegel was an editor of Regulatory Affairs Focus from 1999 to 2001. Klappentext A step-by-step, integrated approach for successful, FDA-approved combination drug productsUsing a proven integrated approach to combination drug development, this book guides you step by step through all the preclinical, clinical, and manufacturing stages. Written from an FDA regulatory perspective, the book not only enables you to bring a successful combination drug product to market, it also sets forth the most efficient and effective path to FDA approval.The book begins with an introductory chapter presenting definitions and basic regulatory principles of combination products. Next, it reviews manufacturing and controls, preclinical testing models, pharmacology, clinical testing, regulatory submissions, FDA reviews, and approvals. Among the key topics examined are:*The pharmacology, safety pharmacology, and toxicology supporting human clinical trials of combination products*Approaches to clinical trial protocol design and execution*Chemical, physicochemical, and analytical aspects of manufacturing controls and validation that lead to stable components for combination products*Key sponsor/FDA meetings and negotiations essential for approval and commercializationCase studies involving such actual combination products as Mylotarg, Herceptin, and HercepTest help you better understand how to implement the author's practical guidelines. References at the end of each chapter enable you to find more information on any stage of the development, manufacturing and approval processes.This book is ideal for researchers, regulators, academics, project managers, and executives involved in the complex process of combination product development. Not only does itoffer a comprehensive guide to the technical aspects of the field, it also integrates all ofthese technical aspects into a unified, effective approach to help ensure a successful, approved product. Zusammenfassung A step-by-step, integrated approach for successful, FDA-approved combination drug products Using a proven integrated approach to combination drug development, this book guides you step by step through all the preclinical, clinical, and manufacturing stages. Inhaltsverzeichnis Preface xi Acknowledgments xiii Contributors xv 1 Overview of Combination Products Development and Regulatory Review 1 Evan B. Siegel 2 Detailed Regulatory Approaches to Development, Review, and Approval 5 James Barquest 2.1 Introduction 5 2.2 General Background 6 2.2.1 Definitions 6 2.2.2 FDA Organization and Jurisdiction 7 2.2.3 Clinical Investigation and Premarket Review Requirements for Drugs, Biological Products, and Medical Devices 11 2.2.4 FDA Information Resources 15 2.3 Combination Products: Regulatory Background 16 2.3.1 Definition 16 2.3.2 Intercenter Agreements 18 2.3.3 Office of Combination Products 19 2.3.4 Primary Mode of Action 20 2.3.5 Intended Use 30 2.3.6 Strategic Regulatory Considerations 31 2.3.7 The Request for Designation (RFD) Process 34 2.3.8 User Fees 44 2.3.9 FDA Meetings: Successful Regulatory Interactions 50 2.3.10 Current Good Manufacturing Practice for Combination Products 59 2.4 Postmarketing Considerations 67 2.4.1 Adverse Event Report...

Auteur

Evan B. Siegel, PhD, is President and CEO of Ground Zero Pharmaceuticals, Inc., and an Adjunct Professor at the University of Queensland in Australia. Dr. Siegel has held positions as a Toxicology Reviewer at the U.S. Food and Drug Administration and Supervising Toxicologist in the California Department of Health Services. He has also served in regulatory affairs and executive positions in CROs, the pharmaceutical industry, and trade associations. In addition, Dr. Siegel was an editor of Regulatory Affairs Focus from 1999 to 2001.

Texte du rabat

A step-by-step, integrated approach for successful, FDA-approved combination drug products Using a proven integrated approach to combination drug development, this book guides you step by step through all the preclinical, clinical, and manufacturing stages. Written from an FDA regulatory perspective, the book not only enables you to bring a successful combination drug product to market, it also sets forth the most efficient and effective path to FDA approval. The book begins with an introductory chapter presenting definitions and basic regulatory principles of combination products. Next, it reviews manufacturing and controls, preclinical testing models, pharmacology, clinical testing, regulatory submissions, FDA reviews, and approvals. Among the key topics examined are: The pharmacology, safety pharmacology, and toxicology supporting human clinical trials of combination products Approaches to clinical trial protocol design and execution Chemical, physicochemical, and analytical aspects of manufacturing controls and validation that lead to stable components for combination products Key sponsor/FDA meetings and negotiations essential for approval and commercialization Case studies involving such actual combination products as Mylotarg, Herceptin, and HercepTest help you better understand how to implement the author's practical guidelines. References at the end of each chapter enable you to find more information on any stage of the development, manufacturing and approval processes. This book is ideal for researchers, regulators, academics, project managers, and executives involved in the complex process of combination product development. Not only does itoffer a comprehensive guide to the technical aspects of the field, it also integrates all ofthese technical aspects into a unified, effective approach to help ensure a successful, approved product.

Contenu

Preface xi

Acknowledgments xiii

Contributors xv

1 Overview of Combination Products Development and Regulatory Review 1
Evan B. Siegel

2 Detailed Regulatory Approaches to Development, Review, and Approval 5
James Barquest

2.1 Introduction 5

2.2 General Background 6

2.2.1 Definitions 6

2.2.2 FDA Organization and Jurisdiction 7

2.2.3 Clinical Investigation and Premarket Review Requirements for Drugs, Biological Products, and Medical Devices 11

2.2.4 FDA Information Resources 15

2.3 Combination Products: Regulatory Background 16

2.3.1 Definition 16

2.3.2 Intercenter Agreements 18

2.3.3 Office of Combination Products 19

2.3.4 Primary Mode of Action 20

2.3.5 Intended Use 30

2.3.6 Strategic Regulatory Considerations 31

2.3.7 The Request for Designation (RFD) Process 34

2.3.8 User Fees 44

2.3.9 FDA Meetings: Successful Regulatory Interactions 50

2.3.10 Current Good Manufacturing Practice for Combination Products 59

2.4 Postmarketing Considerations 67

2.4.1 Adverse Event Reporting 68

2.4.1.1 Device Malfunction Reporting (21 CFR 803.3(r)(2)(ii), 21 CFR 803.20) 68

2.4.1.2 Five-Day MDR Reporting (21 CFR 803.10(c)(2)(i)) 68

2.4.1.3 Drug and Biological Product "Alert" Reporting (21 CFR 314.80(c)(1) and 600.80(c)(1)) 73

2.4.1.4 Blood-Related Deaths (21 CFR 606.170) 73

2.4.2 Other Compliance Issues 73

References 74

**3 Nonclinical Recommendations for Successful Characterization and Development…