BIOTECHNOLOGY OPERATIONS

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1 BIOTECHNOLOGY OPERATIONS Principles and Practices Michael J. Roy TECHNISCHE INFORMATION SBIBLIOTHEK UNIVERSITATSBIBLIOTHEK HANNOVER CRC Press TaylorStFrancis Croup Boca Raton London New York CRC Press Is an Imprint of the Taylor & Francis an Croup, Informs business

2 Contents Preface Acknowledgments The Author xi xiii xv 1. Introduction to Biotechnology Operations: Planning for Success 1 Biotechnology Operations 1 Marketing, Financial and Business Considerations for Development 4 Product Development Planning 7 Rationale for Product Development Planning 7 Targeted Product Profile 10 Product Development Plan 16 Clinical Development Planning 18 Project Management - Planning 19 Regulatory Planning 20 Nonclinical Planning 22 Biomanufacturing Planning 23 Quality Control Planning 24 Quality Systems and Quality Assurance Planning 26 Additional Elements of Product Planning 26 Summary of Planning for Success Project Management 29 Project Management in Biotechnology 29 Background of Project Management 31 Project Management in Biotechnology 32 Project Management Environment 33 Project Objectives and Schedules 35 Sociotechnical Considerations 36 Participants in Project Management 37 Project Management in Biotechnology Operations 40 Establishing Project Management 40 Work Breakdown Structure 42 Forming a Project Team and Hands-On Project Management 42 Communication and Feedback 47 Team Dynamics 47 Project Risk Assessment and Management 50 Metrics and Tracking Progress 51

3 iv Contents Resources: Planning and Usage 52 Human Factors in Project Management 53 Project Completion 55 Project Management with Contracts and Collaborations 56 Tools for Effective Project Management 57 Summary of Project Management in Biotechnology Development Regulatory Affairs 61 The U.S. Food and Drug Administration: Law and Regulations for Biopharmaceuticals 61 Historical Basis for FDA Regulation 61 Regulatory Organization of FDA 63 Food and Drug Law, Regulation and Guidance 66 FDA-Regulated Products 67 Biologies 67 Drugs 70 Medical Devices 72 Combination Products 72 Other Classes of Biotechnology Products and Their Review at the FDA 73 Products for Veterinary Use 73 Cosmetics, Food, Dietary Supplements, Homeopathic or Nutritional Products 73 U.S. FDA Regulatory Information and Resources: Regulatory Intelligence 75 Regulatory Operations for FDA Applications 78 Regulatory Planning and the Regulatory Environment 78 Risk versus Benefit 78 Applications Seeking FDA Investigational Use or Marketing Approval 80 Investigational Use Applications: Investigational New Drug (IND) Application 81 Marketing Applications: Biologies License Application (BLA) and New Drug Application (NDA) 86 Medical Device Applications: 510(k) and Premarket Approval (PMA) 89 Special Documents, Pathways or Exemptions 91 Generic Drugs and Biosimiliar or Follow-On Biologies 93 Other Regulatory Activities 94 Public Meetings and Advisory Committees 94 Postmarketing Requirements and Activities 96 Advertising and Promotion 97 Summary on Regulatory Affairs Activities in Biotechnology Operations 98

4 Contents v 4. Regulatory Compliance 99 Regulatory Compliance 99 Quality Systems to Meet Regulatory Compliance 99 Compliance and Quality Systems 99 cgmps: Current Good Manufacturing Practices for Manufacture and Quality Control 100 cglps: Current Good Laboratory Practices for Nonclinical Lab Studies 100 cgcps: Current Good Clinical Practices for Clinical Studies 102 Compliance for Biopharmaceuticals: Other Regulations of Importance 104 Compliance for Import of Biopharmaceuticals into the United States 104 Compliance for Medical Devices 105 Inspection and Enforcement 105 Inspections 106 Enforcement Actions 108 Product Liability 110 Compliance with Non-FDA Regulations: International, National, State and Local 110 International and Foreign National Regulatory Authorities for Medical Biotechnology Products 110 Transporting Infectious or Otherwise Hazardous Materials 114 Importing, Possessing or Transferring Controlled Biotechnology Materials 116 Public Health Security and Bioterrorism Preparedness and Response Act of Importation or Exportation of Biotechnology Products for the Purpose of Treatment of Diseases in Man 119 Occupational Health and Safety 120 Environmental Regulations in Biotechnology 121 Genetically Modified Organisms or Molecules 122 U.S. Regulatory Agencies Unified Biotechnology Web Site 124 International Diligence in Biotechnology Operations 124 Summary of Non-FDA Compliance Quality Systems 127 Overview of Quality in Biotechnology 127 History: Evolution of Quality Concepts and Practices 128 Quality Systems Approach to Product Development 130 Planning a Quality System 132 Defining Objectives and Ensuring Management Support 132 The Quality Manual 133 The Quality Plan 134

5 vi Contents Hallmarks of Quality: Fundamental Criteria for Building Effective Quality Systems 136 Management Responsibility 137 Defined Quality System 137 Quality by Design and Design Control 138 Quality by Design 138 Design Control 139 Design Change 143 Contractor, Vendor and Consultant Control 143 Product Identification and Traceability 146 Process Control 146 Environmental Controls 147 Inspection or Testing (Quality Control) 147 Release of Material, Service or Product 148 Change Control and Corrective or Preventive Actions 149 Packaging and Labeling 150 Preservation, Storage and Handling 150 Servicing 151 Customer Concerns and Adverse Event Reports 151 Document Control 151 Training 151 Auditing 152 Quality Assurance Unit 152 Manage the Quality Assurance Function 153 Control Documents and Manage the Documentation System 154 Investigate Situations: Manage and Control Change 156 Ensure Qualified and Trained Staff 156 Perform Audits 157 Initiate a Quality System for a Biotechnology Operation 158 Unique and Effective Approaches to Quality Management 160 Risk-Based Approaches to Quality Systems 160 Total Quality Management (TQM) 160 Six Sigma 161 Quality Systems for Research 161 Resolving Quality Issues or Problems 162 Summary of Quality Systems Biomanufacture 165 Overview of Biomanufacturing Requirements 165 Design in Biomanufacture 165 Technical Considerations for Biomanufacture 169 Phases and Scale Up: The Biomanufacturing Life Cycle 171 Raw Material Considerations 175

6 Contents vii Compliance and Quality in Biomanufacture: Current Good Manufacturing Practices 176 Biomanufacturing Processes for Biotechnology Products 179 Expression of Recombinant Proteins and Nucleic Acids 179 Production of Recombinant Molecules from Expression Vectors 179 Genes, Vectors and Host Cells 180 Bacterial Cell Expression Systems 182 Yeast Cell Expression Systems 182 Mammalian or Insect Cell Expression Systems 183 Production of Master Cell Banks and Working Cell Banks 184 Biomanufacture of Recombinant Proteins 186 Planning Production of a Recombinant Protein 186 Upstream Process: Production by Bacterial or Yeast Cell Fermentation 187 Upstream Process: Production by Mammalian or Insect Cell Culture 190 Upstream Process: Recovery 191 Downstream Process: Purification 191 In-Process Testing and Analysis of Bulk Substance 199 Production of Bacterial Plasmid DNA 201 Production of Live Recombinant Organisms: Bacteria and Virus 201 Production of Products Composed of Mammalian Somatic Cells or Tissues 204 Production of Cellular Products Derived from Pluripotent (Stem) Cells 204 Production of Biological Molecules by Transgenic Animals or Transgenic Plants 206 Production of Biologically Active Lipids, Glycolipids and Complex Carbohydrates 210 Production of Biologically Active Peptides 212 Production of Combination Products: Biopharmaceutical with a Drug or Medical Device 212 Final Product: Formulation Fill, Finish and Labeling 214 Biomanufacturing Facilities, Utilities and Equipment 218 Facility Design Considerations 218 Facility and Utilities'. A Controlled Environment 219 Operation of Clean Work Areas for Biomanufacture 221 Biomanufacturing Equipment 222 Contract Manufacturing Options 223 Validation of Biomanufacturing Facilities, Utilities, Equipment and Processes 223 Summary of Biomanufacture 226

7 viii Contents 7. Quality Control 227 Quality Control Overview 227 Define Product Attributes 230 Analytical Methods Measure Attributes 231 Traits of Analytical Methods 231 Draft a Certificate of Analysis (Bulk Substance) 232 Select Analytical Methods 234 Develop Specifications Enter Test Results 246 Certificate of Analysis for Drug Product 246 In-Process Testing 249 Analytical Methods 250 Additional Analytical Tools and Concepts 256 Quality Control of Cell Banks 258 Samples and Sampling 259 Analytical Controls and Reference Standards 260 Test Failures, Out-of-Specification Results and Retesting 261 Testing for Product Stability 262 Quality Control Testing of Raw Materials 266 Quality Control and the Manufacturing Environment 269 Qualification, Validation and Verification of Analytical Methods 269 Assay Validation 270 Application of Statistics in Assay Performance and Validation 274 Summary of Quality Control Nonclinical Studies 277 Nonclinical Studies and Risk Assessment 277 Biopharmaceutical Delivery, Pharmacokinetics and Pharmacodynamics 279 Product Delivery to the Body 279 Absorption, Distribution, Elimination and Metabolism (ADME) 280 Absorption 281 Distribution 282 Metabolism and Biotransformation 283 Excretion 283 Pharmacokinetics and Pharmacodynamics 283 Application of Pharmacokinetics and Pharmacodynamics in Biopharmaceutical Development 289 Safety Assessment of Biopharmaceuticals 291 Toxicology 291 Design of a Safety Assessment Program 292 In Vitro Screens: Surrogate Measures of Toxicity 295 In Vivo Safety Testing of Biopharmaceuticals 297 Animal Model Development

8 Contents ix Test Product Formulations, Routes of Delivery and Dosing Designs 299 Protocols and Performance of Biopharmaceutical Safety Studies in Animals 301 Elements of a Nonclinical Study Design 302 Acute Toxicity Testing 305 Subchronic and Chronic Toxicity Testing 306 Reproductive, Developmental and Teratogenicity Toxicity Testing 308 Carcinogenicity Testing 310 Immunotoxicology 310 Genetic Toxicology 312 Tissue Binding or Local Tissue Tolerance 313 Quality of Nonclinical Studies: Current Good Laboratory Practices 314 Summary of Nonclinical Studies Clinical Trials 317 Introduction to Clinical Trials 317 Background of Clinical Research 319 Introduction 319 Historical Information on Clinical Trials 320 Organization of Clinical Research 321 Phases of Clinical Trials 321 The Science of Clinical Research 322 Quality in Clinical Research and Current Good Clinical Practices 322 Clinical Development Planning 323 Infrastructure for a Clinical Trial: Individuals, Documents and Investigational Product 323 Design of Clinical Trials and the Clinical Protocol 324 Human Subjects, Patients and Volunteers 332 The Sponsor 332 The Principal Investigator and His/Her Study Staff 335 Institutional Review Boards (IRB): Process of Informed Consent (IC) and IC Form 336 Investigational Product 339 Collection of Clinical Data: Case Report Forms and the Patient Diary 339 Clinical Testing Laboratories 340 Reporting Results of Clinical Trials: Clinical Summary Reports 341 Clinical Trial Operations 341 Activities Leading to a Clinical Trial 342 Phase 1 Clinical Trial: First-Time-in-Man 344

9 X Contents Clinical Pharmacology Studies of Biopharmaceuticals in Man 345 Phase 2 Clinical Trial: Proof-of-Concept 348 Phase 3 Clinical Trial: Therapeutic Confirmatory 349 Phase 4 Clinical Study and REMS 351 Clinical Trials for New Populations or Indications 351 Global Clinical Trials 352 Quality Systems for Clinical Trials: Current Good Clinical Practices 353 Quality and cgcp in Clinical Trial Operations 353 Integrity of Clinical Study Data and Documents 355 Monitoring and Auditing Clinical Trials 356 Ethical Behavior and the Well-Being of Clinical Trial Subjects 356 Summary on Clinical Trials 358 Glossary 359 Additional Reading 383 Practical Problems and Questions 387 Index 395

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