This second edition of Fundamentals of Biologicals Regulation: Vaccines and Biotechnology Medicines serves as an introduction to the international regulatory arena in which biologicals are developed and offers an overview of the processes and insight into the scientific concepts underpinning global regulations. In this new edition the book provides more in-depth coverage of Quality by Design, Combination Products, Quality Target Product Profiles, Risk Management Plans and REMS, and switching from reliance on in vivo to in vitro analytical methods. This new edition also reflects the new developments and processes accelerated by the COVID-19 vaccine experience, including emergency use authorizations and conditional marking authorizations. New pedagogic elements and case studies have been added to improve learning outcomes for students. This book provides multiple levels of readership with guidance on basic concepts, a detailed look at regulatory challenges, and practical insight into how regulators consider regulatory science and regulatory process issues across various regions with emphasis on the European Medicines Agency and the U.S. Food and Drug Administration. Graduate students will benefit from the new pedagogic features. Regulatory professionals will have in Fundamentals of Biologicals Regulation: Vaccines and Biotechnology Medicines a complete guide to navigate through the approval process.
• New chapters on Advanced Therapy Medicinal Products (including cell and gene therapies), Combination Products, and Pharmacovigilance1. Chapter 1. Introduction to the regulatory process for biologicals and to international regulatory convergence - p. 5 2. Chapter 2. Discovery and development - p. 37 3. Chapter 3. Communications and formal meetings with regulators: Focusing on the process before clinical trial authorization (a.k.a. pre-IND) - p. 49 4. Chapter 4. Clinical trial authorization and investigational new drug applications - p. 69 5. Chapter 5. Marketing authorization - p. 87 6. Chapter 6. Alternative regulatory pathways and special programs - p. 107 7. Chapter 7. Variations or changes to an approved application - p. 129 8. Chapter 8. Good “X” Practices - p. 141 9. Chapter 9. Audits and regulatory compliance inspections - p. 157 10. Written assignments exercise - p. 177 11. Assignment Scenarios - p. 181 12. Section II. Regulatory science - p. S2 13. Chapter 10. Preclinical safety and toxicology - p. 187 14. Chapter 11. Preclinical pharmacology, proof-of-principle - p. 217 15. Chapter 12. Institutional biosafety committees and regulation of genetically modified organisms - p. 231 16. Chapter 13. Risk assessments - p. 243 17. Chapter 14. Quality by design and product design, construction, development, manufacture, and process validation - p. 255 18. Chapter 15. Lot release, analytics, and analytical validation - p. 287 19. Chapter 16. Regulatory aspects of clinical trials - p. 309 20. Chapter 17. Pharmacovigilance, safety reporting, and risk minimization - p. 343 21. Chapter 18. Clinical trial ethics, human subjects protections, and the informed consent process - p. 363 22. Chapter 19. Independent ethics committees and institutional review boards - p. 381 23. Section III. Specific product types - p. S3 24. Chapter 20. Biosimilars - p. 393 25. Chapter 21. Cell and gene therapies - p. 413 26. Chapter 22. In vitro diagnostics and biotech medical devices - p. 461 27. Chapter 23. Combination Products - p. 473 28. 23.1 Fundamentals - p. 473 29. 23.2 Some details - p. 494 30. 23.3 Case studies - p. 538 31. 23.4 Conclusion - p. 541 32. Glossary terms & definitions - p. 542 33. Glossary - p. 549 34. Index - p. 579
This second edition of Fundamentals of Biologicals Regulation: Vaccines and Biotechnology Medicines serves as an introduction to the international regulatory arena in which biologicals are developed and offers an overview of the processes and insight into the scientific concepts underpinning global regulations. In this new edition the book provides more in-depth coverage of Quality by Design, Combination Products, Quality Target Product Profiles, Risk Management Plans and REMS, and switching from reliance on in vivo to in vitro analytical methods. This new edition also reflects the new developments and processes accelerated by the COVID-19 vaccine experience, including emergency use authorizations and conditional marking authorizations. New pedagogic elements and case studies have been added to improve learning outcomes for students. This book provides multiple levels of readership with guidance on basic concepts, a detailed look at regulatory challenges, and practical insight into how regulators consider regulatory science and regulatory process issues across various regions with emphasis on the European Medicines Agency and the U.S. Food and Drug Administration. Graduate students will benefit from the new pedagogic features. Regulatory professionals will have in Fundamentals of Biologicals Regulation: Vaccines and Biotechnology Medicines a complete guide to navigate through the approval process.
• New chapters on Advanced Therapy Medicinal Products (including cell and gene therapies), Combination Products, and Pharmacovigilance1. Chapter 1. Introduction to the regulatory process for biologicals and to international regulatory convergence - p. 5 2. Chapter 2. Discovery and development - p. 37 3. Chapter 3. Communications and formal meetings with regulators: Focusing on the process before clinical trial authorization (a.k.a. pre-IND) - p. 49 4. Chapter 4. Clinical trial authorization and investigational new drug applications - p. 69 5. Chapter 5. Marketing authorization - p. 87 6. Chapter 6. Alternative regulatory pathways and special programs - p. 107 7. Chapter 7. Variations or changes to an approved application - p. 129 8. Chapter 8. Good “X” Practices - p. 141 9. Chapter 9. Audits and regulatory compliance inspections - p. 157 10. Written assignments exercise - p. 177 11. Assignment Scenarios - p. 181 12. Section II. Regulatory science - p. S2 13. Chapter 10. Preclinical safety and toxicology - p. 187 14. Chapter 11. Preclinical pharmacology, proof-of-principle - p. 217 15. Chapter 12. Institutional biosafety committees and regulation of genetically modified organisms - p. 231 16. Chapter 13. Risk assessments - p. 243 17. Chapter 14. Quality by design and product design, construction, development, manufacture, and process validation - p. 255 18. Chapter 15. Lot release, analytics, and analytical validation - p. 287 19. Chapter 16. Regulatory aspects of clinical trials - p. 309 20. Chapter 17. Pharmacovigilance, safety reporting, and risk minimization - p. 343 21. Chapter 18. Clinical trial ethics, human subjects protections, and the informed consent process - p. 363 22. Chapter 19. Independent ethics committees and institutional review boards - p. 381 23. Section III. Specific product types - p. S3 24. Chapter 20. Biosimilars - p. 393 25. Chapter 21. Cell and gene therapies - p. 413 26. Chapter 22. In vitro diagnostics and biotech medical devices - p. 461 27. Chapter 23. Combination Products - p. 473 28. 23.1 Fundamentals - p. 473 29. 23.2 Some details - p. 494 30. 23.3 Case studies - p. 538 31. 23.4 Conclusion - p. 541 32. Glossary terms & definitions - p. 542 33. Glossary - p. 549 34. Index - p. 579
