Fundamentals of Biologicals Regulation

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.

Dr. Rebecca Sheets is currently the Vice President for the International Alliance of Biologicals Standardization, North American affiliate. She is the former Vaccine Scientific and Regulatory Specialist at the National Institute of Allergy and Infectious Diseases. Dr. Sheets served 9 years as a Scientific Reviewer in the Viral Vaccines branch of the Division of Vaccines and Related Products Applications at the Center for Biologics Evaluation and Research at the FDA. From 2006-2014, she served as Co-Chair of the World Health Organization's Study Group on Cell Substrates and as Chair of the Adventitious Agents Sub-committee, as well as authoring several WHO guidelines and publications over the past two decades. She also taught for a decade at the Catholic University of America. Before retirement, she was the Principal Consultant at Grimalkin Partners from 2013-2021.

About the authors Foreword Preface How to obtain documents from ICH and USSECTION I: Regulatory process1 Introduction to the regulatory process for biologicals and to international regulatory convergence 2 Discovery and development 3 Communications and formal meetings with regulators 4 Clinical trial authorization and investigational new drug applications 5 Marketing authorization 6 Alternative regulatory pathways and special programs 7 Variations or changes to an approved application 8 Good "X" Practices 9 Audits and regulatory compliance inspections Written assignments exercise Assignment Scenarios SECTION II: Regulatory science10 Preclinical safety and toxicology 11 Preclinical pharmacology, proof-of-principle 12 Institutional biosafety committees and regulation of genetically modified organisms 13 Risk assessments 14 Quality by design and product design, construction, development, manufacture, and process validation 15 Lot release, analytics, and analytical validation 16 Regulatory aspects of clinical trials 17 Pharmacovigilance, safety reporting, and risk minimization 18 Clinical trial ethics, human subjects protections, and the informed consent process 19 Independent ethics committees and institutional review boards SECTION III: Specific product types 20 Biosimilars 21 Cell and gene therapies 22 In vitro diagnostics and biotech medical devices 23 Combination products Glossary Index