The Challenge Of Cmc Regulatory Compliance For Biopharmaceuticals

Each year for the past three years, there have been about 50 new molecular medicines approved by the United States Food & Drug Administration (FDA), of which approximately 25% were new biopharmaceuticals. Over 200 recombinant proteins, monoclonal antibodies, antibody drug conjugates, fusion proteins, and Fab fragments are now in the marketplace in both the United States of America (USA) and European Union (EU). There are also now over 60 biosimilars available for all major classes of recombinant proteins and monoclonal antibodies. In addition, gene therapies using genetically engineered viruses and genetically engineered cells are now in the marketplace, and continually growing. This degree of change is reflected in the over 400 CMC regulatory compliance references listed in this book that were either issued or updated since the release of the third edition. Deficiencies in biopharmaceutical CMC regulatory compliance rarely result in termination of a product, but in can readily cause months if not years of delay in initiating clinical trials, or advancing clinical development stages, or even market approval. In summary, this book: Updates real-world CMC deficiency examples with current examples; Addresses current FDA and EMA requirements and expectations for CMC regulatory compliance; Now includes CMC regulatory compliance for the new gene-based biopharmaceuticals.

This book highlights the challenges facing quality assurance/quality control (QA/QC) in today's biopharmaceutical environment and presents the strategic importance and value generated by QA/QC for their involvement in control of manufacturing. It will put into perspective the need for a graded approach to QA/QC from early clinical trials through market approval. Since the first edition published in 2004, there have been more than 50 new regulatory guidances released by the Food and Drug Administration (FDA), European Medicines Agency (EMA) and ICH that affect the CMC regulatory compliance of biopharmaceuticals; also the application of biosimilars has been developed in Europe and is under development in the USA. The revised update will be broadened to include not only biopharmaceuticals (biotech drugs) but also other biologics (vaccines, cell therapy, plasma-derived proteins, etc.)

The International Conference of Harmonization (ICH) has worked on har- nizing the stability regulations in the US, Europe, and Japan since the early 1990s. Even though the Stability Guidelines Q1A (R2) was issued over a decade ago, issues surrounding this arena continue to surface as the principles described in the guideline are applied to different technical concentrations. As a result, the stability community has continued to discuss concerns and find ways of harmonizing regulatory requirements, streamlining practices, improving processes in order to bring safe and effective medical supplies to the patients around the world. In 2007, the American Association of Pharmaceutical Scientists (AAPS) Stability Focus Group organized two workshops – the Stability Workshop and the Degradation Mechanism Workshop. These meetings attracted many industry scientists as well as representatives from several regulatory agencies in the world to discuss important topics related to pharmaceutical stability practices. Recognizing the importance of documenting these discussions and with the permission of AAPS, I have worked with speakers to assemble a collection of 30 articles from presentations given at these two meetings, mainly the Stability Workshop. I trust that this book will be beneficial to all of you in providing guidance and up-to-date information for building quality stability programs. v Freedom of our mind is Mother of all inventions.

Biocontamination Control for Pharmaceuticals and Healthcare outlines a biocontamination strategy that tracks bio-burden control and reduction at each transition in classified areas of a facility. The first edition of the book covered many of the aspects of the strategy, but the new official guidance signals that a roadmap is required to fully comply with its requirements. Completely updated with the newest version of the EU-GPM (EN17141) the new edition expands the coverage of quality risk management and new complete examples to help professionals bridge the gap between regulation and implementation. Biocontamination Control for Pharmaceuticals and Healthcare offers professionals in pharma quality control and related areas guidance on building a complete biocontamination strategy. Includes the most current regulations Contains three new chapters, including Application of Quality Risk Management and its Application in Biocontamination Control, Designing an Environmental Monitoring Programme, and Synthesis: An Anatomy of a Contamination Control Strategy Offers practical guidance on building a complete biocontamination strategy

Recently, there has been a fundamental shift in the global health and wellness industry from disease treatment to preventing chronic diseases. The use of nutraceuticals and functional foods in prevention efforts could lead to a decreased dependency on drugs. The pharmaceutical industry recognizes this shift; however, serious concerns have arisen regarding the claimed efficacy, quality, and safety of products used as medical foods. This book examines the consumer and industry mindshift, including the scientific evidence of these foods as effective adjuncts to pharmacotherapy during all stages of treatment of various diseases, thus indicating that pharmaceuticals and nutraceuticals can and should coexist. It details quality, safety, and efficacy of foods, drugs, and nutrients; marketing and product positioning; regulatory perspectives; biomarkers and metabolites; probiotics; food/drug interactions; and future industry trends. In addition, food bioactives represent diet-based molecules that perform physiological roles related to disease prevention and treatment. As such, a considerable overlap exists between food bioactives and drugs—this book presents the case for comparing and contrasting foods versus drugs in several models of health and disease.

This book covers the principles, concepts, techniques and practical tools of financial accounting in supporting overall business decisions. Using a comprehensive theoretical framework, this book is enhanced by practical examples, exercises, chapter summaries and complex cases in each of the eleven chapters. With the International Financial Reporting Standards (IFRS) and their specific Conceptual Framework as the baseline approach, this book also highlights Romanian regulation and practice specifically. Despite the globalisation efforts, this book demonstrates that there are still discrepancies and cultural challenges and will be of interest to students and academics of financial accounting.

A guide to make your Enterprise Risk Management program effective and to avoid the most common mis-steps. Examines the four most common mistakes in existing Enterprise Risk Assessment programs and provides a framework to avoid them and create an effective Enterprise Risk Management program (eERM).

Presents the many recent innovations and advancements in the field of biotechnological processes This book tackles the challenges and potential of biotechnological processes for the production of new industrial ingredients, bioactive compounds, biopolymers, energy sources, and compounds with commercial/industrial and economic interest by performing an interface between the developments achieved in the recent worldwide research and its many challenges to the upscale process until the adoption of commercial as well as industrial scale. Bioprocessing for Biomolecules Production examines the current status of the use and limitation of biotechnology in different industrial sectors, prospects for development combined with advances in technology and investment, and intellectual and technical production around worldwide research. It also covers new regulatory bodies, laws and regulations, and more. Chapters look at biological and biotechnological processes in the food, pharmaceutical, and biofuel industries; research and production of microbial PUFAs; organic acids and their potential for industry; second and third generation biofuels; the fermentative production of beta-glucan; and extremophiles for hydrolytic enzymes productions. The book also looks at bioethanol production from fruit and vegetable wastes; bioprocessing of cassava stem to bioethanol using soaking in aqueous ammonia pretreatment; bioprospecting of microbes for bio-hydrogen production; and more. Provides up to date information about the advancements made on the production of important biotechnological ingredients Complete visualization of the general developments of world research around diverse products and ingredients of technological, economic, commercial and social importance Investigates the use and recovery of agro-industrial wastes in biotechnological processes Includes the latest updates from regulatory bodies for commercialization feasibility Offering new products and techniques for the industrial development and diversification of commercial products, Bioprocessing for Biomolecules Production is an important book for graduate students, professionals, and researchers involved in food technology, biotechnology; microbiology, bioengineering, biochemistry, and enzymology.

This handbook has been written for the students of pharmacy and pharmaceutical industry. It gives pocket guide of good manufacturing practices in pharmaceutical industry. The purpose of this handbook is to provide easily accessible knowledge about the pharmaceutical industry and to assist individuals to know about pharmaceutical world.This handbook has also incorporated the current trends and expectations of the evolving pharmaceutical industry and regulatory oversight. In current pharmaceutical world we need a fast and reliable source of techniques to implement the system and resolve problem. This handbook gives pathway for us to take right decision. Nothing comes in a one box for us. Changes happen with or without us. The higher we go in the organization, the more complex our challenges become. This book gives overall view of quality management system We hope this handbook can contribute to assemble lots of related materials and package them in one place for easy reference and access. I encourage you to read, enjoy, study, and learn from this book, and go forth and empower your teams to lead you and your organization to world class results

This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.