This book provides a comprehensive examination of the newest biopharmaceutical drugs. Among the drugs discussed are ones in the categories of monoclonal antibodies for in-vivo use, cytokines, growth factors, enzymes, immunomodulators, thrombolytics, and immonotherapies including vaccines. Additionally, the volume examines new and emerging technologies, and contains a review of the Human Genome Project.

This volume explores the application of Quality by Design (QbD) to biopharmaceutical drug product development. Twenty-eight comprehensive chapters cover dosage forms, liquid and lyophilized drug products. The introductory chapters of this book define key elements of QbD and examine how these elements are integrated into drug product development. These chapters also discuss lessons learned from the FDA Office of Biotechnology Products pilot program. Following chapters demonstrate how QbD is used for formulation development ranging from screening of formulations to developability assessment to development of lyophilized and liquid formats. The next few chapters study the use of small-scale and surrogate models as well as QbD application to drug product processes such as drug substance freezing and thawing, mixing, sterile filtration, filling, lyophilization, inspection and shipping and handling. Later chapters describe more specialized applications of QbD in the drug product realm. This includes the use of QbD in primary containers, devices and combination product development. The volume also explores QbD applied to vaccine development, automation, mathematical modeling and monitoring, and controlling processes and defining control strategies. It concludes with a discussion on the application of QbD to drug product technology transfer as well as overall regulatory considerations and lifecycle management. Quality by Design for Biopharmaceutical Drug Product Development is an authoritative resource for scientists and researchers interested in expanding their knowledge on QbD principles and uses in creating better drugs.

Biopharmaceutical Processing: Development, Design, and Implementation of Manufacturing Processes covers bioprocessing from cell line development to bulk drug substances. The methods and strategies described are essential learning for every scientist, engineer or manager in the biopharmaceutical and vaccines industry. The integrity of the bioprocess ultimately determines the quality of the product in the biotherapeutics arena, and this book covers every stage including all technologies related to downstream purification and upstream processing fields. Economic considerations are included throughout, with recommendations for lowering costs and improving efficiencies. Designed for quick reference and easy accessibility of facts, calculations and guidelines, this book is an essential tool for industrial scientists and managers in the biopharmaceutical industry. - Offers a comprehensive, go-to reference for daily work decisions - Covers both upstream and downstream processes - Includes case studies that emphasize financial outcomes - Presents summaries, decision grids, graphs and overviews for quick reference

Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials.

A comprehensive introduction to using modeling and simulation programs in drug discovery and development Biopharmaceutical modeling has become integral to the design and development of new drugs. Influencing key aspects of the development process, including drug substance design, formulation design, and toxicological exposure assessment, biopharmaceutical modeling is now seen as the linchpin to a drug's future success. And while there are a number of commercially available software programs for drug modeling, there has not been a single resource guiding pharmaceutical professionals to the actual tools and practices needed to design and test safe drugs. A guide to the basics of modeling and simulation programs, Biopharmaceutics Modeling and Simulations offers pharmaceutical scientists the keys to understanding how they work and are applied in creating drugs with desired medicinal properties. Beginning with a focus on the oral absorption of drugs, the book discusses: The central dogma of oral drug absorption (the interplay of dissolution, solubility, and permeability of a drug), which forms the basis of the biopharmaceutical classification system (BCS) The concept of drug concentration How to simulate key drug absorption processes The physiological and drug property data used for biopharmaceutical modeling Reliable practices for reporting results With over 200 figures and illustrations and a peerless examination of all the key aspects of drug research—including running and interpreting models, validation, and compound and formulation selection—this reference seamlessly brings together the proven practical approaches essential to developing the safe and effective medicines of tomorrow.

The biotechnology/biopharmaceutical sector has tremendously grown which led to the invention of engineered antibodies such as Antibody Drug Conjugates (ADCs), Bispecific T-cell engager (BITES), Dual Variable Domain (DVD) antibodies, and fusion proteins that are currently being used as therapeutic agents for immunology, oncology and other disease conditions. Regulatory agencies have raised the bar for the development and manufacture of antibody-based products, expecting to see the use of Quality by Design (QbD) elements demonstrating an in-depth understanding of product and process based on sound science. Drug delivery systems have become an increasingly important part of the therapy and most biopharmaceuticals for self-administration are being marketed as combination products. A survey of the market indicates that there is a strong need for a new book that will provide “one stop shopping” for the latest information and knowledge of the scientific and engineering advances made over the last few years in the area of biopharmaceutical product development. The new book entitled Development of Biopharmaceutical Drug Device Products is a reference text for scientists and engineers in the biopharmaceutical industry, academia or regulatory agencies. With insightful chapters from experts in the field, this new book reviews first principles, covers recent technological advancements and provides case studies and regulatory strategies relating to the development and manufacture of antibody-based products. It covers topics such as the importance of early preformulation studies during drug discovery to influence molecular selection for development, formulation strategies for new modalities, and the analytical techniques used to characterize them. It also addresses important considerations for later stage development such as the development of robust formulations and processes, including process engineering and modeling of manufacturing unit operations, the design of analytical comparability studies, and characterization of primary containers (pre-filled syringes and vials).Finally, the latter half of the book reviews key considerations to ensure the development and approval of a patient-centered delivery system design. This involves the evolving regulatory framework with perspectives from both the US and EU industry experts, the role of international standards, design control/risk management, human factors and its importance in the product development and regulatory approval process, as well as review of the risk-based approach to bridging between devices used in clinical trials and the to-be-marketed device. Finally, case studies are provided throughout.The typical readership would have biology and/or engineering degrees and would include researchers, scientific leaders, industry specialists and technology developers working in the biopharmaceutical field.

As with all of pharmaceutical production, the regulatory environment for the production of therapeutics has been changing as a direct result of the US FDA-initiated Quality by Design (QbD) guidelines and corresponding activities of the International Committee for Harmonization (ICH). Given the rapid growth in the biopharmaceutical area and the complexity of the molecules, the optimum use of which are still being developed, there is a great need for flexible and proactive teams in order to satisfy the regulatory requirements during process development. Process Analytical Technologies (PAT) applied in biopharmaceutical process development and manufacturing have received significant attention in recent years as an enabler to the QbD paradigm. PAT Applied in Biopharmaceutical Process Development and Manufacturing covers technological advances in measurement sciences, data acquisition, monitoring, and control. Technical leaders present real-life case studies in areas including measuring and monitoring raw materials, cell culture, purification, and cleaning and lyophilization processes via advanced PAT. They also explore how data are collected and analyzed using advanced analytical techniques such as multivariate data analysis, monitoring, and control in real-time. Invaluable for experienced practitioners in PAT in biopharmaceuticals, this book is an excellent reference guide for regulatory officials and a vital training aid for students who need to learn the state of the art in this interdisciplinary and exciting area.