Since the early 2000s, there has been increasing interest within the pharmaceutical industry in the application of Bayesian methods at various stages of the research, development, manufacturing, and health economic evaluation of new health care interventions. In 2010, the first Applied Bayesian Biostatistics conference was held, with the primary objective to stimulate the practical implementation of Bayesian statistics, and to promote the added-value for accelerating the discovery and the delivery of new cures to patients. This book is a synthesis of the conferences and debates, providing an overview of Bayesian methods applied to nearly all stages of research and development, from early discovery to portfolio management. It highlights the value associated with sharing a vision with the regulatory authorities, academia, and pharmaceutical industry, with a view to setting up a common strategy for the appropriate use of Bayesian statistics for the benefit of patients. The book covers: Theory, methods, applications, and computing Bayesian biostatistics for clinical innovative designs Adding value with Real World Evidence Opportunities for rare, orphan diseases, and pediatric development Applied Bayesian biostatistics in manufacturing Decision making and Portfolio management Regulatory perspective and public health policies Statisticians and data scientists involved in the research, development, and approval of new cures will be inspired by the possible applications of Bayesian methods covered in the book. The methods, applications, and computational guidance will enable the reader to apply Bayesian methods in their own pharmaceutical research.

This volume focuses on the potential use of probiotics in treating metabolic disorders such as diabetes mellitus, metabolic acidosis, and gut dysbiosis. Chapters draw an association between gut microbiota and its diversity with metabolic diseases like diabetes, obesity, related liver and gut disorders; gut-brain axis; increased inflammation, and a compromised immune system resulting from these manifestations and scope of intervention with probiotics. Special attention is laid on describing the mechanisms of action of such beneficial effects of probiotic administration. The ability of probiotics to decrease metabolic endotoxemia by restoring the disrupted intestinal mucosal barrier is also included. The volume is a comprehensive compilation describing the scope and application of probiotic and prebiotic therapy in treating metabolic disorders. Readers will discover how probiotics are not just confined to the microbiology industry but are showing promising results in the medical and pharmaceutical sectors.

Drug Discovery Targeting Drug-Resistant Bacteria explores the status and possible future of developments in fighting drug-resistant bacteria. The book covers the majority of microbial diseases and the drugs targeting them. In addition, it discusses the potential targeting strategies and innovative approaches to address drug resistance. It brings together academic and industrial experts working on discovering and developing drugs targeting drug-resistant (DR) bacterial pathogens. New drugs active against drug-resistant pathogens are discussed, along with new strategies being used to discover molecules acting via new modes of action. In addition, alternative therapies such as peptides and phages are included. Pharmaceutical scientists, microbiologists, medical professionals, pathologists, researchers in the field of drug discovery, infectious diseases and microbial drug discovery both in academia and in industrial settings will find this book helpful.

Generic Drug Product Development: Specialty Dosage Forms explores the issues related to providing evidence of pharmaceutical equivalence and bioequivalence for specialty drug products. It describes various scientific approaches and regulatory requirements for manufacturers who need to demonstrate the therapeutic equivalence of generic specialty drug products to brand name alternatives. The contributors discuss measurement of drug product quality and performance, as well as the regulatory and scientific requirements of topical, nasal and inhalation, and transdermal drug delivery products, along with generic biologics and modified release parenteral drug products. The book is essential reading for specialists and researchers in pharmaceutical drug development, regulation, manufacturing, and others in the pharmaceutical sciences.

Handbook of Innovations in CNS Regenerative Medicine provides a comprehensive overview of the CNS regenerative medicine field. The book describes the basic biology and anatomy of the CNS and how injury and disease affect its balance and the limitations of the present therapies used in the clinics. It also introduces recent trends in different fields of CNS regenerative medicine, including cell transplantation, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies. Finally, the book presents successful cases of translation of basic research to first-in-human trials and the steps needed to follow this path. Areas such as cell transplantation approaches, bio and neuro-engineering, molecular/pharmacotherapy therapies and enabling technologies are key in regenerative medicine are covered in the book, along with regulatory and ethical issues.

Pharmaceutical Applications of Dendrimers explores the applications of dendrimers in the solubilization of hydrophobic active ingredients, drug delivery, gene delivery, imaging, diagnosis and photodynamic therapy. The book discusses the diagnostic applications of dendrimers, including their use as MRI contrast agents and in the imaging of diseased areas. In addition, the anti-inflammatory, antimicrobial and antiviral properties of PPI and PAMAM are also covered, along with a discussion on photosensitizers, such as rose Bengal and protoporphyrin IX that have been delivered using PAMAM and PPI dendrimers for the treatment of cancer. This book is an important research reference for those who want to learn more about the development of dendrimer-based solutions for drug delivery.

Contents:
Part 1 Identifying the issues: control of microbial contamination in cosmetics, toiletries and non-sterile pharmaceuticals; hazards associated with the microbiological contamination of cosmetics, toiletries and pharmaceuticals. Part 2 Control in manufacture: microbiological control of raw materials; control of microbial contamination during manufacture. Part 3 Control through preservation: natural and physical preservative systems; the effect of container materials and multiple phase formulation components on the activity of antimicrobial agents; formulation aspects of the preservation of skin and hair products; preservation of solid oral dosage forms; development of preservative systems; microbial resistance to preservative systems; safety evaluation of preservatives. Part 4 Microbiological control - methods and standards: preservative efficacy testing of pharmaceuticals, cosmetics and toiletries and its limitations; challenge tests and their predictive ability; new methodology for microbial quality assurance; microbial contamination of manufactured products - official and unofficial limits.

D ring the past ten ears or so, there has been an increasing n mber of p blications describing ario s aspects of oligon cleotide chemistr and the application of oligon cleotides in basic, as ell as applied science. More recentl , ario s oligon cleotide conj gates nthesi ed that are c rrentl being st died for their ha e been s potential diagnostic and therape tic applications. With the introd- tion of ne oligon cleotide conj gates, anal tical methods for the anal sis of these oligon cleotides are also being de eloped and op- mi ed. The present ol me, Protocols for Oligon cleotide Con- gates: S nthesis and Anal tical Techniq es, is designed to bring readers p-to-date on the protocols for the s nthesis of ario s oli- gates and on all the important techniq es for the n cleotide conj nthetic oligon cleotides and their analogs. The s- anal sis of s thetic chemistrof man of these oligon cleotides and their ario s analogs has been co ered in a companion ol me entitledProtocols for Oligon cleotides and Analogs: S nthesis and Properties. The first chapter ofProtocols for Oligon cleotide Conj gates- scribes the protecting gro ps in oligon cleotide s nthesis.

This third volume of the second edition offers information on specialized products such as emulsions, liposomes, polymers and polymeric pharmaceutical excipients. It explains the requirements for conducting clinical research and obtaining marketing approval for new drug products

Dynamic Treatment Regimes: Statistical Methods for Precision Medicine provides a comprehensive introduction to statistical methodology for the evaluation and discovery of dynamic treatment regimes from data. Researchers and graduate students in statistics, data science, and related quantitative disciplines with a background in probability and statistical inference and popular statistical modeling techniques will be prepared for further study of this rapidly evolving field. A dynamic treatment regime is a set of sequential decision rules, each corresponding to a key decision point in a disease or disorder process, where each rule takes as input patient information and returns the treatment option he or she should receive. Thus, a treatment regime formalizes how a clinician synthesizes patient information and selects treatments in practice. Treatment regimes are of obvious relevance to precision medicine, which involves tailoring treatment selection to patient characteristics in an evidence-based way. Of critical importance to precision medicine is estimation of an optimal treatment regime, one that, if used to select treatments for the patient population, would lead to the most beneficial outcome on average. Key methods for estimation of an optimal treatment regime from data are motivated and described in detail. A dedicated companion website presents full accounts of application of the methods using a comprehensive R package developed by the authors. The authors' website www.dtr-book.com includes updates, corrections, new papers, and links to useful websites.

With the continued advancement of better-quality control and patient outcome reporting systems, changes in the development, control, and regulation of all pharmaceutical delivery systems including transdermal and topical products have been happening on a continuous basis. In light of various quality issues that have been reported by patients and practitioners resulting in the recall or removal of products from the market, both the pharmaceutical industries and regulatory agencies have been adopting new measures to address these issues. With chapters written by experts in this field, this book takes a 21st century multidisciplinary and cross-functional look at these dosage forms to improve the development, design, manufacturing, quality, clinical performance, safety, and regulation of these products. This book offers a wealth of up-to-date information organized in a logical sequence corresponding to various stages of research, development, and commercialization of dermal drug delivery products. The authors have been carefully selected from different sectors of pharmaceutical science for their expertise in their selected areas to present objectively a balanced view of the current state of these products development and commercialization via regulatory approval. Their insights will provide useful information to others to ensure the successful development of the next generation dermal drug products. Key Features: Presents current advancements including new technologies of transdermal and topical dosage forms. Presents challenges in the development of the new generation of transdermal and topical dosage forms. Introduces new technologies and QbD (quality by design) aspects of manufacturing and control strategies. Includes new perspectives on pre-clinical and clinical development, regulatory considerations, safety and quality. Discusses regulatory challenges, gaps, and future considerations for dermal drug delivery systems.

Handbook of Methods for Designing, Monitoring, and Analyzing Dose-Finding Trials gives a thorough presentation of state-of-the-art methods for early phase clinical trials. The methodology of clinical trials has advanced greatly over the last 20 years and, arguably, nowhere greater than that of early phase studies. The need to accelerate drug development in a rapidly evolving context of targeted therapies, immunotherapy, combination treatments and complex group structures has provided the stimulus to these advances. Typically, we deal with very small samples, sequential methods that need to be efficient, while, at the same time adhering to ethical principles due to the involvement of human subjects. Statistical inference is difficult since the standard techniques of maximum likelihood do not usually apply as a result of model misspecification and parameter estimates lying on the boundary of the parameter space. Bayesian methods play an important part in overcoming these difficulties, but nonetheless, require special consideration in this particular context. The purpose of this handbook is to provide an expanded summary of the field as it stands and also, through discussion, provide insights into the thinking of leaders in the field as to the potential developments of the years ahead. With this goal in mind we present: An introduction to the field for graduate students and novices A basis for more established researchers from which to build A collection of material for an advanced course in early phase clinical trials A comprehensive guide to available methodology for practicing statisticians on the design and analysis of dose-finding experiments An extensive guide for the multiple comparison and modeling (MCP-Mod) dose-finding approach, adaptive two-stage designs for dose finding, as well as dose-time-response models and multiple testing in the context of confirmatory dose-finding studies. John O'Quigley is a professor of mathematics and research director at the French National Institute for Health and Medical Research based at the Faculty of Mathematics, University Pierre and Marie Curie in Paris, France. He is author of Proportional Hazards Regression and has published extensively in the field of dose finding. Alexia Iasonos is an associate attending biostatistician at the Memorial Sloan Kettering Cancer Center in New York. She has over one hundred publications in the leading statistical and clinical journals on the methodology and design of early phase clinical trials. Dr. Iasonos has wide experience in the actual implementation of model based early phase trials and has given courses in scientific meetings internationally. Bjoern Bornkamp is a statistical methodologist at Novartis in Basel, Switzerland, researching and implementing dose-finding designs in Phase II clinical trials. He is one of the co-developers of the MCP-Mod methodology for dose finding and main author of the DoseFinding R package. He has published numerous papers on dose finding, nonlinear models and Bayesian statistics, and in 2013 won the Royal Statistical Society award for statistical excellence in the pharmaceutical industry.

Biopharmaceuticals:Biochemistry and Biotechnology, Second Edition, provides a comprehensive, up-to-date overview of the science and medical applications of biopharmaceutical products.

Extensively revised and updated, the Second Edition of this highly successful textbook discusses the latest developments in the field, providing a greater focus on actual commercial products and how they are manufactured. Substantial new sections on detailing biopharmaceutical drug delivery and how advances in genomics and proteomics will impact upon (bio)pharmaceutical drug development are also included.

• Provides a balanced overview of the biopharmaceutical industry, including material on key developments in the field.
• Each chapter supplemented with an extensive further reading section
• Aimed at students taking courses in biotechnology, pharmaceutical science, biochemistry, microbiology or medicine
• An ideal reference for those already employed in the (bio)pharmaceutical sector wishing to gain a better overview of the industry.

Reviews of Biopharmaceuticals:

‘Biopharmaceuticals: Biochemistry and Biotechnology contain(s) some very useful information and ultimately serves as a comprehensive introduction to both the biopharmaceutical industry and its role within the larger arena of pharmaceuticals. For those new to, or keen to be involved with biopharmaceuticals, the book will undoubtedly provide a solid grounding of not just the technology, but of the industry as a whole’ Pharmaceutical Technology Europe

‘This great compliment of scientific literature, is easy-to-read and understand, up-to-date as well as comprehensive,’ Bioseparation

‘Biopharmaceuticals……is well written and provides an excellent overview of the biotechnology industry….This book is an excellent and balanced source of information about the biotechnology industry’ FEBS Letters

Plant diseases are a serious threat to food production. This unique volume provides the fundamental knowledge and practical use of B.subtilis as a promising biocontrol agent. In order to replace chemical pesticides, one possibility is microbial pesticides using safe microbes. Bacillus subtilis is one of several candidates. Screening of the bacterium, the application of plant tests, clarification of its suppressive mechanism to plant pathogens and engineering aspects of suppressive peptides production are presented here. The author illustrates how B. subtilis is far more advantageous than, for example, Pseudomonas in biocontrol and can be considered as an useful candidate. Features: Bacterium B. subtilis suppresses many plant pathogens and is a biocontrol agent to replace chemical pesticides The book presents the bacterium's suppressive mechanism to plant pathogens, and engineering aspects of suppressive peptides production Biological control of plant disease plays an important role in sustainable agricultural production practices and is expected to replace agricultural chemicals

The First Book to Describe the Technical and Practical Elements of Chemical Text Mining Explores the development of chemical structure extraction capabilities and how to incorporate these technologies in daily research work For scientific researchers, finding too much information on a subject, not finding enough information, or not being able to access full text documents often costs them time, money, and quality. Addressing these concerns, Chemical Information Mining: Facilitating Literature-Based Discovery presents strategic ideas for properly selecting and successfully using the best text mining tools for scientific research. Links chemical and biological entities at the heart of life science research The book focuses on information extraction issues, highlights available solutions, and underscores the value of these solutions to academic and commercial scientists. After introducing the drivers behind chemical text mining, it discusses chemical semantics. The contributors describe the tools that identify and convert chemical names and images to structure-searchable information. They also explain natural language processing, name entity recognition concepts, and semantic web technologies. Following a section on current trends in the field, the book looks at where information mining approaches fit into the research needs within the life sciences. Shaping the future of scientific information and knowledge management By building knowledge and competency in the growing area of literature-based discovery, this book shows how text mining of the chemical literature can increase drug discovery opportunities and enhance life science research.

Medicinal Chemistry of Neglected and Tropical Diseases: Advances in the Design and Synthesis of Antimicrobial Agents consolidates and describes modern drug discovery and development approaches currently employed to identify effective chemotherapeutic agents for the treatment of Neglected Tropical Diseases (NTDs) from a medicinal chemistry perspective. Chapters are designed to cater to the needs of medicinal chemists who work with chemotherapeutic developments for NTDs, as well as serve as a guide to budding medicinal chemists who wish to work in this area. It will introduce rational drug design approaches adopted in designing chemotherapeutics and validated targets available for the purpose.

Essential information for architects, designers, engineers, equipment suppliers, and other professionals who are working in or entering the biopharmaceutical manufacturing field Biomanufacturing facilities that are designed and built today are radically different than in the past. The vital information and knowledge needed to design and construct these increasingly sophisticated biopharmaceutical manufacturing facilities is difficult to find in published literature--and it's rarely taught in architecture or design schools. This is the first book for architects and designers that fills this void. Process Architecture in Biomanufacturing Facility Design provides information on design principles of biopharmaceutical manufacturing facilities that support emerging innovative processes and technologies, use state-of-the-art equipment, are energy efficient and sustainable, and meet regulatory requirements. Relying on their many years of hands-on design and operations experience, the authors emphasize concepts and practical approaches toward design, construction, and operation of biomanufacturing facilities, including product-process-facility relationships, closed systems and single use equipment, aseptic manufacturing considerations, design of biocontainment facility and process based laboratory, and sustainability considerations, as well as an outlook on the facility of the future. Provides guidelines for meeting licensing and regulatory requirements for biomanufacturing facilities in the U.S.A and WHO--especially in emerging global markets in India, China, Latin America, and the Asia/Pacific regions Focuses on innovative design and equipment, to speed construction and time to market, increase energy efficiency, and reduce footprint, construction and operational costs, as well as the financial risks associated with construction of a new facility prior to the approval of the manufactured products by regulatory agencies Includes many diagrams that clarify the design approach Process Architecture in Biomanufacturing Facility Design is an ideal text for professionals involved in the design of facilities for manufacturing of biopharmaceuticals and vaccines, biotechnology, and life-science industry, including architects and designers of industrial facilities, construction, equipment vendors, and mechanical engineers. It is also recommended for university instructors, advanced undergraduates, and graduate students in architecture, industrial engineering, mechanical engineering, industrial design, and industrial interior design.

The vast and exciting Brazilian flora biodiversity is still underexplored. Several research groups are devoted to the study of the chemical structure richness found in the different Biomes. This volume presents a comprehensive account of the research collated on natural products produced from Brazilian medicinal plants and focuses on various aspects of the field. The authors describe the key natural products and their extracts with emphasis upon sources, an appreciation of these complex molecules and applications in science. Many of the extracts are today associated with important drugs, nutrition products, beverages, perfumes, cosmetics and pigments, and these are highlighted. Key Features: Presents Brazilian biodiversity: its flora, its people, and its research Describes the emergence of natural products research in Brazil Emphasizes the increasing global interests in botanical drugs Aids the international natural product communities to better understand the herbal resources in Brazil Discusses Brazilian legislation to work with native plants

Statistical methods that are commonly used in the review and approval process of regulatory submissions are usually referred to as statistics in regulatory science or regulatory statistics. In a broader sense, statistics in regulatory science can be defined as valid statistics that are employed in the review and approval process of regulatory submissions of pharmaceutical products. In addition, statistics in regulatory science are involved with the development of regulatory policy, guidance, and regulatory critical clinical initiatives related research. This book is devoted to the discussion of statistics in regulatory science for pharmaceutical development. It covers practical issues that are commonly encountered in regulatory science of pharmaceutical research and development including topics related to research activities, review of regulatory submissions, recent critical clinical initiatives, and policy/guidance development in regulatory science. Devoted entirely to discussing statistics in regulatory science for pharmaceutical development. Reviews critical issues (e.g., endpoint/margin selection and complex innovative design such as adaptive trial design) in the pharmaceutical development and regulatory approval process. Clarifies controversial statistical issues (e.g., hypothesis testing versus confidence interval approach, missing data/estimands, multiplicity, and Bayesian design and approach) in review/approval of regulatory submissions. Proposes innovative thinking regarding study designs and statistical methods (e.g., n-of-1 trial design, adaptive trial design, and probability monitoring procedure for sample size) for rare disease drug development. Provides insight regarding current regulatory clinical initiatives (e.g., precision/personalized medicine, biomarker-driven target clinical trials, model informed drug development, big data analytics, and real world data/evidence). This book provides key statistical concepts, innovative designs, and analysis methods that are useful in regulatory science. Also included are some practical, challenging, and controversial issues that are commonly seen in the review and approval process of regulatory submissions. About the author Shein-Chung Chow, Ph.D. is currently a Professor at Duke University School of Medicine, Durham, NC. He was previously the Associate Director at the Office of Biostatistics, Center for Drug Evaluation and Research, United States Food and Drug Administration (FDA). Dr. Chow has also held various positions in the pharmaceutical industry such as Vice President at Millennium, Cambridge, MA, Executive Director at Covance, Princeton, NJ, and Director and Department Head at Bristol-Myers Squibb, Plainsboro, NJ. He was elected Fellow of the American Statistical Association and an elected member of the ISI (International Statistical Institute). Dr. Chow is Editor-in-Chief of the Journal of Biopharmaceutical Statistics and Biostatistics Book Series, Chapman and Hall/CRC Press, Taylor & Francis, New York. Dr. Chow is the author or co-author of over 300 methodology papers and 30 books.

This up-to-the-minute Second Edition of an incomparable resource describes in detail the bases for developing dosage forms for use in animals-highlighting the data necessary to meet regulatory approval. Demonstrates the successful characterization, control, and registration of new veterinary medicines! Thoroughly rewritten and enlarged to reflect the technical advances that have occurred since the previous edition, Development and Formulation of Veterinary Dosage Forms, Second Edition discusses the reasons for dosage form selection explains the latest available technologies examines new drug therapeutics reveals up-to-date techniques and applications for pharmacokinetic data covers the formulation of products derived from biotechnology elucidates recent analytical methods shows how to determine the type of dosage form appropriate for particular species and more! Written by a team of international authorities from North America and Europe and containing over 1100 bibliographic citation, figures, and tables, Development and Formulation of Veterinary Dosage Forms, Second Edition is an essential reference for pharmaceutical, animal, and quality control scientists; research pharmacists and pharmacologists; veterinarians; drug quality assurance and regulatory personnel in government and industry; pathologists; microbiologists; virologists; physiologists; toxicologists; and upper-level undergraduate and graduate students in these disciplines.

This book highlights recently discovered aspects of "middle-size molecules," focusing on (1) their unique bio-functions on the basis of derivatives and conjugates of natural products, saccharides, peptides, and nucleotides; (2) the synthesis of structurally complex natural products; (3) special synthetic methods for -conjugated functional molecules; and (4) novel synthetic methods using flow chemistry. Given its scope, the book is of interest to industrial researchers and graduate students in the fields of organic chemistry, medicinal chemistry, and materials science.

This book is directed to the student or professional who has difficulty with basic pharmacy math. It covers Roman and Arabic numerals, fractions, decimals, ratios, proportions, percentages, systems of measurement including household conversions, and interpretation of medication orders.

This book highlights how terpenoids act as biological messengers and can be used as medicine against liver disease, neurodegenerative disease, cancer, infectious disease, cardiovascular disease, and inflammatory diseases. It emphasizes the metabolic engineering approach of terpenoids production and their toxicity.

Biological drug and vaccine manufacturing has quickly become one of the highest-value fields of bioprocess engineering, and many bioprocess engineers are now finding job opportunities that have traditionally gone to chemical engineers. Fundamentals of Modern Bioprocessing addresses this growing demand. Written by experts well-established in the field, this book connects the principles and applications of bioprocessing engineering to healthcare product manufacturing and expands on areas of opportunity for qualified bioprocess engineers and students. The book is divided into two sections: the first half centers on the engineering fundamentals of bioprocessing; while the second half serves as a handbook offering advice and practical applications. Focused on the fundamental principles at the core of this discipline, this work outlines every facet of design, component selection, and regulatory concerns. It discusses the purpose of bioprocessing (to produce products suitable for human use), describes the manufacturing technologies related to bioprocessing, and explores the rapid expansion of bioprocess engineering applications relevant to health care product manufacturing. It also considers the future of bioprocessing-the use of disposable components (which is the fastest growing area in the field of bioprocessing) to replace traditional stainless steel. In addition, this text: Discusses the many types of genetically modified organisms Outlines laboratory techniques Includes the most recent developments Serves as a reference and contains an extensive bibliography Emphasizes biological manufacturing using recombinant processing, which begins with creating a genetically modified organism using recombinant techniques Fundamentals of Modern Bioprocessing outlines both the principles and applications of bioprocessing engineering related to healthcare product manufacturing. It lays out the basic concepts, definitions, methods and applications of bioprocessing. A single volume comprehensive reference developed to meet the needs of students with a bioprocessing background; it can also be used as a source for professionals in the field.

This valuable new book offers a new perspective on dendrimers that bridges the gap between basic research and applied nanomedicine. It explores the ultimate effectiveness of dendrimers in theranostics, a promising field that combines therapeutics and diagnostics into single multifunctional formulations used to affect therapy or treatment of a disease state. The authors examine the potential uses of dendrimers, which have proven their capabilities in local/systemic drug delivery, physical stabilization of the drug, solubility enhancement of the poorly soluble drugs, and gene delivery.