Real-world evidence (RWE) has been at the forefront of pharmaceutical innovations. It plays an important role in transforming drug development from a process aimed at meeting regulatory expectations to an operating model that leverages data from disparate sources to aid business, regulatory, and healthcare decision making. Despite its many benefits, there is no single book systematically covering the latest development in the field. Written specifically for pharmaceutical practitioners, Real-World Evidence in Drug Development and Evaluation, presents a wide range of RWE applications throughout the lifecycle of drug product development. With contributions from experienced researchers in the pharmaceutical industry, the book discusses at length RWE opportunities, challenges, and solutions. Features Provides the first book and a single source of information on RWE in drug development Covers a broad array of topics on outcomes- and value-based RWE assessments Demonstrates proper Bayesian application and causal inference for real-world data (RWD) Presents real-world use cases to illustrate the use of advanced analytics and statistical methods to generate insights Offers a balanced discussion of practical RWE issues at hand and technical solutions suitable for practitioners with limited data science expertise

The growing interest in using combination drugs to treat various complex diseases has spawned the development of many novel statistical methodologies. The theoretical development, coupled with advances in statistical computing, makes it possible to apply these emerging statistical methods in in vitro and in vivo drug combination assessments. However, despite these advances, no book has served as a single source of information for statistical methods in drug combination research, nor has there been any guidance for experimental strategies. Statistical Methods in Drug Combination Studies fills that gap, covering all aspects of drug combination research, from designing in vitro drug combination studies to analyzing clinical trial data. Featuring contributions from researchers in industry, academia, and regulatory agencies, this comprehensive reference: Describes statistical models used to characterize dose-response patterns of monotherapies and evaluate the combination drug synergy Offers guidance for estimating interaction indices and constructing their associated confidence intervals to assess drug interaction Introduces a practical and innovative Bayesian approach to Phase I cancer trials, including actual trial examples to illustrate use Examines strategies in the fixed-dose combination therapy clinical development via case studies stemming from regulatory reviews Evaluates computational tools and software packages used to apply novel statistical methods in combination drug development Statistical Methods in Drug Combination Studies provides researchers with a solid understanding of the available statistical methods and computational tools and how to apply them in drug combination studies. The book is equally useful for statisticians to become better equipped to deal with drug combination study design and analysis in their practice.

Discover the Latest Statistical Approaches for Modeling Exposure-Response Relationships Written by an applied statistician with extensive practical experience in drug development, Exposure-Response Modeling: Methods and Practical Implementation explores a wide range of topics in exposure-response modeling, from traditional pharmacokinetic-pharmacodynamic (PKPD) modeling to other areas in drug development and beyond. It incorporates numerous examples and software programs for implementing novel methods. The book describes using measurement error models to treat sequential modeling, fitting models with exposure and response driven by complex dynamics, and survival analysis with dynamic exposure history. It also covers Bayesian analysis and model-based Bayesian decision analysis, causal inference to eliminate confounding biases, and exposure-response modeling with response-dependent dose/treatment adjustments (dynamic treatment regimes) for personalized medicine and treatment adaptation. Many examples illustrate the use of exposure-response modeling in experimental toxicology, clinical pharmacology, epidemiology, and drug safety. Some examples demonstrate how to solve practical problems while others help with understanding concepts and evaluating the performance of new methods. The provided SAS and R codes enable readers to test the approaches in their own scenarios. Although application oriented, this book also gives a systematic treatment of concepts and methodology. Applied statisticians and modelers can find details on how to implement new approaches. Researchers can find topics for or applications of their work. In addition, students can see how complicated methodology and models are applied to practical situations.

Liposome Technology, Volume I: Liposome Preparation and Related Techniques, Third Edition, is a thoroughly updated and expanded new edition of a classic text in the field. Including step-by-step technical details, Volume I illustrates numerous methods for liposome preparation and auxiliary techniques necessary for the stabilization and characterization of liposomes. This source also offers critical discussions of the methodologies of each technology described so that readers can examine the benefits and limitations and compare it to other approaches.

The first edition of Pharmaceutical Extrusion Technology, published in 2003, was deemed the seminal book on pharmaceutical extrusion. Now it is expanded and improved, just like the usage of extrusion has expanded, improved and evolved into an accepted manufacturing technology to continuously mix active pharmaceutical ingredients with excipients for a myriad of traditional and novel dosage forms. Pharmaceutical Extrusion Technology, Second Edition reflects how this has spawned numerous research activities, in addition to hardware and process advancements. It offers new authors, expanded chapters and contains all the extrusion related technical information necessary for the development, manufacturing, and marketing of pharmaceutical dosage forms. Key Features: Reviews how extrusion has become an accepted technology to continuously mix active pharmaceutical ingredients with excipients Focuses on equipment and process technology Explains various extrusion system configurations as a manufacturing methodology for a variety of dosage forms Presents new opportunities available only via extrusion and future trends Includes contributions of experts from the process and equipment fields

Comparative effectiveness research (CER) is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care (IOM 2009). CER is conducted to develop evidence that will aid patients, clinicians, purchasers, and health policy makers in making informed decisions at both the individual and population levels. CER encompasses a very broad range of types of studies-experimental, observational, prospective, retrospective, and research synthesis. This volume covers the main areas of quantitative methodology for the design and analysis of CER studies. The volume has four major sections-causal inference; clinical trials; research synthesis; and specialized topics. The audience includes CER methodologists, quantitative-trained researchers interested in CER, and graduate students in statistics, epidemiology, and health services and outcomes research. The book assumes a masters-level course in regression analysis and familiarity with clinical research.

In a global clinical development strategy, multiregional clinical trials (MRCTs) are vital in the development of innovative medicines. Multiregional Clinical Trials for Simultaneous Global New Drug Development presents a comprehensive overview on the current status of conducting MRCTs in clinical development. International experts from academia, industry, and health organizations address various aspects of the important problems in global clinical development and MRCTs. The book first provides a high-level introduction to the context, motivation, opportunities, and challenges in simultaneous global clinical development using MRCTs. It then focuses on the design, monitoring, and analysis/interpretation of MRCTs. The book concludes with an examination of the latest research topics from MRCT perspectives, such as special considerations by local health authorities, health economic evaluations, benefit-risk assessment, and medical devices. Explaining how to design, conduct, and interpret MRCTs, this book will help biostatisticians working in the late-stage clinical development of medical products. It will also be useful for statisticians and clinicians in the biopharmaceutical industry, regulatory agencies, and medical research institutes.

This book focuses on analytical similarity assessment in biosimilar product development following the FDA's recommended stepwise approach for obtaining totality-of-the-evidence for approval of biosimilar products. It covers concepts such as the tiered approach for assessment of similarity of critical quality attributes in the manufacturing process of biosimilar products, models/methods like the statistical model for classification of critical quality attributes, equivalence tests for critical quality attributes in Tier 1 and the corresponding sample size requirements, current issues, and recent developments in analytical similarity assessment.

Develop Effective Immunogenicity Risk Mitigation Strategies Immunogenicity assessment is a prerequisite for the successful development of biopharmaceuticals, including safety and efficacy evaluation. Using advanced statistical methods in the study design and analysis stages is therefore essential to immunogenicity risk assessment and mitigation strategies. Statistical Methods for Immunogenicity Assessment provides a single source of information on statistical concepts, principles, methods, and strategies for detection, quantification, assessment, and control of immunogenicity. The book first gives an overview of the impact of immunogenicity on biopharmaceutical development, regulatory requirements, and statistical methods and strategies used for immunogenicity detection, quantification, and risk assessment and mitigation. It then covers anti-drug antibody (ADA) assay development, optimization, validation, and transfer as well as the analysis of cut point, a key assay performance parameter in ADA assay development and validation. The authors illustrate how to apply statistical modeling approaches to establish associations between ADA and clinical outcomes, predict immunogenicity risk, and develop risk mitigation strategies. They also present various strategies for immunogenicity risk control. The book concludes with an explanation of the computer codes and algorithms of the statistical methods. A critical issue in the development of biologics, immunogenicity can cause early termination or limited use of the products if not managed well. This book shows how to use robust statistical methods for detecting, quantifying, assessing, and mitigating immunogenicity risk. It is an invaluable resource for anyone involved in immunogenicity risk assessment and control in both non-clinical and clinical biopharmaceutical development.

Dendrimers are a new class of macromolecule increasingly used in the fields of synthetic organic chemistry, biology, medicine and biotechnology. Dendrimers in Medicine and Biotechnology: New Molecular Tools looks at this exciting and rapidly growing area of science. Using an interdisciplinary approach with particular emphasis on biological applications, the book discusses the relationship between the dendrimer molecular motif and its biological properties. A general introduction to the subject of dendrimers, including definitions of terms and symbols, is provided. Subsequent sections discuss topics including dendrimers in biological systems, dendrimers as drug delivery devices, dendrimers in diagnostics and dendrimer drugs. Throughout the book examples from current research are also provided. This book will appeal to a wide range of scientists, including non specialists who require an introduction to dendrimers, as well as those wishing to know more about the application of dendrimers in the field of biology and medicine.

A practice-oriented desktop reference for medical professionals, toxicologists and pharmaceutical researchers, this handbook provides
systematic coverage of the metabolic pathways of all major classes of xenobiotics in the human body. The first part comprehensively reviews
the main enzyme systems involved in biotransformation and how they are orchestrated in the body, while parts two to four cover the three
main classes of xenobiotics: drugs, natural products, environmental pollutants. The part on drugs includes more than 300 substances from
five major therapeutic groups (central nervous system, cardiovascular system, cancer, infection, and pain) as well as most drugs of abuse
including nicotine, alcohol and "designer" drugs. Selected, well-documented case studies from the most important xenobiotics classes illustrate general principles of metabolism, making this equally useful for teaching courses on pharmacology, drug metabolism or molecular
toxicology.
Of particular interest, and unique to this volume is the inclusion of a wide range of additional xenobiotic compounds, including food supplements, herbal preparations, and agrochemicals.

Structure-based drug discovery is a collection of methods that exploits the ability to determine and analyse the three dimensional structure of biological molecules. These methods have been adopted and enhanced to improve the speed and quality of discovery of new drug candidates. After an introductory overview of the principles and application of structure-based methods in drug discovery, this book then describes the essential features of the various methods. Chapters on X-ray crystallography, NMR spectroscopy, and computational chemistry and molecular modelling describe how these particular techniques have been enhanced to support rational drug discovery, with discussions on developments such as high throughput structure determination, probing protein-ligand interactions by NMR spectroscopy, virtual screening and fragment-based drug discovery. The concluding chapters complement the overview of methods by presenting case histories to demonstrate the major impact that structure-based methods have had on discovering drug molecules. Written by international experts from industry and academia, this comprehensive introduction to the methods and practice of structure-based drug discovery not only illustrates leading-edge science but also provides the scientific background for the non-expert reader. The book provides a balanced appraisal of what structure-based methods can and cannot contribute to drug discovery. It will appeal to industrial and academic researchers in pharmaceutical sciences, medicinal chemistry and chemical biology, as well as providing an insight into the field for recent graduates in the biomolecular sciences.

Offering step-by-step technical details, Liposome Technology, Third Edition, Three Volume Set provides comprehensive coverage of all aspects of liposome technology, including liposome preparation and analysis, entrapment of drugs and other materials into liposomes, and liposome interaction with the biological environment to be applied in the detection, therapy, or prevention of disease. The text offers critical discussions of the methodologies of each technology discussed so that readers can examine the benefits and limitations and compare it to other methods. This Third Edition features 55 chapters written by leading international experts. Because of the considerable progress in liposome related techniques and their application in therapy since the publication of the Second Edition in 1992, over half of the chapters are new to the edition, and the other chapters have been extensively updated. Liposome Technology, Third Edition, Three Volume Set is an ideal resource for pharmaceutical scientists, researchers, regulatory personnel, FDA personnel, and medicinal chemists working in this discipline.

This book on hollow fiber contractors presents an up-to-date compilation of the latest developments and milestones in this membrane technology. Hollow Fiber Membrane Contactors: Module Fabrication, Design and Operation, and Potential Applications provides a comprehensive discussion of hollow fiber membrane applications (including a few case studies) in biotechnology, chemical, food, and nuclear engineering. The chapters in this book have been classified using the following, based on different ways of contacting fluids with each other: Gas-liquid contacting; Liquid-liquid contacting; Supported liquid membrane; Supported gas membrane; Fluid-fluid contacting. Other features include: Discusses using non-dispersive solvent extraction, hollow fiber strip dispersion, hollow fiber supported liquid membranes and role of process intensification in integrated use of these processes Provides technical and economic perspectives with several case studies related to specific scenarios Demonstrates module fabrication, design, operation and maintenance of hollow fiber contactors for different applications and performance Presents discussion on newer concepts like membrane emulsification, membrane nanoprecipitation, membrane crystallization and membrane condenser Special focus on emerging areas such as the use of hollow fiber contactor in back end of nuclear fuel cycle, membrane distillation, dehumidification of air and gas absorption and stripping Discusses theoretical analysis including computational modeling of different hollow fiber membrane processes, and presents emphasis on newly developed area of hollow fiber membrane based analytical techniques Presents discussion on upcoming area dealing with hollow fiber contactors-based technology in fermentation and enzymatic transformation and in chiral separations This book is equally suited for newcomers to the field, as well as for engineers and scientists that have basic knowledge in this field but are interested in obtaining more information about specific future applications.

The proliferation of herbal remedies worldwide has most dangerously outpaced quality information on their safe use. This book fills a tremendous void by offering authoritative information on the actions of herbal remedies and the results of their interactions with standard medications. It offers specific, authoritative information with a conceptual approach that focuses not only on the specific interactions, but also on the mechanisms behind those interactions and their clinical significance. With contributions from leading experts on herb-drug interactions, the text examines the overall use of herbs, includes sections on individual herbs, and considers regulatory issues and concerns.

Selecting illustrative examples from the recent literature, this reference studies the underlying principles and physics of a wide range of spectroscopic techniques utilized in the pharmaceutical sciences and demonstrates various applications for each method analyzed in the text-showing how knowledge of the mechanisms of spectroscopic phenomena may facilitate more advanced technologies in the field.

This book offers a broad summary of the wild plants and their usage, as well as the growing interest in ethnopharmacology research. The book comprises of important issues such as diversity of wild plants with emphasis on medicinal and food plants, threats to wild plants and traditional ethnobotanical knowledge, their uses in skin diseases, snake-bites, in cosmeceuticals, etc. Moreover, the ethnopharmacological relevance of wild plants in Latin America has been discussed. The chapters include a wide range of case studies, giving updated evidence on the importance of their wild plant resources from different countries including Peru, Nepal, Bangladesh, India, Pakistan, Brazil. In addition, some specific species are used to explain their potential properties, as well as the dangers of their use without guidance of trained natural healers. The book discusses traditional usage and properties of wild plants and is entirely different from other related publications and useful for the researchers working in the areas of conservation biology, botany, ethnobiology, ethnopharmacology, policymakers, etc.

Divided into three expansive sections on biotechnological advances, applications, and research prospects, this reference provides expert summaries of the state-of-the-science in personal care product development-clearly depicting the latest breakthroughs and practices in biotechnology for the formulation and increased safety of new personal care ingredients.

This book provides a broad range of applications and recent advances in the search for biofilm materials in nature. It also explains the future implications for biofilms in the areas of advanced molecular genetics, pharmaceuticals, pharmacology, and toxicology. This book is comprised of 20 chapters from leading experts in the field and it examines immunology and microbiological studies derived from biofilms as well as explores environmental, agricultural, and chemical impacts on biofilms. It is divided into five subdivisions: biofilms and its complications, biofilm infections in human body, detection of biofilm-forming pathogens, antibiofilm chemotherapy, and biofilms production tools in aquaculture. This book may be used as a text or reference for everyone interested in microbial biofilms and their current applications. It is also highly recommended for environmental microbiologists, medical microbiologists, bioremediation experts, and microbiologists working in biocorrosion, biofouling, biodegradation, water microbiology, quorum sensing, and many other related areas. Scientists in academia, research laboratories, and industry will also find it of interest. This book includes chapter homework problems and case studies. Powerpoints are also available for adopting instructors. Discusses and clarifies the resource of isolation and chemical properties from biofilms Discusses the latest pharmaceutical, pharmacological, and medicinal approaches toward the treatment of chronic and uncured diseases, such as Alzheimer's osteoporotic, sexual dysfunction, sleep sickness, allergy treatment, asthma, hair loss, AIDS, hypertension, antiaging, etc. Examines immunology and microbiological studies derived from biofilms Explores environmental, agricultural, and chemical impacts on biofilms. Dr. Bakrudeen Ali Ahmed Abdul is an Associate Professor, the Head of the Department of Biochemistry and Dean of the School of Life Sciences, Centre for Research and Development (CRD), PRIST Deemed University, Vallam, Thanjavur, Tamil Nadu, India. His research areas include the application of plant biochemistry, bioactive compound production, biotechnological methods, development of pharmaceutical products and pharmacological studies.

The perceived lack of drug discovery productivity in recent times has led to much debate in the pharmaceutical/biotechnology industry as escalating R&D costs are not being matched by increased output. Few observers doubt that selecting the right targets, ie those which are critical to disease pathology and are druggable, is the best starting point for improved productivity.
The seven chapters of this volume describe recent progress towards drugs acting at a range of druggable targets. One chapter addresses kinases, one covers an ion channel, two proteases are featured and three of the chapters cover G-protein coupled receptors, which has historically perhaps been the most fruitful area for medicinal chemists.
*Presents the latest research in the field of drug discovery
*Publishes on an annual basis to bring you the most innovative updates in medicinal chemistry
*Available as an online resource via ScienceDirect

The need to screen targets faster and more efficiently, coupled with advances in parallel and multiplex chemical synthesis, has contributed to the increasing use of multiwell assays for drug discovery. The Handbook of Assay Development in Drug Discovery is a reference that describes the complete armament of tools currently available for performing various assay techniques. Featuring contributions from assay developers in the pharmaceutical and vendor communities, the book presents descriptions of methods, laboratory guidelines and protocols used to perform such methods, specific examples of each assay system, and troubleshooting tools. The handbook describes biochemical assay classes as well as non-class specific assay development for cell-based assays. It covers a wide range of target classes-including kinases, proteases, nuclear receptors, and GPCRs-and describes currently employed methods and assay types, such as radioligand binding assays, image analysis assays, enzyme fragment complementation, and bioluminescent and fluorescent-based assays. Designed as a guide to running an assay from start to finish, the Handbook of Assay Development in Drug Discovery is an ideal bench top companion for discovery researchers, laboratory managers, academics, and other scientists involved in drug discovery screening, lead profiling, therapeutic target evaluation, and assay development and implementation in the pharmaceutical and biotechnology industries. Daniel E. Levy, editor of the Drug Discovery Series, is the founder of DEL BioPharma, a consulting service for drug discovery programs. He also maintains a blog that explores organic chemistry.

Genetic toxicology is considered to be an important assessment tool as there is genetic impact of artificial chemicals. Insight on Genotoxicity discusses testing, mechanism, prediction, and bioindicator of genotoxicity taking into consideration recent advances in nano-engineered particles. Corollary of DNA dent is also discussed in detail taking into consideration the impact of ICH guidelines on genotoxicity testing, which is important for drug discovery innovation and development. Perspective review of genotoxicity evaluation in phytopharmaceuticals has been mentioned along with the prevention of genotoxicity in brief viewpoint. Salient Features Presents methods, standard protocols, and guidelines for genotoxicity testing Examines the impact of ICH Guidelines on genetic toxicity testing which is a regulatory requirement for drug discovery and development Defines appropriate strategies about advances in in vivo genotoxicity testing which have been listed along with progress and prospects Discusses advancement in the high-throughput approaches for genotoxicity testing Details computational prediction of genotoxicity with consideration of mutagenicity, chromosomal damage caused and strategies for computational prediction in drug development

With a high diversity of vegetation in Iran, over 8000 plant species are in existence. More than 2300 species of these plants have medicinal, edible and industrial properties, and more than 1700 species of them are endemic. Natural Products and Botanical Medicines of Iran provides an overview on important endemic plants and their usages. All results have been tabulated and key detailed information of each species is presented with background data. Features: Provides an understanding of indigenous plant-derived natural medicines of the most important medicinal plants in the region Includes discussions and critical views on the potentials and challenges for further development of the selected plants in a modern setting Details the important plants and sets out the chapters based on either taxonomy or medical use

Melville's Monumental Imagination explores the connection between the contested 19th century American monument tradition and one of the nation's most revered authors, Herman Melville (1819-1891). The book was written to fill a void in recent Melville scholarship. To date, there has not been a monograph that focuses exclusively on Melville's incorporation of monuments in his fictional world. The book charts the territory of Melville's novels in order to provide a trajectory of the monumental image in one particular literary form. This feature allows the reader to gradually see the monumental image as an important marker that sheds light into Melville's eventual abandonment of long fiction. Melville's Monumental Imagination combines literary analysis and cultural criticism for a long neglected aspect of our nation's iconic development in statuary.

Presenting breakthrough research pertinent to scientists in a wide range of disciplines-from medicine and biotechnology to cosmetics and pharmacy-this Second Edition provides practical approaches to complex formulation problems encountered in the development of particulate delivery systems at the micro- and nano-size level. Completely revised and expanded to represent the most up-to-date studies in the field, this guide covers advances in pulsatile delivery systems, injectable microparticulate systems, and site-specific delivery systems, as well as potential therapeutic applications of nanotechnology.