Many practitioners in the pharmaceutical industry are still largely unfamiliar with benefit-risk assessment, despite its growing prominence in drug development and commercialization. Helping to alleviate this knowledge gap, Benefit-Risk Assessment in Pharmaceutical Research and Development provides a succinct overview of the key considerations relevant to benefit-risk assessment across the pharmaceutical R&D spectrum, from early clinical development to late-stage development to regulatory review to post-launch assessment. The book first presents interpretations of benefit and risk in the context of a molecule moving from preclinical evaluation into its early testing in humans. It next considers benefit and risk characterization and assessment during a molecule's journey from its clinical evaluation in humans through its submission to regulators for marketing approval. Throughout these sections, the book offers insight into the role of benefit-risk assessment in heightening understanding among key stakeholders by shaping questions and guiding discussions among scientists, physicians, developers, and regulatory agencies. The book also focuses on a molecule's entry into the marketplace as a drug available for consumption by people. It explores the role of benefit-risk assessment as the relevance of carefully collected clinical efficacy and safety metrics fades in the wake of real-world use and evidence of effectiveness and safety. Bringing together the expertise of 15 contributors from academia and the industry, this book offers an easy-to-read guide to the various facets of benefit-risk assessment in the major stages of pharmaceutical R&D. Suitable for those in both technical and managerial roles, it enables readers to communicate more effectively across their development chain as well as rationally and thoughtfully embed benefit-risk assessment into their R&D processes.

Used routinely in drug control laboratories, forensic laboratories, and as a research tool, thin layer chromatography (TLC) plays an important role in pharmaceutical drug analyses. It requires less complicated or expensive equipment than other techniques, and has the ability to be performed under field conditions. Filling the need for an up-to-date, complete reference, Thin Layer Chromatography in Drug Analysis covers the most important methods in pharmaceutical applications of TLC, namely, analysis of bulk drug material and pharmaceutical formulations, degradation studies, analysis of biological samples, optimization of the separation of drug classes, and lipophilicity estimation. The book is divided into two parts. Part I is devoted to general topics related to TLC in the context of drug analysis, including the chemical basis of TLC, sample pleparation, the optimization of layers and mobile phases, detection and quantification, analysis of ionic compounds, and separation and analysis of chiral substances. The text addresses the newest advances in TLC instrumentation, two-dimensional TLC, quantification by slit scanning densitometry and image analysis, statistical processing of data, and various detection and identification methods. It also describes the use of TLC for solving a key issue in the drug market-the presence of substandard and counterfeit pharmaceutical products. Part II provides an in-depth overview of a wide range of TLC applications for separation and analysis of particular drug groups. Each chapter contains an introduction about the structures and medicinal actions of the described substances and a literature review of their TLC analysis. A useful resource for chromatographers, pharmacists, analytical chemists, students, and R&D, clinical, and forensic laboratories, this book can be utilized as a manual, reference, and teaching source.

Take Your NI Trial to the Next Level Reflecting the vast research on noninferiority (NI) designs from the past 15 years, Noninferiority Testing in Clinical Trials: Issues and Challenges explains how to choose the NI margin as a small fraction of the therapeutic effect of the active control in a clinical trial. Requiring no prior knowledge of NI testing, the book is easily accessible to both statisticians and nonstatisticians involved in drug development. With over 20 years of experience in this area, the author introduces the basic elements of the NI trials one at a time in a logical order. He discusses issues with estimating the effect size based on historical placebo control trials of the active control. The book covers fundamental concepts related to NI trials, such as assay sensitivity, constancy assumption, discounting, and preservation. It also describes patient populations, three-arm trials, and the equivalence of three or more groups.

Piper betle (betel vine) a pan-Asiatic, tropical plant, which can also grow under mild subtropical areas, is essentially grown for leaves which are chewed with array of additives besides slaked lime. The plant is cultivated widely in India and its surrounding areas. Phytochemistry of Piper betel landraces presents a brief on the distribution, historical and cultural aspects, and properties ascribed to this plant in the ancient texts. Phytochemical and pharmacological information has also been included to underscore the importance of this plant in the present time. A detailed account on metabolic profiling employing modern methods is included, such as real-time, direct analysis of the flight mass spectrometric method and chemometric analysis for characterization of the available biodiversity and signatures specific to gender and geographical location. It was also possible to identify the gender of unknown landraces, with the help of principal component analysis. Features: Elaborates on the chemical diversity within Piper betle. Piper betle leaves have mouth freshening antimicrobial compounds. Use of chemical signatures for the identification of different Piper betle landraces, their gender and geographical locations.

This book discusses fabrication of functionalized gold nanoparticles (GNPs) and multifunctional nanocomposites, their optical properties, and applications in biological studies. This is the very first book of its kind to comprehensively discuss published data on in vitro and in vivo biodistribution, toxicity, and uptake of GNP by mammalian cells providing a systematization of data over the GNP types and parameters, their surface functionalization, animal and cell models. As distinct from other related books, Gold Nanoparticles in Biomedical Applications discusses the immunological properties of GNPs and summarizes their applications as an antigen carrier and adjuvant in immunization for the preparation of antibodies in vivo. Although the potential of GNPs in nanobiotechnology has been recognized for the past decade, new insights into the unique properties of multifunctional nanostructures have recently emerged. With these developments in mind, this book unites ground breaking experimental data with a discussion of hybrid nanoparticle systems that combine different nanomaterials to create multifunctional structures. These novel hybrids constitute the material basis of theranostics, bringing together the advanced properties of functionalized GNPs and composites into a single multifunctional nanostructure with simultaneous diagnostic and therapeutic functions. Such nanohybrids can be physically and chemically tailored for a particular organ, disease, and patient thus making personalized medicine available.

Focusing on group sequential procedures, summarizes the sequential statistical methods used in anticancer, antiviral, cardiovascular, and gastrointestinal drug research and screening. The clinical and preclinical applications are mainly presented as case studies, many of which form part of New Drug

Interval-Censored Time-to-Event Data: Methods and Applications collects the most recent techniques, models, and computational tools for interval-censored time-to-event data. Top biostatisticians from academia, biopharmaceutical industries, and government agencies discuss how these advances are impacting clinical trials and biomedical research. Divided into three parts, the book begins with an overview of interval-censored data modeling, including nonparametric estimation, survival functions, regression analysis, multivariate data analysis, competing risks analysis, and other models for interval-censored data. The next part presents interval-censored methods for current status data, Bayesian semiparametric regression analysis of interval-censored data with monotone splines, Bayesian inferential models for interval-censored data, an estimator for identifying causal effect of treatment, and consistent variance estimation for interval-censored data. In the final part, the contributors use Monte Carlo simulation to assess biases in progression-free survival analysis as well as correct bias in interval-censored time-to-event applications. They also present adaptive decision making methods to optimize the rapid treatment of stroke, explore practical issues in using weighted logrank tests, and describe how to use two R packages. A practical guide for biomedical researchers, clinicians, biostatisticians, and graduate students in biostatistics, this volume covers the latest developments in the analysis and modeling of interval-censored time-to-event data. It shows how up-to-date statistical methods are used in biopharmaceutical and public health applications.

Analytical ultracentrifugation (AUC) can supply rich information on the mass, shape, size distribution, solvation, and composition of macromolecules and nanoscopic particles. It also provides a detailed view of their reversible single- or multi-component interactions over a wide range of affinities. Yet this powerful technique has been hard to master in mainstream molecular sciences due to a lack of comprehensive books on the subject. Filling this gap in the literature of biophysical methodology, Basic Principles of Analytical Ultracentrifugation explains the fundamentals in the theory and practice of AUC. The book provides you with up-to-date experimental information to confidently practice AUC. You will understand the basic concepts, full potential, and possible pitfalls of AUC as well as appreciate the current relevance of past work in the field. The book first introduces the basic principles and technical setup of an AUC experiment and briefly describes the optical systems used for detection. It then explores the ultracentrifugation experiment from a macromolecular standpoint, offering a detailed physical picture of the sedimentation process and relevant macromolecular parameters. The authors present important practical aspects for conducting an experiment, including sample preparation, data acquisition and data structure, and the execution of the centrifugal experiment. They also cover instrument calibration and quality control experiments.

This book traces the evolution of the pharmacist from compounder-dispenser to advisor counselor. It explores the impact on pharmacy of broad sociologic, demographic, and economic trends and examines controversial issues such as professional versus business objectives, managed cost care, coginitive services, pharmaceutical care, and the future roles of pharmacists. Drawing on the literature in sociology and social pathology, the book relates some theoretical constructs to the changing roles of the pharmacist. It includes coverage of pharmaceutical education, disease state management, managed care, and new technologies.

In the fast-developing field of nanomedicine, a broad variety of materials have been used for the development of advanced delivery systems for drugs, genes, and diagnostic agents. With the recent breakthroughs in the field, we are witnessing a new age of disease management, which is governed by precise regulation of dosage and delivery. This book presents the advances in the use of lipid-based and inorganic nanomaterials for medical imaging, diagnosis, theranostics, and drug delivery. The materials discussed include liposome-scaffold systems, elastic liposomes, targeted liposomes, solid lipid nanoparticles, lipoproteins, exosomes, porous inorganic nanomaterials, silica nanoparticles, and inorganic nanohybrids. The book provides all available information about them and describes in detail their advantages and disadvantages and the areas where they could be utilized successfully.

The Textbook of Ion Channels is a set of three volumes providing a wide-ranging reference source on ion channels for students, instructors, and researchers. Ion channels are membrane proteins that control the electrical properties of neurons and cardiac cells, mediate the detection and response to sensory stimuli like light, sound, odor, and taste, and regulate the response to physical stimuli like temperature and pressure. In non-excitable tissues, ion channels are instrumental for the regulation of basic salt balance that is critical for homeostasis. Ion channels are located at the surface membrane of cells, giving them the unique ability to communicate with the environment, as well as the membrane of intracellular organelles, allowing them to regulate internal homeostasis. Ion channels are fundamentally important for human health and diseases, and are important targets for pharmaceuticals in mental illness, heart disease, anesthesia, pain and other clinical applications. The modern methods used in their study are powerful and diverse, ranging from single ion-channel measurement techniques to models of ion channel diseases in animals, and human clinical trials for ion channel drugs. Volume I, Part 1 covers fundamental topics such as the basic principles of ion permeation and selectivity, voltage-dependent, ligand-dependent and mechano-dependent ion channel activation mechanisms, the mechanisms for ion channel desensitization and inactivation and basic ion channel pharmacology and inhibition. Volume I, Part 2 aims to offer a practical guide of cardinal methods for researching ion channels, including heterologous expression and voltage-clamp and patch-clamp electrophysiology, isolation of native currents using patch-clamp, modelling ion channel gating, structures and its dynamics, crystallography and cryo- electron microscopy, fluorescence and paramagnetic resonance spectroscopy methods and genetics approaches in model organisms. All three volumes give the reader an introduction to fundamental concepts needed to understand the mechanism of ion channels, a guide to the technical aspects of ion channel research, offer a modern guide to the properties of major ion channel families, and include coverage of key examples of regulatory, physiological, and disease roles for ion channels.

Nervous system diseases represent a major health concern worldwide. Although important financial and professional investment, their etiology and pathophysiology still remain mostly elusive. Moreover, the clinical need of disease-modifying therapies is still unmet. In the last decades, traditional R&D has failed in identifying new effective therapies in many medical areas and drug repositioning has recently emerged as a promising alternative strategy to de novo drug discovery to improve and accelerate therapeutic development. For the first time, Drug Repositioning: Approaches and Applications for Neurotherapeutics reviews history and advances in drug repositioning, with a special focus on therapeutics for nervous system diseases. International experts from Academia, Industry and Non-profit organisations will provide different views on drug repositioning advantages, challenges and specific applications, which will be covered for nervous system diseases including Alzheimer's, Parkinson's, Huntington's diseases, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, ischemic stroke, and psychiatric disorders. This book provides a balanced overview and synthesis of drug repositioning concept, methods and applications for neurotherapeutics. It represents a valuable resource for students, scientists and clinicians working in academic settings, industry and government agencies within the fields of neuroscience, pharmacology, neurology, pharmaceutical sciences, drug discovery and development.

Connecting past, present, and future instrument development and use, Biocalorimetry: Foundations and Contemporary Approaches explores biocalorimetry's history, fundamentals, methodologies, and applications. Some of the most prominent calorimeter developers and users share invaluable personal accounts of discovery, discussing innovative techniques as well as special and original applications. Wide in scope, the book also covers calorimetry use on membranes, nucleic acids, and proteins and addresses both thermodynamics and kinetics. The book begins with a look at the historical development of calorimeters needed for biological research. It then describes advanced approaches that use high-quality commercial calorimeters to study biochemical and other biological processes. It also shows how novel experimental designs and data analysis procedures are applied to proteins, DNA, membranes, and living matter.

With the advent of analytical techniques and capabilities to measure particle sizes in nanometer ranges, there has been tremendous interest in the use of nanoparticles for more efficient methods of drug delivery. Nanoparticulate Drug Delivery Systems addresses the scientific methodologies, formulation, processing, applications, recent trends, and emerging technologies in the research of nanoparticulate drug delivery systems (NPDDS). It extensively covers applications of NPDDS- including lipid nanoparticles for dermal applications; nanocarriers for the treatment of restenosis; and for ocular, central nervous system, and gastrointestinal applications. It also explores its use as an adjuvant for vaccine development. Supplying insight from international experts, this guide Discusses formulation for poorly soluble drugs Presents biological requirements for nanotherapeutic applications Demonstrates the role of nanobiotechnology in the development of nanomedicine Documents recent advances in nanoparticulate technology-including electrospining, formation of microcrystals, and production of liquid crystalline phases Examines the technology of metallic nanoparticles

During the last two decades, the pharmaceutical industry has been under pressure to reduce development costs and the time needed to bring drugs to market in order to maximize return on investment and bring treatments to patients sooner. To meet these ends, pharmaceutical scientists working in the differing areas of pharmacy, pharmaceutics, and pharmacokinetics are collaborating to address physicochemical and biological issues in the early stages of development to avoid problems in later stages. In Vitro-In Vivo Correlation (IVIVC) is a multidisciplinary tool that has been successfully applied in testing the effectiveness of a drug substance. The only comprehensive guide available on IVIVC, this source illustrates the emerging importance of IVIVC in the drug development process, and covers the most recent advances and regulatory perspectives on the role of IVIVC in the pharmaceutical industry.

The over-riding premise for biotechnology in this book is bringing novel products to market to substantially advance patient care and disease mitigation. Biotechnology, over its relatively brief existence of 40 years, has experienced a mercurial growth. The vast educational need for biotechnology information in this rapidly burgeoning field is a basic rationale here. However a more prominent underpinning is that, bringing biotech products to market for patient care involves success in the following four areas of engagement simultaneously - scientific advances for healthcare technologies, novel and varied products for untreated diseases, regulatory authorities, and biotech companies. Features Comprehensive coverage of biotechnology science topics used in development and manufacturing Addresses all the scientific technologies within biotechnology responsible for products on the market and the pipeline Presents business issues such as marketing and sales of the products, as well as companies engaged, and how biotech business has evolved

A comprehensive guide for physicians conducting clinical research, this second edition addresses a broader research perspective. It includes information on the implications of the ICH Guidelines, current FDA regulations, and an Internet address directory. Everything the clinical trial manager, planner, monitor, and investigator need to know about the design, establishment, monitoring, and close-out of a trial is in this book. The chapters address the elements of clinical research, professional interactions, FDA regulations and good clinical practices guidelines, investigational agent management, designing a study and protocol development, conducting the study, and more.

This book addresses the recent trends and clinical research being reported in last 5 to 10 years in the field of nasal drug delivery systems. In recent years, interest in using nasal passage as drug absorption site has received increased attention from formulation scientists. Nasal passages, even though a small surface area of the body as compared to other absorption passage such as Gastrointestinal tract or skin,  show significant possibility for drug absorption at a quicker rate. There is also a possibility of delivering drugs to the brain using this passage and targeting drugs through the nasal passage. The book has 19 chapters addressing various aspects of nasal drug delivery systems such as an overview of anatomy and physiology of the nasal passage from a drug delivery point of view to global market opportunities for nasal drug delivery. In between, it addresses various aspects of nasal drug delivery. There are very few titles exclusively dedicated to nasal drug delivery, covering the formulation and developmental aspects, and addressing the challenges and solutions. The primary audiences for the book are graduate students in field of medicine, pharmacy and also various researchers who are working in the area of nasal drug delivery in addition to students who are specializing in field of medicine in ENT. This book provides comprehensive information on all the aspects related to the nasal drug delivery of various drug molecules.

This authoritative reference presents an up-to-date review of the testing methods, emerging technologies, and analytical systems and procedures used to prevent the microbial contamination of pharmaceutical processes, products, and environments. It identifies new tools for sample analysis and evaluation and the impact of these advancements on the continuous supply and manufacturing of pharmaceutical products. With more than 100 tables and 430 current references, the book contains a detailed analysis of microbial contamination recalls for nonsterile and sterile pharmaceutical products, demonstrating the distribution of microorganisms worldwide and the identification by geographical regions.

1) Discusses the affordability of future therapy and the possible challenges for health insurance systems 2) Highlights the gradual move from repeated treatment administration to one single administration with potential for a definite cure 3) Describes the potential change of paradigm which will challenge all payers and may question the sustainability of our health care systems

If you have ever wondered when visiting the pharmacy how the dosage of your prescription is determined this book will answer your questions. Dosing information on drug labels is based on discussion between the pharmaceutical manufacturer and the drug regulatory agency, and the label is a summary of results obtained from many scientific experiments. The book introduces the drug development process, the design and the analysis of clinical trials. Many of the discussions are based on applications of statistical methods in the design and analysis of dose response studies. Important procedural steps from a pharmaceutical industry perspective are also examined.

Validation of Computerized Analytical and Networked Systems provides the definitive rationales, logic, and methodology for validation of computerized analytical systems. Whether you are involved with formulation or analytical development laboratories, chemical or microbiological quality control laboratories, LIMS installations, or any aspect of robotic in a healthcare laboratory, this book furnishes complete validation details. International and FDA regulations and requirements are discussed and juxtaposed with numerous practical examples that show you how to cost-effectively and efficiently accomplish validation acceptable to FDA GCP/GLP/GMP, NAMAS, and EN45001 standards. The templates included provide documentation examples and the many checklists found throughout the book assure that all aspects of covered in a logical sequence. The chapters describe and explain such topics as the Product Life Cycle revalidation, change control, documentation requirements, qualifications, testing, data validation and traceability, inspection, SOPs, and many other that help streamline the validation process.

Emphasizing the role of good statistical practices (GSP) in drug research and formulation, this book outlines important statistics applications for each stage of pharmaceutical development to ensure the valid design, analysis, and assessment of drug products under investigation and establish the safety and efficacy of pharmaceutical compounds. Coverage include statistical techniques for assay validation and evaluation of drug performance characteristics, testing population/individual bioequivalence and in vitro bioequivalence according to the most recent FDA guidelines, basic considerations for the design and analysis of therapeutic equivalence and noninferiority trials.

Providing a significant cross-fertilization of ideas across several disciplines, Enhancement in Drug Delivery offers a unique comprehensive review of both theoretical and practical aspects of enhancement agents and techniques used for problematic administration routes. It presents an integrated evaluation of absorption enhancers and modes for promoting absorption that is especially valuable to those involved with the development of pharmaceutical, cosmetic, bioengineered, and medical products, as well as graduate students looking to study this intriguing field and those professionals involved with patents and regulatory issues. Organized by routes of administration, the book is divided into eight major sections: oral, rectal, buccal/sublingual, dermal/transdermal, nasal, vaginal/uterine, ocular, and brain. It offers fundamental as well specialized information including current findings on- * Surfactant use to accelerate macromolecule input * Targeted gastrointestinal delivery and enhanced absorption of lipophilic drugs * Permeation issues in rectal absorption * Chemical means of enhancement * Carriers for enhanced delivery to and across the skin * Methods associated with breaching the skin * Promoted buccal and sublingual absorption * Emerging ocular, nasal, vaginal, and uterine delivery systems * Carriers for overcoming the blood brain barrier Those investigators primarily involved with one specific route of delivery will be able to learn of helpful concepts and find additional stimulat

Covering regulatory requirements stipulated by the FDA, this book delineates the organization, planning, verification, and documentation activities and procedural controls required for compliance with worldwide computer systems validation regulations. The author introduces supporting technologies such as encryption and digital signatures and places regulatory compliance within the context of quality assurance. He demonstrates the importance of integrating validation activities into the system lifecycle using a structured top-down approach. He covers practical applications of quality assurance and engineering techniques as they relate to the development of systems fit to meet user and regulatory requirements.