The concepts of estimands, analyses (estimators), and sensitivity are interrelated. Therefore, great need exists for an integrated approach to these topics. This book acts as a practical guide to developing and implementing statistical analysis plans by explaining fundamental concepts using accessible language, providing technical details, real-world examples, and SAS and R code to implement analyses. The updated ICH guideline raises new analytic and cross-functional challenges for statisticians. Gaps between different communities have come to surface, such as between causal inference and clinical trialists, as well as among clinicians, statisticians, and regulators when it comes to communicating decision-making objectives, assumptions, and interpretations of evidence. This book lays out a path toward bridging some of these gaps. It offers ? A common language and unifying framework along with the technical details and practical guidance to help statisticians meet the challenges ? A thorough treatment of intercurrent events (ICEs), i.e., postrandomization events that confound interpretation of outcomes and five strategies for ICEs in ICH E9 (R1) ? Details on how estimands, integrated into a principled study development process, lay a foundation for coherent specification of trial design, conduct, and analysis needed to overcome the issues caused by ICEs: ? A perspective on the role of the intention-to-treat principle ? Examples and case studies from various areas ? Example code in SAS and R ? A connection with causal inference ? Implications and methods for analysis of longitudinal trials with missing data Together, the authors have offered the readers their ample expertise in clinical trial design and analysis, from an industrial and academic perspective.

Recent regulations on heavy metal testing have required the pharmaceutical industry to monitor a suite of elemental impurities in pharmaceutical raw materials, drug products and dietary supplements. These new directives s are described in the new United States Pharmacopeia (USP) Chapters <232>, <233>, and <2232>, together with Q3D, Step 4 guidelines for elemental impurities, drafted by the ICH (International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use), a consortium of global pharmaceutical associations, including the European Pharmacopeia (Ph.Eur.), the Japanese Pharmacopeia (JP) and the USP. This book provides a complete guide to the analytical methodology, instrumental techniques and sample preparation procedures used for measuring elemental impurities in pharmaceutical and nutraceutical materials. It offers readers the tools to better understand plasma spectrochemistry to optimize detection capability for the full suite of elemental PDE (Permitted Daily Exposure) levels in the various drug delivery categories. Other relevant information covered in the book includes: The complete guide to measuring elemental impurities in pharmaceutical and nutraceutical materials. Covers heavy metals testing in the pharmaceutical industry from an historical perspective. Gives an overview of current USP Chapters <232> <233> and <2232> and ICH Q3D Step 4 Guidelines. Explains the purpose of validation protocols used in Chapter <233>, including how J-values are calculated Describes fundamental principles and practical capabilities of ICP-MS and ICP-OES. Offers guidelines about the optimum strategy for risk assessment Provides tips on how best to prepare and present your data for regulatory inspection. An indispensable resource, the fundamental principles and practical benefits of ICP-OES and ICP-MS are covered in a reader-friendly format that a novice, who is carrying out elemental impurities testing in the pharmaceutical and nutraceutical communities, will find easy to understand.

Provide readers and front line health care products manufacturers, R&D management and biotech laboratories all the information they need to know to develop a GMP oriented industry with trained and skilled personnel and manufacture products that meet GMP and regulatory requirements Covers basic quality concepts and the elements of vendor's assessment, qualification and approval required by the pharmaceutical educational institutions and professional certification bodies Provides stepwise guidance on how to evaluate, audit, qualify and approve an API and packaging material manufacturer and supplier to enhance the GMP within the industry Provides ready to use regulatory documentation, e.g. letter of commitment, Questionnaire, SOP etc. required for API and Packaging Materials contract Provided material can be easily tailored to incorporate changes to add in-house vendor's qualification requirements

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

Provides information about the different surfactants. Describes constraints in production and novel production strategies. Includes application oriented aspects of biosurfactants in various fields such as agriculture, Environmental, Pharmaceutical, and Food industry. Authored by experts in the field of biosurfactants.

Drug products are complex mixtures of drugs and excipients and, as such, their chemical and physical stability kinetics are complex. This book discusses the stability of these dosage forms with preformulation studies through to the studies on the final products. The book is intended for graduate students, researchers and professionals in the field of Pharmaceutics and Pharmaceutical Chemistry.

Phylogenetic presentation of medicinal plants and a chemotaxonomical rationale of antiviral, antibacterial, and antifungal action. Discusses chemical structure-activity relationship, pharmacokinetics, and oral bioavailability of antimicrobial principles Introduces the molecular mechanism of natural products on viruses, bacteria, and fungi. Contains a selection of hand-made botanical plates and useful bibliographic references A useful research tool for postgraduates, academics, and the pharmaceutical, herbal, or nutrition industries.

Confidence Intervals for Discrete Data in Clinical Research is designed as a toolbox for biomedical researchers. Analysis of discrete data is one of the most used yet vexing areas in clinical research. The array of methodologies available in the literature to address the inferential questions for binomial and multinomial data can be a double-edged sword. On the one hand, these methods open a rich avenue of exploration of data; on the other, the wide-ranging and competing methodologies potentially lead to conflicting inferences, adding to researchers' confusion and frustration and also leading to reporting bias. This book addresses the problems that many practitioners experience in choosing and implementing fit for purpose data analysis methods to answer critical inferential questions for binomial and count data. The book is an outgrowth of the authors' collective experience in biomedical research and provides an excellent overview of inferential questions of interest for binomial proportions and rates based on count data, and reviews various solutions to these problems available in the literature. Each chapter discusses the strengths and weaknesses of the methods and suggests practical recommendations. The book's primary focus is on applications in clinical research, and the goal is to provide direct benefit to the users involved in the biomedical field.

With contributions from leading researchers in the nanomedicine field from industry, academia, and government and private research institutions across the globe, the volume provides an up-to-date report on topical issues in nano-drug delivery and nanotechnological approaches to tissue engineering. The volume offers research on a variety of diverse nano-based drug delivery systems along with discussions of their efficacy, safety, toxicology, and applications for different purposes. Focusing on nanotechnology approaches to tissue engineering, this volume considers the use of hydrogel systems, nanoceria and micro- and nano-structured biomaterials for bone tissue engineering, mesenchymal stem cells, and more.

Describes 'real life' examples in Medicinal Chemistry. Integrates the use of physical, chemical, and biological concepts that are important in drug design. Highlights the "ips" and "downs" that come with drug discovery. Aims to inspire students who may be struggling with the challenges and thought process in drug design. Intends to be an excellent companion text, illustrating real life experiences, for graduate and postgraduate students.

Coherent treatment of a variety of approaches to multiple comparisons Broad coverage of topics, with contributions by internationally leading experts Detailed treatment of applications in medicine and life sciences Suitable for researchers, lecturers / students, and practitioners

Before now, biological systems could only be expressed in terms of linear relationships, however, as knowledge grows and new techniques of analysis on biological systems is made available, we are realizing the non-linearity of these systems. The concepts and techniques of nonlinear analysis allow for more realistic and accurate models in science. The Future of Pharmaceuticals: A Nonlinear Analysis provides an opportunity to understand the non-linearity of biological systems and its application in various areas of science, primarily pharmaceutical sciences. This book will benefit professionals in pharmaceutical industries, academia, and policy who are interested in an entirely new approach to how we will treat disease in the future. Key Features: Addresses a new approach of nonlinear analysis. Applies a theory of projection to chalk out the future, instead of basing on linear evolution. Provides an opportunity to better understand the non-linearity in biological systems and its applications in various areas of science, primarily pharmaceutical sciences. Helps change the thought process for those looking for answers to their questions which they do not find in the linear relationship approach. Encourages a broader perspective for the creative process of drug development.

Covers recent research on the following aspects of vitamin B12: structure, constitution, properties, nomenclature, organometallic reactivity, usage in diagnosis and therapy, and role in biological systems Discusses the chemistry of the carbon cobalt bond Is illustrated throughout with excellent figures and references in each chapter

Vitamin C holds a unique place in scientific and cultural history. In this book, a group of leading scientific researchers describe new insights into the myriad ways vitamin C is employed during normal physiological functioning. In addition, the text provides an extensive overview of the following: the rationale for utilizing vitamin C in the clinic, updates on recent uses of vitamin C in cancer treatment through high-dose intravenous therapies, the role vitamin C plays in the treatment of sepsis and infectious disease, management of the ways vitamin C can improve stem cell differentiation, as well as vitamin C use in other important health situations. Features Includes chapters from a team of leading international scholars Reviews the history and recent research on the functions, benefits, and uses of vitamin C Focuses special attention on the way vitamin C can be used in the treatment of cancers Discusses how vitamin C can be employed against infectious disease

The discovery and use of medicines is just as fascinating a human scientific endeavor as space flight or the tracing of human evolution. It is also the everyday task of hundreds of thousands of pharmacists, pharmaceutical chemists and researchers worldwide. Based on his profound knowledge of past and present paradigms in the development of medicines, Enrique Ravina takes the reader from the very beginnings of pharmacology to the multibillion-dollar business it represents today. Recounting the often spectacular successes and failures of innovative drugs as well as the people who discovered them, he brings abstract science to life in anecdotal form. For anyone with a more than superficial interest in the science of drugs and all those interested in knowing how drugs have been developed, how they have reached us, and became part of our daily life. This book is beautifully illustrated, containing many rare and historical photographs of drugs and their discoverers, and abounds with references to the primary literature, listing seminal publications alongside more modern reviews for readers seeking further details. With a Foreword by Hugo Kubinyi

The advanced AI techniques are essential for resolving various problematic aspects emerging in the field of bioinformatics. This book covers the recent approaches in artificial intelligence and machine learning methods and their applications in Genome and Gene editing, cancer drug discovery classification, and the protein folding algorithms among others. Deep learning, which is widely used in image processing, is also applicable in bioinformatics as one of the most popular artificial intelligence approaches. The wide range of applications discussed in this book are an indispensable resource for computer scientists, engineers, biologists, mathematicians, physicians, and medical informaticists. Features: Focusses on the cross-disciplinary relation between computer science and biology and the role of machine learning methods in resolving complex problems in bioinformatics Provides a comprehensive and balanced blend of topics and applications using various advanced algorithms Presents cutting-edge research methodologies in the area of AI methods when applied to bioinformatics and innovative solutions Discusses the AI/ML techniques, their use, and their potential for use in common and future bioinformatics applications Includes recent achievements in AI and bioinformatics contributed by a global team of researchers

Drug Discovery with Privileged Building Blocks traces back PharmaBlock's founding philosophy of designing privileged building blocks. High-quality building blocks are crucial not only to biological activities of different molecules but also to ADMET properties, which eventually will impact the success rate of drug discovery projects. A thorough study of how building blocks perform in drug molecules and a regular analysis of new building block structures in the latest researches have proven to be a fruitful strategy to generate novel building blocks. Using this strategy, PharmaBlock has supplied the drug industry with a great number of building blocks, which are increasingly being adopted by drug hunters, and these are identified in this book. Each chapter may be read and studied without learning the previous chapters. This book will be a good starting point for novice medicinal chemists, and veteran medicinal chemists will find it useful as well. Key Feature The book covers privileged building blocks appearing most frequently on patents for novel drugs. The latest relevant tactics are explained in the context of drug design and medicinal chemistry. Key synthesis, especially large-scale synthesis, is described. The most recent literature references are cited.

Drug Discovery with Privileged Building Blocks traces back PharmaBlock's founding philosophy of designing privileged building blocks. High-quality building blocks are crucial not only to biological activities of different molecules but also to ADMET properties, which eventually will impact the success rate of drug discovery projects. A thorough study of how building blocks perform in drug molecules and a regular analysis of new building block structures in the latest researches have proven to be a fruitful strategy to generate novel building blocks. Using this strategy, PharmaBlock has supplied the drug industry with a great number of building blocks, which are increasingly being adopted by drug hunters, and these are identified in this book. Each chapter may be read and studied without learning the previous chapters. This book will be a good starting point for novice medicinal chemists, and veteran medicinal chemists will find it useful as well. Key Feature The book covers privileged building blocks appearing most frequently on patents for novel drugs. The latest relevant tactics are explained in the context of drug design and medicinal chemistry. Key synthesis, especially large-scale synthesis, is described. The most recent literature references are cited.

This book details the novel nanocarriers named 'invasomes" and how they are used for dermal and transdermal drug delivery. The text describes their composition, usage of skin as a drug delivery route and liposomes as skin delivery systems. Included are reviewed studies revealing the importance of invasomes in this field.

This book presents an overview of the current status of translating the RNAi cancer therapeutics in the clinic, a brief description of the biological barriers in drug delivery, and the roles of imaging in aspects of administration route, systemic circulation, and cellular barriers for the clinical translation of RNAi cancer therapeutics, and with partial content for discussing the safety concerns. It then focuses on imaging-guided delivery of RNAi therapeutics in preclinical development, including the basic principles of different imaging modalities, and their advantages and limitations for biological imaging. With growing number of RNAi therapeutics entering the clinic, various imaging methods will play an important role in facilitating the translation of RNAi cancer therapeutics from bench to bedside. RNAi technique has become a powerful tool for basic research to selectively knock down gene expression in vitro and in vivo. Our scientific and industrial communities have started to develop RNAi therapeutics as the next class of drugs for treating a variety of genetic disorders, such as cancer and other diseases that are particularly hard to address with current treatment strategies. Key Features Provides insight into the current advances and hurdles of RNAi therapeutics. Accelerates RNAi, miRNAs, and siRNA drug development for cancer therapy from bench to bedside. Addresses various modifications and novel delivery strategies for miRNAs, piRNAs and siRNA delivery in anticancer therapeutics. Explores the need for the interaction of hematologists,cell biologists, immunologists, and material scientists in the development of novel cancer therapies. Describes the current status of clinical trials related to miRNA and siRNA-based cancer therapy Presents remaining issues that need to be overcome to establish successful therapies.

Ocimum species has been used as a traditional remedy for various ailments such as arthritis, bronchitis, cold, conjunctivitis, diarrhea, dysentery, and flatulence, as well as for healing wounds and lowering blood glucose level. These are characterized by variations in their morphology such as the shape, size and pigmentation of leaves, which cause differences in chemical composition and affect the commercial value of this genus. This book describes phytochemical investigations of Ocimum species using LC-MS/MS instruments to study qualitative and quantitative variations of phytochemicals in different Ocimum species. Features: Collection of Ayurvedic features and scientific analytical and pharmacological evidence of most important medicinal plants of genus Ocimum. Chemical signatures for the identification of Ocimum species. Easy-to-use analytical procedure for quality control of plants of Ocimum species and its herbal products.

This publication is based on peer-reviewed manuscripts from the 2019 Conference on Drug Design & Discovery Technologies (CDDT) held at Ramaiah University of Applied Sciences, India. Providing a wide range of up to date topics on the latest advancements in drug design and discovery technologies, this book ensures the reader receives a good understanding of the scope of the field. Aimed at scientists, students, regulators, academics and consultants throughout the world, this book is an ideal resource for anyone interested in the state of the art in drug design and discovery.

The cost for bringing new medicine from discovery to market has nearly doubled in the last decade and has now reached $2.6 billion. There is an urgent need to make drug development less time-consuming and less costly. Innovative trial designs/ analyses such as the Bayesian approach are essential to meet this need. This book will be the first to provide comprehensive coverage of Bayesian applications across the span of drug development, from discovery, to clinical trial, to manufacturing with practical examples. This book will have a wide appeal to statisticians, scientists, and physicians working in drug development who are motivated to accelerate and streamline the drug development process, as well as students who aspire to work in this field. The advantages of this book are: Provides motivating, worked, practical case examples with easy to grasp models, technical details, and computational codes to run the analyses Balances practical examples with best practices on trial simulation and reporting, as well as regulatory perspectives Chapters written by authors who are individual contributors in their respective topics Dr. Mani Lakshminarayanan is a researcher and statistical consultant with more than 30 years of experience in the pharmaceutical industry. He has published over 50 articles, technical reports, and book chapters besides serving as a referee for several journals. He has a PhD in Statistics from Southern Methodist University, Dallas, Texas and is a Fellow of the American Statistical Association. Dr. Fanni Natanegara has over 15 years of pharmaceutical experience and is currently Principal Research Scientist and Group Leader for the Early Phase Neuroscience Statistics team at Eli Lilly and Company. She played a key role in the Advanced Analytics team to provide Bayesian education and statistical consultation at Eli Lilly. Dr. Natanegara is the chair of the cross industry-regulatory-academic DIA BSWG to ensure that Bayesian methods are appropriately utilized for design and analysis throughout the drug-development process.

This book provides insight into the world of pharmaceutical quality systems and the key elements that must be in place to change the business and organizational dynamics from task-oriented procedure-based cultures to truly integrated quality business systems that are self-detecting and correcting. Chapter flow has been changed to adopt a quality systems organization approach, and supporting chapters have been updated based on current hot topics including the impact of the worldwide supply chain complexity and current regulatory trends. Key Features: Presents insight into the world of pharmaceutical quality systems Analyzes regulatory trends and expectations Includes approaches and practices used in the industry to comply with regulatory requirements Discusses recent worldwide supply chain issues Delivers valuable information to a worldwide audience regarding the current GMP practices in the industry

Medicinal Plants in the Asia Pacific for Zoonotic Pandemics provides an unprecedented, comprehensive overview of the phylogeny, botany, ethnopharmacology, and pharmacology of more than 100 plants used in the traditional medical systems of Asia and Pacific. It discusses their actions and potentials against viruses, bacteria, and fungi that represent a threat of epidemic and pandemic diseases, with an emphasis on the molecular basis and cellular pathways. This book presents scientific names, the botanical classification, traditional medicinal uses, active chemical constituents, and pharmacology. This volume is a critical reference for anyone involved in the discovery of lead molecules or phytopharmaceutical products for the prevention or treatment of pandemic viral, bacterial, or fungal infections. FEATURES Phylogenetic presentation of medicinal plants and a chemotaxonomical rationale of antiviral, antibacterial, and antifungal actions Discusses the chemical structure-activity relationship, pharmacokinetics, and oral bioavailability of antimicrobial principles Introduces the molecular mechanism of natural products on viruses, bacteria, and fungi Contains a selection of botanical plates and useful bibliographic references This book is a useful research tool for postgraduates, academics, and the pharmaceutical, herbal, and nutrition industries. Medicinal Plants in the Asia Pacific for Zoonotic Pandemics includes commentary sections that invite further research and reflection on the fascinating and timely subject of the development of drugs and herbals from Asia-Pacific medicinal plants to safeguard humanity and other life forms against the forthcoming waves of viral, bacterial, or fungal pandemics. This book is an ideal reference text for medicinal plant enthusiasts.