Genomics in Drug Discovery and Development


Book Description

Early characterization of toxicity and efficacy would significantly impact the overall productivity of pharmaceutical R&D and reduce drug candidate attrition and failure. By describing the available platforms and weighing their relative advantages and disadvantages, including microarray data analysis, Genomics in Drug Discovery and Development introduces readers to the biomarker, pharmacogenomic, and toxicogenomics toolbox. The authors provide a valuable resource for pharmaceutical discovery scientists, preclinical drug safety department personnel, regulatory personnel, discovery toxicologists, and safety scientists, drug development professionals, and pharmaceutical scientists.




Biomarkers, Diagnostics and Precision Medicine in the Drug Industry


Book Description

The high failure rate in the pharmaceutical industry has positioned biomarkers and personalized medicine in the frontline, as possible solutions. If executed right, biomarkers and companion diagnostics (CDx) can potentially help the drug industry enhance the probability of success, accelerate the time to market, and, more importantly, benefit patients by supporting accurate diagnosis and selection of the most effective and least toxic therapies. This book aims to examine the challenges and limitations in biomarkers and laboratory tests. It also offers advice on best practices to ensure proper application of biomarkers and bridges the gap between diagnostic business development claims and real-life deliverables. The book covers biomarkers for different purposes, provides examples from different technologies, which includes standard-of-care approved assays as well as for-investigational-use and for-research-use-only assays. It also includes new data for biomarkers in different therapeutic indications and offers case studies and practical examples. This book serves as a reference to drug developers, IVD providers, clinical labs, healthcare givers, academicians, and researchers for best practices to help increase the probability of success in drug development and improve patient management. - Provides the unique insight of an expert with extensive experience in diagnostics and clinical laboratory on one side and drug discovery and development on the other side - Addresses the challenges of drug development and precision medicine and suggests how to eliminate or mitigate these challenges through better utilization of biomarkers and diagnostics in drug development and patient management - Features case studies and real-life examples from different classes of biomarkers on different platforms for different therapeutic areas and includes more than 200 illustrations




Biomarkers in Drug Development


Book Description

Discover how biomarkers can boost the success rate of drug development efforts As pharmaceutical companies struggle to improve the success rate and cost-effectiveness of the drug development process, biomarkers have emerged as a valuable tool. This book synthesizes and reviews the latest efforts to identify, develop, and integrate biomarkers as a key strategy in translational medicine and the drug development process. Filled with case studies, the book demonstrates how biomarkers can improve drug development timelines, lower costs, facilitate better compound selection, reduce late-stage attrition, and open the door to personalized medicine. Biomarkers in Drug Development is divided into eight parts: Part One offers an overview of biomarkers and their role in drug development. Part Two highlights important technologies to help researchers identify new biomarkers. Part Three examines the characterization and validation process for both drugs and diagnostics, and provides practical advice on appropriate statistical methods to ensure that biomarkers fulfill their intended purpose. Parts Four through Six examine the application of biomarkers in discovery, preclinical safety assessment, clinical trials, and translational medicine. Part Seven focuses on lessons learned and the practical aspects of implementing biomarkers in drug development programs. Part Eight explores future trends and issues, including data integration, personalized medicine, and ethical concerns. Each of the thirty-eight chapters was contributed by one or more leading experts, including scientists from biotechnology and pharmaceutical firms, academia, and the U.S. Food and Drug Administration. Their contributions offer pharmaceutical and clinical researchers the most up-to-date understanding of the strategies used for and applications of biomarkers in drug development.




General Methods in Biomarker Research and their Applications


Book Description

In the past decade there has been a major sea change in the way disease is diagnosed and investigated due to the advent of high throughput technologies, such as microarrays, lab on a chip, proteomics, genomics, lipomics, metabolomics etc. These advances have enabled the discovery of new and novel markers of disease relating to autoimmune disorders, cancers, endocrine diseases, genetic disorders, sensory damage, intestinal diseases etc. In many instances these developments have gone hand in hand with the discovery of biomarkers elucidated via traditional or conventional methods, such as histopathology or clinical biochemistry. Together with microprocessor-based data analysis, advanced statistics and bioinformatics these markers have been used to identify individuals with active disease or pathology as well as those who are refractory or have distinguishing pathologies. New analytical methods that have been used to identify markers of disease and is suggested that there may be as many as 40 different platforms. Unfortunately techniques and methods have not been readily transferable to other disease states and sometimes diagnosis still relies on single analytes rather than a cohort of markers. There is thus a demand for a comprehensive and focused evidenced-based text and scientific literature that addresses these issues. Hence the formulation of Biomarkers in Disease. The series covers a wide number of areas including for example, nutrition, cancer, endocrinology, cardiology, addictions, immunology, birth defects, genetics and so on. The chapters are written by national or international experts and specialists.




Drug Discovery and Evaluation: Methods in Clinical Pharmacology


Book Description

Drug Discovery and Evaluation has become a more and more difficult, expensive and time-consuming process. The effect of a new compound has to be detected by in vitro and in vivo methods of pharmacology. The activity spectrum and the potency compared to existing drugs have to be determined. As these processes can be divided up stepwise we have designed a book series "Drug Discovery and Evaluation" in the form of a recommendation document. The methods to detect drug targets are described in the first volume of this series "Pharmacological Assays" comprising classical methods as well as new technologies. Before going to man, the most suitable compound has to be selected by pharmacokinetic studies and experiments in toxicology. These preclinical methods are described in the second volume „Safety and Pharmacokinetic Assays". Only then are first studies in human beings allowed. Special rules are established for Phase I studies. Clinical pharmacokinetics are performed in parallel with human studies on tolerability and therapeutic effects. Special studies according to various populations and different therapeutic indications are necessary. These items are covered in the third volume: „Methods in Clinical Pharmacology".




Neuroscience Biomarkers and Biosignatures


Book Description

Biomarkers, or biological markers, are quantitative measurements that offer researchers and clinicians valuable insight into diagnosis, treatment and prognosis for many disorders and diseases. A major goal in neuroscience medical research is establishing biomarkers for disorders of the nervous system. Given the promising potential and necessity for neuroscience biomarkers, the Institute of Medicine Forum on Neuroscience and Nervous System Disorders convened a public workshop and released the workshop summary entitled Neuroscience Biomarkers and Biosignatures: Converging Technologies, Emerging Partnerships. The workshop brought together experts from multiple areas to discuss the most promising and practical arenas in neuroscience in which biomarkers will have the greatest impact. The main objective of the workshop was to identify and discuss biomarker targets that are not currently being aggressively pursued but that could have the greatest near-term impact on the rate at which new treatments are brought forward for psychiatric and neurological disorders.




Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease


Book Description

Many people naturally assume that the claims made for foods and nutritional supplements have the same degree of scientific grounding as those for medication, but that is not always the case. The IOM recommends that the FDA adopt a consistent scientific framework for biomarker evaluation in order to achieve a rigorous and transparent process.




Generating Evidence for Genomic Diagnostic Test Development


Book Description

Ten years after the sequencing of the human genome, scientists have developed genetic tests that can predict a person's response to certain drugs, estimate the risk of developing Alzheimer's disease, and make other predictions based on known links between genes and diseases. However, genetic tests have yet to become a routine part of medical care, in part because there is not enough evidence to show they help improve patients' health. The Institute of Medicine (IOM) held a workshop to explore how researchers can gather better evidence more efficiently on the clinical utility of genetic tests. Generating Evidence for Genomic Diagnostic Test Development compares the evidence that is required for decisions regarding clearance, use, and reimbursement, to the evidence that is currently generated. The report also addresses innovative and efficient ways to generate high-quality evidence, as well as barriers to generating this evidence. Generating Evidence for Genomic Diagnostic Test Development contains information that will be of great value to regulators and policymakers, payers, health-care providers, researchers, funders, and evidence-based review groups.




Improving and Accelerating Therapeutic Development for Nervous System Disorders


Book Description

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




Genomic Biomarkers for Pharmaceutical Development


Book Description

Genomic Biomarkers for Pharmaceutical Development: Advancing Personalized Health Care provides an in-depth review of the state of translational science across all stages of pharmaceutical development with a special focus on personalized health care. This book provides a complete picture of biomarker development and validation in a pharmaceutical setting while addressing the inherent challenges of targeting the appropriate indications, biomarker robustness, regulatory hurdles, commercialization and much more. It features case studies devoted to the applications of pharmacogenomics, toxicogenomics, and other genetic technologies as they support drug discovery and development. With chapters written by international authorities in industry and academia, this work is a truly unique presentation of the thoughts and approaches that lead to the development of personalized medicine. Intended for all those involved in clinical translational research, this book is the ideal resource for scientists searching for the applications, strategies and successful approaches of translational science in pharmaceutical development. - Provides case studies in applications of pharmacodynamic and predictive markers in drug development in oncology, autoimmunity, respiratory diseases and infectious diseases - Shows how to identify potential new therapeutic targets in different diseases and provides examples of potential new disease indications for life cycle management of drugs - Authored by leading international experts from industry and academia