This book focuses on the new discipline of translational medicine as it pertains to drug development within the pharmaceutical and biotechnology industry. It is essential for anyone interested in translational medicine from a variety of backgrounds: university institutes, medical schools, pharmaceutical companies and drug development researchers and decision-makers.

"A lot of hard-won knowledge is laid out here in a brief but informative way. Every topic is well referenced, with citations from both the primary literature and relevant resources from the internet." Review from Nature Chemical Biology Written by the founders of the SPARK program at Stanford University, this book is a practical guide designed for professors, students and clinicians at academic research institutions who are interested in learning more about the drug development process and how to help their discoveries become the novel drugs of the future. Often many potentially transformative basic science discoveries are not pursued because they are deemed ‘too early’ to attract industry interest. There are simple, relatively cost-effective things that academic researchers can do to advance their findings to the point that they can be tested in the clinic or attract more industry interest. Each chapter broadly discusses an important topic in drug development, from preclinical work in assay design through clinical trial design, regulatory issues and marketing assessments. After the practical overview provided here, the reader is encouraged to consult more detailed texts on specific topics of interest. "I would actually welcome it if this book’s intended audience were broadened even more. Younger scientists starting out in the drug industry would benefit from reading it and getting some early exposure to parts of the process that they’ll eventually have to understand. Journalists covering the industry (especially the small startup companies) will find this book a good reality check for many an over-hopeful press release. Even advanced investors who might want to know what really happens in the labs will find information here that might otherwise be difficult to track down in such a concentrated form."

Translational Medicine: Tools and Techniques provides a standardized path from basic research to the clinic and brings together various policy and practice issues to simplify the broad interdisciplinary field. With discussions from academic and industry leaders at international institutions who have successfully implemented translational medicine techniques and tools in various settings, readers will be guided through implementation strategies relevant to their own needs and institutions. The book also addresses regulatory processes in USA, EU, Japan and China. By providing details on omics sciences techniques, biomarkers, data mining and management approaches, case reports from industry, and tools to assess the value of different technologies and techniques, this book is the first to provide a user-friendly go-to guide for key opinion leaders (KOLs), industry administrators, faculty members, clinicians, researchers, and students interested in translational medicine. - Includes detailed and standardized information about the techniques and tools used in translational medicine - Provides specific industry case scenarios - Explains how to use translational medicine tools and techniques to plan and improve infrastructures and capabilities while reducing cost and optimizing resources

Filling a real knowledge gap, this handbook and ready reference is both modern and forward-looking in its emphasis on the "bench to bedside" translational approach to drug development. Clearly structured into three major parts, the book stakes out the boundaries of peptide drug development in the preclinical as well as clinical stages. The first part provides a general background and focuses on the characteristic strengths and weaknesses of peptide drugs. The second section contains five cases studies of peptides from diverse therapeutic fields, and the lessons to be learned from them, while the final part looks at new targets and opportunities, discussing several drug targets and diseases for which peptide drugs are currently being developed.

Pharmaceutical Medicine and Translational Clinical Research covers clinical testing of medicines and the translation of pharmaceutical drug research into new medicines, also focusing on the need to understand the safety profile of medicine and the benefit-risk balance. Pharmacoeconomics and the social impact of healthcare on patients and public health are also featured. It is written in a clear and straightforward manner to enable rapid review and assimilation of complex information and contains reader-friendly features.As a greater understanding of these aspects is critical for students in the areas of pharmaceutical medicine, clinical research, pharmacology and pharmacy, as well as professionals working in the pharmaceutical industry, this book is an ideal resource. - Includes detailed coverage of current trends and key topics in pharmaceutical medicine, including biosimilars, biobetters, super generics, and - Provides a comprehensive look at current and important aspects of the science and regulation of drug and biologics discovery

Principles of Translational Science in Medicine: From Bench to Bedside, Second Edition, provides an update on major achievements in the translation of research into medically relevant results and therapeutics. The book presents a thorough discussion of biomarkers, early human trials, and networking models, and includes institutional and industrial support systems. It also covers algorithms that have influenced all major areas of biomedical research in recent years, resulting in an increasing numbers of new chemical/biological entities (NCEs or NBEs) as shown in FDA statistics. The book is ideal for use as a guide for biomedical scientists to establish a systematic approach to translational medicine. - Provides an in-depth description of novel tools for the assessment of translatability of trials to balance risk and improve projects at any given stage of product development - New chapters deal with translational issues in the fastest growing population (the elderly), case studies, translatability assessment tools, and advances in nanotherapies - Details IPR issues of translation, especially for public-private-partnerships - Contains contributions from world leaders in translational medicine, including the former NIH director and authorities from various European regulatory institutions

Clinical and Translational Science: Principles of Human Research, Second Edition, is the most authoritative and timely resource for the broad range of investigators taking on the challenge of clinical and translational science, a field that is devoted to investigating human health and disease, interventions, and outcomes for the purposes of developing new treatment approaches, devices, and modalities to improve health. This updated second edition has been prepared with an international perspective, beginning with fundamental principles, experimental design, epidemiology, traditional and new biostatistical approaches, and investigative tools. It presents complete instruction and guidance from fundamental principles, approaches, and infrastructure, especially for human genetics and genomics, human pharmacology, research in special populations, the societal context of human research, and the future of human research. The book moves on to discuss legal, social, and ethical issues, and concludes with a discussion of future prospects, providing readers with a comprehensive view of this rapidly developing area of science. Introduces novel physiological and therapeutic strategies for engaging the fastest growing scientific field in both the private sector and academic medicine Brings insights from international leaders into the discipline of clinical and translational science Addresses drug discovery, drug repurposing and development, innovative and improved approaches to go/no-go decisions in drug development, and traditional and innovative clinical trial designs

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.

Biomarkers can be defined as indicators of any biologic state, and they are central to the future of medicine. As the cost of developing drugs has risen in recent years, reducing the number of new drugs approved for use, biomarker development may be a way to cut costs, enhance safety, and provide a more focused and rational pathway to drug development. On October 24, 2008, the IOM's Forum on Drug Discovery, Development, and Translation held "Assessing and Accelerating Development of Biomarkers for Drug Safety," a one-day workshop, summarized in this volume, on the value of biomarkers in helping to determine drug safety during development.

Basic Principles of Drug Discovery and Development presents the multifaceted process of identifying a new drug in the modern era, which requires a multidisciplinary team approach with input from medicinal chemists, biologists, pharmacologists, drug metabolism experts, toxicologists, clinicians, and a host of experts from numerous additional fields. Enabling technologies such as high throughput screening, structure-based drug design, molecular modeling, pharmaceutical profiling, and translational medicine are critical to the successful development of marketable therapeutics. Given the wide range of disciplines and techniques that are required for cutting edge drug discovery and development, a scientist must master their own fields as well as have a fundamental understanding of their collaborator's fields. This book bridges the knowledge gaps that invariably lead to communication issues in a new scientist's early career, providing a fundamental understanding of the various techniques and disciplines required for the multifaceted endeavor of drug research and development. It provides students, new industrial scientists, and academics with a basic understanding of the drug discovery and development process. The fully updated text provides an excellent overview of the process and includes chapters on important drug targets by class, in vitro screening methods, medicinal chemistry strategies in drug design, principles of in vivo pharmacokinetics and pharmacodynamics, animal models of disease states, clinical trial basics, and selected business aspects of the drug discovery process. - Provides a clear explanation of how the pharmaceutical industry works, as well as the complete drug discovery and development process, from obtaining a lead, to testing the bioactivity, to producing the drug, and protecting the intellectual property - Includes a new chapter on the discovery and development of biologics (antibodies proteins, antibody/receptor complexes, antibody drug conjugates), a growing and important area of the pharmaceutical industry landscape - Features a new section on formulations, including a discussion of IV formulations suitable for human clinical trials, as well as the application of nanotechnology and the use of transdermal patch technology for drug delivery - Updated chapter with new case studies includes additional modern examples of drug discovery through high through-put screening, fragment-based drug design, and computational chemistry