Rare Diseases and Orphan Products


Book Description

Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.




Non-Biological Complex Drugs


Book Description

The rise of bio- and nano-technology in the last decades has led to the emergence of a new and unique type of medicine known as non-biological complex drugs (NBCDs). This book illustrates the challenges associated with NBCD development, as well as the complexity of assessing the effects of manufacturing changes on innovator and follow-on batches of NBCDs. It also touches upon proven marketing authorization requirements for biosimilars that could be effective in evaluating follow-on NBCDs, including a demonstration of control over the manufacturing process and a need for detailed physico-chemical characterization and (pre)clinical tests. This book is meant to be used for years to come as a standard reference work for the development of NBCDs. Moreover, this book aims to stimulate discussions and further our thinking to ensure that decisions regarding the approval of complex drugs are made with relevant scientific data on the table.




Chiral Drugs


Book Description

An integrated view of chiral drugs from concept and synthesisto pharmaceutical properties Chirality greatly influences a drug's biological and pharmacological properties. In an effort to achieve more predictable results from chiral drugs, the Food and Drug Administration now requires that these medicines be as pure as possible, which places great demands on drug synthesis, purification, analysis, and testing. To assist researchers in acquiring the essential knowledge to meet these rigid guidelines, Chiral Drugs focuses on three vital chiral technologies asymmetric synthesis, biocatalytic process, and chiral resolution to offer details on the basic concepts, key developments, and recent trends in chiral drug discovery, along with: The history of chiral drugs development and industrial applications of chiral technologies A section listing twenty-five approved or advanced-trial chiral drugs that lists each drug name, chemical name and properties, a representative synthetic pathway, pharmacological characterizations, and references An interdisciplinary approach combining synthetic organic chemistry, medicinal chemistry, and pharmacology Nearly two-thirds of the drugs on today's market are chiral drugs. Reducing and eliminating their negative characteristics is an ongoing and serious challenge for the pharmaceutical industry. With its well-balanced approach to covering each important aspect of chirality, Chiral Drugs champions important strategies for tipping the medical scale in a positive direction for the production of more effective and safer drugs.




The Analysis of Drugs in Biological Fluids


Book Description

This new edition focuses on a variety of techniques available for the analysis of drugs in biological fluids. Over 150 figures and tables help to describe the latest advances and give examples of their applications. Current chiral analysis methods as well as discussions on the impact of chirality are described. Practical aspects of bioanalytical work, including many examples of laboratory problems not often reported in the scientific literature, are examined in depth.




Introduction to Biological and Small Molecule Drug Research and Development


Book Description

Introduction to Biological and Small Molecule Drug Research and Development provides, for the first time, an introduction to the science behind successful pharmaceutical research and development programs. The book explains basic principles, then compares and contrasts approaches to both biopharmaceuticals (proteins) and small molecule drugs, presenting an overview of the business and management issues of these approaches. The latter part of the book provides carefully selected real-life case studies illustrating how the theory presented in the first part of the book is actually put into practice. Studies include Herceptin/T-DM1, erythropoietin (Epogen/Eprex/NeoRecormon), anti-HIV protease inhibitor Darunavir, and more. Introduction to Biological and Small Molecule Drug Research and Development is intended for late-stage undergraduates or postgraduates studying chemistry (at the biology interface), biochemistry, medicine, pharmacy, medicine, or allied subjects. The book is also useful in a wide variety of science degree courses, in post-graduate taught material (Masters and PhD), and as basic background reading for scientists in the pharmaceutical industry. - For the first time, the fundamental scientific principles of biopharmaceuticals and small molecule chemotherapeutics are discussed side-by-side at a basic level - Edited by three senior scientists with over 100 years of experience in drug research who have compiled the best scientific comparison of small molecule and biopharmaceuticals approaches to new drugs - Illustrated with key examples of important drugs that exemplify the basic principles of pharmaceutical drug research and development







Biologics, A History of Agents Made From Living Organisms in the Twentieth Century


Book Description

The use of biologics – drugs made from living organisms – has raised specific scientific, industrial, medical and legal issues. The essays contained in this collection each deal with a case study of a biologic substance, or group of biologics, and its use during the twentieth century.




Biological Research on Addiction


Book Description

Biological Research on Addiction examines the neurobiological mechanisms of drug use and drug addiction, describing how the brain responds to addictive substances as well as how it is affected by drugs of abuse. The book's four main sections examine behavioral and molecular biology; neuroscience; genetics; and neuroimaging and neuropharmacology as they relate to the addictive process. This volume is especially effective in presenting current knowledge on the key neurobiological and genetic elements in an individual's susceptibility to drug dependence, as well as the processes by which some individuals proceed from casual drug use to drug dependence. Biological Research on Addiction is one of three volumes comprising the 2,500-page series, Comprehensive Addictive Behaviors and Disorders. This series provides the most complete collection of current knowledge on addictive behaviors and disorders to date. In short, it is the definitive reference work on addictions. - Each article provides glossary, full references, suggested readings, and a list of web resources - Edited and authored by the leaders in the field around the globe – the broadest, most expert coverage available - Discusses the genetic basis of addiction - Covers basic science research from a variety of animal studies




Biological Drugs


Book Description




Beyond the Molecular Frontier


Book Description

Chemistry and chemical engineering have changed significantly in the last decade. They have broadened their scopeâ€"into biology, nanotechnology, materials science, computation, and advanced methods of process systems engineering and controlâ€"so much that the programs in most chemistry and chemical engineering departments now barely resemble the classical notion of chemistry. Beyond the Molecular Frontier brings together research, discovery, and invention across the entire spectrum of the chemical sciencesâ€"from fundamental, molecular-level chemistry to large-scale chemical processing technology. This reflects the way the field has evolved, the synergy at universities between research and education in chemistry and chemical engineering, and the way chemists and chemical engineers work together in industry. The astonishing developments in science and engineering during the 20th century have made it possible to dream of new goals that might previously have been considered unthinkable. This book identifies the key opportunities and challenges for the chemical sciences, from basic research to societal needs and from terrorism defense to environmental protection, and it looks at the ways in which chemists and chemical engineers can work together to contribute to an improved future.