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.







Disease Control Priorities, Third Edition (Volume 6)


Book Description

Infectious diseases are the leading cause of death globally, particularly among children and young adults. The spread of new pathogens and the threat of antimicrobial resistance pose particular challenges in combating these diseases. Major Infectious Diseases identifies feasible, cost-effective packages of interventions and strategies across delivery platforms to prevent and treat HIV/AIDS, other sexually transmitted infections, tuberculosis, malaria, adult febrile illness, viral hepatitis, and neglected tropical diseases. The volume emphasizes the need to effectively address emerging antimicrobial resistance, strengthen health systems, and increase access to care. The attainable goals are to reduce incidence, develop innovative approaches, and optimize existing tools in resource-constrained settings.




Development of Therapeutic Agents Handbook


Book Description

A comprehensive look at current drug discovery and development methods—and the roadmap for the future Providing both understanding and guidance in characterizing potential drugs and their production and synthesis, Development of Therapeutic Agents Handbook gives professionals a basic tool to facilitate research and development within this challenging process. This comprehensive text brings together, in one resource, a compendium of concepts, approaches, methodologies, and limitations that need to be considered in the formulation of therapeutic agents across a range of therapeutic fields. Both a reference and a call to action for the pharmaceutical industry, Development of Therapeutic Agents Handbook examines recent innovations taking shape in the various medical disciplines involved in drug discovery, and shows why these advances need to be embraced universally among researchers to improve their solution strategies. Additional subject matter includes: Extensive coverage and in-depth look into novel treatments and therapeutics Discussion of hot topics like new drugs and nutraceuticals, the discovery and development of vaccines, cancer therapeutics, and market overviews Coverage of therapeutic drug development for specific disease areas, such as cardiology, oncology, breast cancer, and kidney diseases As research in biology, chemistry, medicine, and technology rapidly progresses, it is becoming increasingly important for medical researchers to maintain an up-to-date knowledge base of emerging trends directing promising new therapies. Development of Therapeutic Agents Handbook serves this purpose, acting as both a one-stop reference rich in valid science, and a tool to carve out new pathways in the pursuit of bringing safer and more effective drugs to the marketplace.




Field Trials of Health Interventions


Book Description

This is an open access title available under the terms of a CC BY-NC 4.0 International licence. It is free to read at Oxford Scholarship Online and offered as a free PDF download from OUP and selected open access locations. Before new interventions are released into disease control programmes, it is essential that they are carefully evaluated in field trials'. These may be complex and expensive undertakings, requiring the follow-up of hundreds, or thousands, of individuals, often for long periods. Descriptions of the detailed procedures and methods used in the trials that have been conducted have rarely been published. A consequence of this, individuals planning such trials have few guidelines available and little access to knowledge accumulated previously, other than their own. In this manual, practical issues in trial design and conduct are discussed fully and in sufficient detail, that Field Trials of Health Interventions may be used as a toolbox' by field investigators. It has been compiled by an international group of over 30 authors with direct experience in the design, conduct, and analysis of field trials in low and middle income countries and is based on their accumulated knowledge and experience. Available as an open access book via Oxford Medicine Online, this new edition is a comprehensive revision, incorporating the new developments that have taken place in recent years with respect to trials, including seven new chapters on subjects ranging from trial governance, and preliminary studies to pilot testing.




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.




Handbook of Pharmaceutical Salts Properties, Selection, and Use


Book Description

This comprehensive up-to-date guide and information source is an instructive companion for all scientists involved in research and development of drugs and, in particular, of pharmaceutical dosage forms. The editors have taken care to address every conceivable aspect of the preparation of pharmaceutical salts and present the necessary theoretical foundations as well as a wealth of detailed practical experience in the choice of pharmaceutically active salts. Altogether, the contributions reflect the multidisciplinary nature of the science involved in selection of suitable salt forms for new drug products.




Therapeutic Plasma Exchange


Book Description

This volume contains papers and discussions of the Vlth Dialyse-Arzte Workshop, which was held in Bernried at Lake Starnberg near Munich the 5th and 6th of March 1980. Generous ly sponsored by Travenol, Munich, the Dialyse-Arzte meetings now have a tradition spanning 16 years. According to the con stitution of these meetings, the topics of earlier years had to cover dialysis and related fields. Thus the sponsor requested that this year also one lecture - incorporated here as part - should deal with the state of art of dialysis, thereby hopefully linking this Workshop to the previous meetings. Dialysis techniques of the 1960s, pioneered by many of attend ing speakers and panelists (see List of Contributors), have never come to a standstill. Indeed, vascular access and extra corporeal circulation have become routine for the nephrologist and have made possible the introductimn of new approaches, such as hemofiltration and hemoperfusion. Also today new membrane technologies provide us with a potentially even more effective therapeutic tool, namely plasma separation.




Targeted Radionuclide Therapy


Book Description

Radioimmunotherapy, also known as systemic targeted radiation therapy, uses antibodies, antibody fragments, or compounds as carriers to guide radiation to the targets. It is a topic rapidly increasing in importance and success in treatment of cancer patients. This book represents a comprehensive amalgamation of the radiation physics, chemistry, radiobiology, tumor models, and clinical data for targeted radionuclide therapy. It outlines the current challenges and provides a glimpse at future directions. With significant advances in cell biology and molecular engineering, many targeting constructs are now available that will safely deliver these highly cytotoxic radionuclides in a targeted fashion. A companion website includes the full text and an image bank.