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.

To explore the role of the National Institutes of Health (NIH) in innovative drug development and its impact on patient access, the Board on Health Care Services and the Board on Health Sciences Policy of the National Academies jointly hosted a public workshop on July 24â€"25, 2019, in Washington, DC. Workshop speakers and participants discussed the ways in which federal investments in biomedical research are translated into innovative therapies and considered approaches to ensure that the public has affordable access to the resulting new drugs. This publication summarizes the presentations and discussions from the workshop.

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.

Systemic lupus erythematosus (SLE), commonly called lupus, is a chronic autoimmune disorder that can affect virtually any organ of the body. In lupus, the body's immune system, which normally functions to protect against foreign invaders, becomes hyperactive, forming antibodies that attack normal tissues and organs, including the skin, joints, kidneys, brain, heart, lungs, and blood. Lupus is characterized by periods of illness, called flares, and periods of wellness or remission. Because its symptoms come and go and mimic those of other diseases, lupus is difficult to diagnose. There is no single laboratory test that can definitively prove that a person has the complex illness. To date, lupus has no known cause or cure. Early detection and treatment are the key to a better health outcome and can usually lessen the progression and severity of the disease. Anti-inflammatory drugs, antimalarials, and steroids (such as cortisone and others) are often used to treat lupus. Cytotoxic chemotherapies, like those used in the treatment of cancer, are also used to suppress the immune system in lupus patients. A new edition of this established and well-regarded reference combines basic science with clinical science to provide a translational medicine model. Systemic Lupus Erythematosus, Sixth Edition, is a useful reference for specialists in the diagnosis and management of patients with SLE, a tool for measurement of clinical activity for pharmaceutical development and basic research of the disease, and a reference work for hospital libraries. Completely updated, revised, and expanded with the most comprehensive and accessible reference on SLE for clinicians and scientists Full-color presentation throughout the book Provides the latest information available on diagnosis and treatment Incorporates an international panel of authors who are experts in their fields, with an emphasis on young, cutting-edge scientists and physicians