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.

New Drug Development: Second Edition provides an overview of the design concepts and statistical practices involved in therapeutic drug development. This wide spectrum of activities begins with identifying a potentially useful drug candidate that can perhaps be used in the treatment or prevention of a condition of clinical concern, and ends with marketing approval being granted by one or more regulatory agencies. In between, it includes drug molecule optimization, nonclinical and clinical evaluations of the drug’s safety and efficacy profiles, and manufacturing considerations. The more inclusive term lifecycle drug development can be used to encompass the postmarketing surveillance that is conducted all the time that a drug is on the market and being prescribed to patients with the relevant clinical condition. Information gathered during this time can be used to modify the drug (for example, dose prescribed, formulation, and mode of administration) in terms of its safety and its effectiveness. The central focus of the first edition of this book is captured by its subtitle, 'Design, Methodology, and Analysis'. Optimum quality study design and experimental research methodology must be employed if the data collected—numerical representations of biological information—are to be of optimum quality. Optimum quality data facilitate optimum quality statistical analysis and interpretation of the results obtained, which in turn permit optimum quality decisions to be made: Rational decision making is predicated on appropriate research questions and optimum quality numerical information. The book took a non-computational approach to statistics, presenting instead a conceptual framework and providing readers with a sound working knowledge of the importance of design, methodology, and analysis. Not everyone needs to be an expert in statistical analysis, but it is very helpful for work (or aspire to work) in the pharmaceutical and biologics industries to be aware of the fundamental importance of a sound scientific and clinical approach to the planning, conduct, and analysis of clinical trials.

The very rapid pace of advances in biomedical research promises us a wide range of new drugs, medical devices, and clinical procedures. The extent to which these discoveries will benefit the public, however, depends in large part on the methods we choose for developing and testing them. Modern Methods of Clinical Investigation focuses on strategies for clinical evaluation and their role in uncovering the actual benefits and risks of medical innovation. Essays explore differences in our current systems for evaluating drugs, medical devices, and clinical procedures; health insurance databases as a tool for assessing treatment outcomes; the role of the medical profession, the Food and Drug Administration, and industry in stimulating the use of evaluative methods; and more. This book will be of special interest to policymakers, regulators, executives in the medical industry, clinical researchers, and physicians.

In an effort to increase knowledge and understanding of the process of assuring data quality and validity in clinical trials, the IOM hosted a workshop to open a dialogue on the process to identify and discuss issues of mutual concern among industry, regulators, payers, and consumers. The presenters and panelists together developed strategies that could be used to address the issues that were identified. This IOM report of the workshop summarizes the present status and highlights possible strategies for making improvements to the education of interested and affected parties as well as facilitating future planning.

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.

A Practical Guide to Managing Clinical Trials is a basic, comprehensive guide to conducting clinical trials. Designed for individuals working in research site operations, this user-friendly reference guides the reader through each step of the clinical trial process from site selection, to site set-up, subject recruitment, study visits, and to study close-out. Topics include staff roles/responsibilities/training, budget and contract review and management, subject study visits, data and document management, event reporting, research ethics, audits and inspections, consent processes, IRB, FDA regulations, and good clinical practices. Each chapter concludes with a review of key points and knowledge application. Unique to this book is "A View from India," a chapter-by-chapter comparison of clinical trial practices in India versus the U.S. Throughout the book and in Chapter 10, readers will glimpse some of the challenges and opportunities in the emerging and growing market of Indian clinical trials.