Financial Incentives to Encourage Development of Therapies That Address Unmet Medical Needs for Nervous System Disorders


Book Description

The Institute of Medicine (IOM) Forum on Neuroscience and Nervous System Disorders, in collaboration with the IOM Forum on Drug Discovery, Development, and Translation, convened a workshop on January 20-21, 2015, to explore policy changes that might increase private sector investment in research and development innovation that fills unmet medical needs for central nervous system (CNS) disorders. Workshop participants strategized about how to incentivize companies to fortify their CNS drug development programs, shrinking obstacles that currently deter ventures. Representatives from academia, government agencies, patient groups, and industry gathered to share information and viewpoints, and to brainstorm about budget-neutral policy changes that could help widen the pipeline toward drugs that address unmet needs for CNS disorders. This report summarizes the presentations and discussion of the workshop.




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.




Dying in America


Book Description

For patients and their loved ones, no care decisions are more profound than those made near the end of life. Unfortunately, the experience of dying in the United States is often characterized by fragmented care, inadequate treatment of distressing symptoms, frequent transitions among care settings, and enormous care responsibilities for families. According to this report, the current health care system of rendering more intensive services than are necessary and desired by patients, and the lack of coordination among programs increases risks to patients and creates avoidable burdens on them and their families. Dying in America is a study of the current state of health care for persons of all ages who are nearing the end of life. Death is not a strictly medical event. Ideally, health care for those nearing the end of life harmonizes with social, psychological, and spiritual support. All people with advanced illnesses who may be approaching the end of life are entitled to access to high-quality, compassionate, evidence-based care, consistent with their wishes. Dying in America evaluates strategies to integrate care into a person- and family-centered, team-based framework, and makes recommendations to create a system that coordinates care and supports and respects the choices of patients and their families. The findings and recommendations of this report will address the needs of patients and their families and assist policy makers, clinicians and their educational and credentialing bodies, leaders of health care delivery and financing organizations, researchers, public and private funders, religious and community leaders, advocates of better care, journalists, and the public to provide the best care possible for people nearing the end of life.




Patient Safety and Quality


Book Description

"Nurses play a vital role in improving the safety and quality of patient car -- not only in the hospital or ambulatory treatment facility, but also of community-based care and the care performed by family members. Nurses need know what proven techniques and interventions they can use to enhance patient outcomes. To address this need, the Agency for Healthcare Research and Quality (AHRQ), with additional funding from the Robert Wood Johnson Foundation, has prepared this comprehensive, 1,400-page, handbook for nurses on patient safety and quality -- Patient Safety and Quality: An Evidence-Based Handbook for Nurses. (AHRQ Publication No. 08-0043)." - online AHRQ blurb, http://www.ahrq.gov/qual/nurseshdbk/




Improving the Utility and Translation of Animal Models for Nervous System Disorders


Book Description

Nervous system diseases and disorders are highly prevalent and substantially contribute to the overall disease burden. Despite significant information provided by the use of animal models in the understanding of the biology of nervous system disorders and the development of therapeutics; limitations have also been identified. Treatment options that are high in efficacy and low in side effects are still lacking for many diseases and, in some cases are nonexistent. A particular problem in drug development is the high rate of attrition in Phase II and III clinical trials. Why do many therapeutics show promise in preclinical animal models but then fail to elicit predicted effects when tested in humans? On March 28 and 29, 2012, the Institute of Medicine Forum on Neuroscience and Nervous System Disorders convened the workshop "Improving Translation of Animal Models for Nervous System Disorders" to discuss potential opportunities for maximizing the translation of new therapies from animal models to clinical practice. The primary focus of the workshop was to examine mechanisms for increasing the efficiency of translational neuroscience research through discussions about how and when to use animal models most effectively and then best approaches for the interpretation of the data collected. Specifically, the workshop objectives were to: discuss key issues that contribute to poor translation of animal models in nervous system disorders, examine case studies that highlight successes and failures in the development and application of animal models, consider strategies to increase the scientific rigor of preclinical efficacy testing, explore the benefits and challenges to developing standardized animal and behavioral models. Improving the Utility and Translation of Animal Models for Nervous System Disorders: Workshop Summary also identifies methods to facilitate development of corresponding animal and clinical endpoints, indentifies methods that would maximize bidirectional translation between basic and clinical research and determines the next steps that will be critical for improvement of the development and testing of animal models of disorders of the nervous system.




Relieving Pain in America


Book Description

Chronic pain costs the nation up to $635 billion each year in medical treatment and lost productivity. The 2010 Patient Protection and Affordable Care Act required the Department of Health and Human Services (HHS) to enlist the Institute of Medicine (IOM) in examining pain as a public health problem. In this report, the IOM offers a blueprint for action in transforming prevention, care, education, and research, with the goal of providing relief for people with pain in America. To reach the vast multitude of people with various types of pain, the nation must adopt a population-level prevention and management strategy. The IOM recommends that HHS develop a comprehensive plan with specific goals, actions, and timeframes. Better data are needed to help shape efforts, especially on the groups of people currently underdiagnosed and undertreated, and the IOM encourages federal and state agencies and private organizations to accelerate the collection of data on pain incidence, prevalence, and treatments. Because pain varies from patient to patient, healthcare providers should increasingly aim at tailoring pain care to each person's experience, and self-management of pain should be promoted. In addition, because there are major gaps in knowledge about pain across health care and society alike, the IOM recommends that federal agencies and other stakeholders redesign education programs to bridge these gaps. Pain is a major driver for visits to physicians, a major reason for taking medications, a major cause of disability, and a key factor in quality of life and productivity. Given the burden of pain in human lives, dollars, and social consequences, relieving pain should be a national priority.




The Fourth Industrial Revolution


Book Description

World-renowned economist Klaus Schwab, Founder and Executive Chairman of the World Economic Forum, explains that we have an opportunity to shape the fourth industrial revolu­tion, which will fundamentally alter how we live and work. Schwab argues that this revolution is different in scale, scope and complexity from any that have come before. Characterized by a range of new technologies that are fusing the physical, digital and biological worlds, the developments are affecting all disciplines, economies, industries and governments, and even challenging ideas about what it means to be human. Artificial intelligence is already all around us, from supercomputers, drones and virtual assistants to 3D printing, DNA sequencing, smart thermostats, wear­able sensors and microchips smaller than a grain of sand. But this is just the beginning: nanomaterials 200 times stronger than steel and a million times thinner than a strand of hair and the first transplant of a 3D printed liver are already in development. Imagine “smart factories” in which global systems of manu­facturing are coordinated virtually, or implantable mobile phones made of biosynthetic materials. The fourth industrial revolution, says Schwab, is more significant, and its ramifications more profound, than in any prior period of human history. He outlines the key technologies driving this revolution and discusses the major impacts expected on government, business, civil society and individu­als. Schwab also offers bold ideas on how to harness these changes and shape a better future—one in which technology empowers people rather than replaces them; progress serves society rather than disrupts it; and in which innovators respect moral and ethical boundaries rather than cross them. We all have the opportunity to contribute to developing new frame­works that advance progress.




Investing in the Health and Well-Being of Young Adults


Book Description

Young adulthood - ages approximately 18 to 26 - is a critical period of development with long-lasting implications for a person's economic security, health and well-being. Young adults are key contributors to the nation's workforce and military services and, since many are parents, to the healthy development of the next generation. Although 'millennials' have received attention in the popular media in recent years, young adults are too rarely treated as a distinct population in policy, programs, and research. Instead, they are often grouped with adolescents or, more often, with all adults. Currently, the nation is experiencing economic restructuring, widening inequality, a rapidly rising ratio of older adults, and an increasingly diverse population. The possible transformative effects of these features make focus on young adults especially important. A systematic approach to understanding and responding to the unique circumstances and needs of today's young adults can help to pave the way to a more productive and equitable tomorrow for young adults in particular and our society at large. Investing in The Health and Well-Being of Young Adults describes what is meant by the term young adulthood, who young adults are, what they are doing, and what they need. This study recommends actions that nonprofit programs and federal, state, and local agencies can take to help young adults make a successful transition from adolescence to adulthood. According to this report, young adults should be considered as a separate group from adolescents and older adults. Investing in The Health and Well-Being of Young Adults makes the case that increased efforts to improve high school and college graduate rates and education and workforce development systems that are more closely tied to high-demand economic sectors will help this age group achieve greater opportunity and success. The report also discusses the health status of young adults and makes recommendations to develop evidence-based practices for young adults for medical and behavioral health, including preventions. What happens during the young adult years has profound implications for the rest of the life course, and the stability and progress of society at large depends on how any cohort of young adults fares as a whole. Investing in The Health and Well-Being of Young Adults will provide a roadmap to improving outcomes for this age group as they transition from adolescence to adulthood.




Reducing Suicide


Book Description

Every year, about 30,000 people die by suicide in the U.S., and some 650,000 receive emergency treatment after a suicide attempt. Often, those most at risk are the least able to access professional help. Reducing Suicide provides a blueprint for addressing this tragic and costly problem: how we can build an appropriate infrastructure, conduct needed research, and improve our ability to recognize suicide risk and effectively intervene. Rich in data, the book also strikes an intensely personal chord, featuring compelling quotes about people's experience with suicide. The book explores the factors that raise a person's risk of suicide: psychological and biological factors including substance abuse, the link between childhood trauma and later suicide, and the impact of family life, economic status, religion, and other social and cultural conditions. The authors review the effectiveness of existing interventions, including mental health practitioners' ability to assess suicide risk among patients. They present lessons learned from the Air Force suicide prevention program and other prevention initiatives. And they identify barriers to effective research and treatment. This new volume will be of special interest to policy makers, administrators, researchers, practitioners, and journalists working in the field of mental health.




Disease Control Priorities, Third Edition (Volume 4)


Book Description

Mental, neurological, and substance use disorders are common, highly disabling, and associated with significant premature mortality. The impact of these disorders on the social and economic well-being of individuals, families, and societies is large, growing, and underestimated. Despite this burden, these disorders have been systematically neglected, particularly in low- and middle-income countries, with pitifully small contributions to scaling up cost-effective prevention and treatment strategies. Systematically compiling the substantial existing knowledge to address this inequity is the central goal of this volume. This evidence-base can help policy makers in resource-constrained settings as they prioritize programs and interventions to address these disorders.