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.

Thanks to remarkable advances in modern health care attributable to science, engineering, and medicine, it is now possible to cure or manage illnesses that were long deemed untreatable. At the same time, however, the United States is facing the vexing challenge of a seemingly uncontrolled rise in the cost of health care. Total medical expenditures are rapidly approaching 20 percent of the gross domestic product and are crowding out other priorities of national importance. The use of increasingly expensive prescription drugs is a significant part of this problem, making the cost of biopharmaceuticals a serious national concern with broad political implications. Especially with the highly visible and very large price increases for prescription drugs that have occurred in recent years, finding a way to make prescription medicinesâ€"and health care at largeâ€"more affordable for everyone has become a socioeconomic imperative. Affordability is a complex function of factors, including not just the prices of the drugs themselves, but also the details of an individual's insurance coverage and the number of medical conditions that an individual or family confronts. Therefore, any solution to the affordability issue will require considering all of these factors together. The current high and increasing costs of prescription drugsâ€"coupled with the broader trends in overall health care costsâ€"is unsustainable to society as a whole. Making Medicines Affordable examines patient access to affordable and effective therapies, with emphasis on drug pricing, inflation in the cost of drugs, and insurance design. This report explores structural and policy factors influencing drug pricing, drug access programs, the emerging role of comparative effectiveness assessments in payment policies, changing finances of medical practice with regard to drug costs and reimbursement, and measures to prevent drug shortages and foster continued innovation in drug development. It makes recommendations for policy actions that could address drug price trends, improve patient access to affordable and effective treatments, and encourage innovations that address significant needs in health care.

Vaccinate children against deadly pneumococcal disease, or pay for cardiac patients to undergo lifesaving surgery? Cover the costs of dialysis for kidney patients, or channel the money toward preventing the conditions that lead to renal failure in the first place? Policymakers dealing with the realities of limited health care budgets face tough decisions like these regularly. And for many individuals, their personal health care choices are equally stark: paying for medical treatment could push them into poverty. Many low- and middle-income countries now aspire to universal health coverage, where governments ensure that all people have access to the quality health services they need without risk of impoverishment. But for universal health coverage to become reality, the health services offered must be consistent with the funds available—and this implies tough everyday choices for policymakers that could be the difference between life and death for those affected by any given condition or disease. The situation is particularly acute in low- and middle income countries where public spending on health is on the rise but still extremely low, and where demand for expanded services is growing rapidly. What’s In, What’s Out: Designing Benefits for Universal Health Coverage argues that the creation of an explicit health benefits plan—a defined list of services that are and are not available—is an essential element in creating a sustainable system of universal health coverage. With contributions from leading health economists and policy experts, the book considers the many dimensions of governance, institutions, methods, political economy, and ethics that are needed to decide what’s in and what’s out in a way that is fair, evidence-based, and sustainable over time.

Since 1972, many victims of endstage renal disease (ESRD) have received treatment under a unique Medicare entitlement. This book presents a comprehensive analysis of the federal ESRD program: who uses it, how well it functions, and what improvements are needed. The book includes recommendations on patient eligibility, reimbursement, quality assessment, medical ethics, and research needs. Kidney Failure and the Federal Government offers a wealth of information on these and other topics: The ESRD patient population. Dialysis and transplantation providers. Issues of patient access and availability of treatment. Ethical issues related to treatment initiation and termination. Payment policies and their relationship to quality of care. This book will have a major impact on the future of the ESRD program and will be of interest to health policymakers, nephrologists and other individual providers, treatment site administrators, and researchers.

This report marks the first of what is expected to be a series of assessments of various global health problems, and this first effort begins with the conceptualization of health as a global issue. It explores the relationship between health and economic growth, trade, innovation, global security and global governance. It focuses in particular on infectious diseases as a significant global health challenge, and looks to the origin, causes, and effectiveness of various interventions employedfor different epidemics. In evaluating the global response to pandemics, it looks at each in terms of the viability and effectiveness of regional and cross-border collaboration to deploy health care systems, surveillance, lab testing, communication, and human resources and equipment.