In the past decade there has been a worldwide evolution in evidence-based medicine that focuses on real-world Comparative Effectiveness Research (CER) to compare the effects of one medical treatment versus another in real world settings. While most of this burgeoning literature has focused on research findings, data and methods, Howard Birnbaum and Paul Greenberg (both of Analysis Group) have edited a book that provides a practical guide to decision making using the results of analysis and interpretation of CER. Decision Making in a World of Comparative Effectiveness contains chapters by senior industry executives, key opinion leaders, accomplished researchers, and leading attorneys involved in resolving disputes in the life sciences industry. The book is aimed at 'users' and 'decision makers' involved in the life sciences industry rather than those doing the actual research. This book appeals to those who commission CER within the life sciences industry (pharmaceutical, biologic, and device manufacturers), government (both public and private payers), as well as decision makers of all levels, both in the US and globally.

Clinical research presents health care providers with information on the natural history and clinical presentations of disease as well as diagnostic and treatment options. In today's healthcare system, patients, physicians, clinicians and family caregivers often lack the sufficient scientific data and evidence they need to determine the best course of treatment for the patients' medical conditions. Initial National Priorities for Comparative Effectiveness Research(CER) is designed to fill this knowledge gap by assisting patients and healthcare providers across diverse settings in making more informed decisions. In this 2009 report, the Institute of Medicine's Committee on Comparative Effectiveness Research Prioritization establishes a working definition of CER, develops a priority list of research topics, and identifies the necessary requirements to support a robust and sustainable CER enterprise. As part of the 2009 American Recovery and Reinvestment Act, Congress appropriated $1.1 billion in federal support of CER, reflecting legislators' belief that better decisions about the use of health care could improve the public's health and reduce the cost of care. The Committee on Comparative Effectiveness Research Prioritization was successful in preparing a list 100 top priority CER topics and 10 recommendations for best practices in the field.

Healthcare decision makers in search of reliable information that compares health interventions increasingly turn to systematic reviews for the best summary of the evidence. Systematic reviews identify, select, assess, and synthesize the findings of similar but separate studies, and can help clarify what is known and not known about the potential benefits and harms of drugs, devices, and other healthcare services. Systematic reviews can be helpful for clinicians who want to integrate research findings into their daily practices, for patients to make well-informed choices about their own care, for professional medical societies and other organizations that develop clinical practice guidelines. Too often systematic reviews are of uncertain or poor quality. There are no universally accepted standards for developing systematic reviews leading to variability in how conflicts of interest and biases are handled, how evidence is appraised, and the overall scientific rigor of the process. In Finding What Works in Health Care the Institute of Medicine (IOM) recommends 21 standards for developing high-quality systematic reviews of comparative effectiveness research. The standards address the entire systematic review process from the initial steps of formulating the topic and building the review team to producing a detailed final report that synthesizes what the evidence shows and where knowledge gaps remain. Finding What Works in Health Care also proposes a framework for improving the quality of the science underpinning systematic reviews. This book will serve as a vital resource for both sponsors and producers of systematic reviews of comparative effectiveness research.

In the past decade there has been a worldwide evolution in evidence-based medicine that focuses on real-world Comparative Effectiveness Research (CER) to compare the effects of one medical treatment versus another in real world settings. While most of this burgeoning literature has focused on research findings, data and methods, Howard Birnbaum and Paul Greenberg (both of Analysis Group) have edited a book that provides a practical guide to decision making using the results of analysis and interpretation of CER. Decision Making in a World of Comparative Effectiveness contains chapters by senior industry executives, key opinion leaders, accomplished researchers, and leading attorneys involved in resolving disputes in the life sciences industry. The book is aimed at 'users' and 'decision makers' involved in the life sciences industry rather than those doing the actual research. This book appeals to those who commission CER within the life sciences industry (pharmaceutical, biologic, and device manufacturers), government (both public and private payers), as well as decision makers of all levels, both in the US and globally.

This User’s Guide is a resource for investigators and stakeholders who develop and review observational comparative effectiveness research protocols. It explains how to (1) identify key considerations and best practices for research design; (2) build a protocol based on these standards and best practices; and (3) judge the adequacy and completeness of a protocol. Eleven chapters cover all aspects of research design, including: developing study objectives, defining and refining study questions, addressing the heterogeneity of treatment effect, characterizing exposure, selecting a comparator, defining and measuring outcomes, and identifying optimal data sources. Checklists of guidance and key considerations for protocols are provided at the end of each chapter. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews. More more information, please consult the Agency website: www.effectivehealthcare.ahrq.gov)

Dieses Lehrbuch, ein wegweisender Klassiker, bietet in der 6. Auflage noch mehr Inhalte für Leser, die aktuelle Informationen zur Pharmakoepidemiologie benötigen. Die vorliegende Auflage wurde vollständig überarbeitet und aktualisiert. Sie bietet einen Überblick über sämtliche Facetten des Fachgebiets, aus Sicht von Lehre und Forschung, aus Sicht der Industrie und von Regulierungsbehörden. Datenquellen, Anwendungen und Methodiken werden verständlich erläutert.

Comparative Effectiveness Research: Evidence, Medicine, and Policy provides the first complete account of how — and why — the federal government decided to make comparative effectiveness research (CER) an important feature of health reform and the Affordable Care Act of 2010.

Randomized controlled trials (RCTs) have traditionally served as the gold standard for generating evidence about medical interventions. However, RCTs have inherent limitations and may not reflect the use of medical products in the real world. Additionally, RCTs are expensive, time consuming, and cannot answer all questions about a product or intervention. Evidence generated from real-world use, such as real-world evidence (RWE) may provide valuable information, alongside RCTs, to inform medical product decision making. To explore the potential for using RWE in medical product decision making, the National Academies of Sciences, Engineering, and Medicine planned a three-part workshop series. The series was designed to examine the current system of evidence generation and its limitations, to identify when and why RWE may be an appropriate type of evidence on which to base decisions, to learn from successful initiatives that have incorporated RWE, and to describe barriers that prevent RWE from being used to its full potential. This publication summarizes the discussions from the entire workshop series.

This User’s Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase understanding of patient outcomes. For the purposes of this guide, a patient registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes. A registry database is a file (or files) derived from the registry. Although registries can serve many purposes, this guide focuses on registries created for one or more of the following purposes: to describe the natural history of disease, to determine clinical effectiveness or cost-effectiveness of health care products and services, to measure or monitor safety and harm, and/or to measure quality of care. Registries are classified according to how their populations are defined. For example, product registries include patients who have been exposed to biopharmaceutical products or medical devices. Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization. Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews.

Integrates the various disciplines of the science of health disparities in one comprehensive volume The Science of Health Disparities Research is an indispensable source of up-to-date information on clinical and translational health disparities science. Building upon the advances in health disparities research over the past decade, this authoritative volume informs policies and practices addressing the diseases, disorders, and gaps in health outcomes that are more prevalent in minority populations and socially disadvantaged communities. Contributions by recognized scholars and leaders in the field—featuring contemporary research, conceptual models, and a broad range of scientific perspectives—provide an interdisciplinary approach to reducing inequalities in population health, encouraging community engagement in the research process, and promoting social justice. In-depth chapters help readers better understand the specifics of minority health and health disparities while demonstrating the importance of advancing theory, refining measurement, improving investigative methods, and diversifying scientific research. In 26 chapters, the book examines topics including the etiology of health disparities research, the determinants of population health, research ethics, and research in African American, Asians, Latino, American Indian, and other vulnerable populations. Providing a unified framework on the principles and applications of the science of health disparities research, this important volume: Defines the field of health disparities science and suggests new directions in scholarship and research Explains basic definitions, principles, and concepts for identifying, understanding and addressing health disparities Provides guidance on both conducting health disparities research and translating the results Examines how social, historical and contemporary injustices may influence the health of racial and ethnic minorities Illustrates the increasing national and global importance of addressing health disparities Discusses population health training, capacity-building, and the transdisciplinary tools needed to advance health equity A significant contribution to the field, The Science of Health Disparities Research is an essential resource for students and basic and clinical researchers in genetics, population genetics, and public health, health care policymakers, and epidemiologists, medical students, and clinicians, particularly those working with minority, vulnerable, or underserved populations.