Observational Studies in a Learning Health System: Workshop Summary

by Institute of Medicine, Roundtable on Value and Science-Driven Health Care, A Learning Health System Activity

Clinical research strains to keep up with the rapid and iterative evolution of medical interventions, clinical practice innovation, and the increasing demand for information on the clinical effectiveness of these advancements. In response to the growing availability of archived and real-time digital health data and the opportunities this data provides for research, as well as the increasing number of studies using prospectively collected clinical data, the Institute of Medicine\'s Roundtable on Value & Science-Driven Health Care convened a workshop on Observational Studies in a Learning Health System. Participants, including experts from a wide range of disciplines - clinical researchers, statisticians, biostatisticians, epidemiologists, health care informaticians, health care analytics, research funders, health products industry, clinicians, payers, and regulators - explored leading edge approaches to observational studies, charted a course for the use of the growing health data utility, and identified opportunities to advance progress. Workshop speakers and individual participants strove to identify stakeholder needs and barriers to the broader application of observational studies. Observational Studies in a Learning Health System is the summary of the workshop. This report explores the role of observational studies in the generation of evidence to guide clinical and health policy decisions. The report discusses concepts of rigorous observational study design and analysis, emerging statistical methods, and opportunities and challenges of observational studies to complement evidence from experimental methods, treatment heterogeneity, and effectiveness estimates tailored toward individual patients.

134 pages, Paperback

First published December 16, 2013

About the author

The National Academy of Medicine (NAM, formerly the Institute of Medicine-IOM) is an American nonprofit, non-governmental organization. The National Academy of Medicine is a part of the National Academies of Sciences, Engineering, and Medicine, along with the National Academy of Sciences (NAS), National Academy of Engineering (NAE), and the National Research Council (NRC).

The National Academy of Medicine provides national advice on issues relating to biomedical science, medicine, and health, and serves as an adviser to the nation to improve health aims to provide unbiased, evidence-based, and authoritative information and advice concerning health and science policy to policy-makers, professionals, leaders in every sector of society, and the public at large.

Operating outside the framework of the U.S. federal government, it relies on a volunteer workforce of scientists and other experts, operating under a formal peer-review system. As a national academy, new members of the organization are elected annually by current members, based on their distinguished and continuing achievements in a relevant field as well as for their willingness to participate actively.