A new report from the Institute of Medicine recommends (IOM) 100 health topics that should get priority attention and funding from a new national research effort to identify which health care services work best. It also spells out actions and resources needed to ensure that this comparative effectiveness research initiative will be a sustained effort with a continuous process for updating priorities as needed and that the results are put into clinical practice.

A committee convened by the IOM developed the list of priority topics at the request of Congress as part of a $1.1 billion effort to improve the quality and efficiency of health care through comparative effectiveness research outlined in the American Recovery and Reinvestment Act of 2009. The committee’s report provides independent guidance — informed by extensive public input — to Congress and the secretary of the US Department of Health and Human Services on how to spend $400 million on research to compare health services and approaches to care.

Health experts and policymakers anticipate that comparative effectiveness research will yield greater value from America’s health care system and better outcomes for patients. Despite spending more on care than any other industrialized nation — $2.4 trillion in 2008 — the US lags behind other countries on many measures of health, such as infant mortality and chronic disease burden. Comparative effectiveness research weighs the benefits and harms of various ways to prevent, diagnose, treat, or monitor clinical conditions to determine which work best for particular types of patients and in different settings and circumstances. Study results can help consumers, clinicians, policymakers, and purchasers make more informed decisions, ultimately improving care for individuals and groups.

Health care decisions too often area matter of guesswork because we lack good evidence to inform them, said committee co-chair Harold C. Sox, editor, Annals of Internal Medicine, American College of Physicians of Internal Medicine, Philadelphia. For example, we spend a great deal on diagnostic tests for coronary heart disease in this country, but we lack sufficient evidence to determine which test is best.

This report lays the foundation for an ongoing enterprise to provide the evidence that health care providers need to make better decisions and achieve better results, added co-chair Sheldon Greenfield, Donald Bren Professor of Medicine and executive director, Health Policy Research Institute, University of California, Irvine. To make the most of this enterprise, HHS will need to ensure that the results are translated into practice and that the public is involved in priority setting to ensure that the research is relevant to everyday health care.

The 100 priority areas reflect the insights of health professionals, consumer advocates, policy analysts, and others who submitted nominations through an online form that was open to any individual or organization and through presentations at public meetings. The committee received 1,268 unique topic suggestions, which it narrowed to 100 based on a set of criteria that included its charge to develop a balanced portfolio. The list reflects a range of clinical categories, populations to be studied, categories of interventions, and research methodologies. The committee developed its list of priorities independent from the comparative effectiveness research activities that other organizations have been charged to do through the American Recovery and Reinvestment Act.

The report also recommends actions necessary to establish an ongoing comparative effectiveness research effort that would not only carry out studies on the 100 recommended initial topics, but also develop priorities for future research and translate the knowledge gained into improvements in clinical care. Effective coordination and governance among the agencies and disciplines involved will be crucial for ensuring the sustainability of the enterprise, the report notes. Moreover, a skilled work force is needed to carry out the research. The committee underscored the importance of having patients, families, and their caregivers actively engaged in identifying research topics of most concern to them. Ultimately, comparative effectiveness research will fall short of its potential without vigorous efforts by HHS to promote adoption of the findings by health care providers and organizations, the committee added.

The study was sponsored by the US Department of Health and Human Services. Established in 1970 under the charter of the National Academy of Sciences, the Institute of Medicine provides independent, objective, evidence-based advice to policymakers, health professionals, the private sector, and the public. The National Academy of Sciences, National Academy of Engineering, Institute of Medicine, and National Research Council make up the National Academies.