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AJN, American Journal of Nursing:
doi: 10.1097/01.NAJ.0000361481.68858.c4
AJN Reports

Comparative Effectiveness Research

Stubenrauch, James M. AJN senior editor

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It could change the way health care decisions are made.

Improve health, reduce costs: that's the mantra health care reform advocates keep repeating. And it's easy to see why: this year, total health care spending in the United States is expected to reach $2.5 trillion, accounting for almost 18% of the gross domestic product.1 By 2018 the total could be $4.4 trillion—and because economic growth is expected to be slower over this period, that total may account for one-fifth of the gross domestic product in 2018. And even with all of this spending, the United States lags behind other industrialized nations on many measures of health and well being.

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While there are many paths to achieving the twin goals of better outcomes and lower costs, a consensus has been growing among health policy experts and economists that part of the solution is to improve the way medical research is conducted and then put it into practice in both providers' and consumers' decision making. Comparative effectiveness research (CER)—a model by which cost–benefit analyses of different treatments for a given condition are compared—provides the means for understanding which interventions yield the best health outcomes for the least amount of money.

President Obama's economic stimulus legislation—the American Recovery and Reinvestment Act of 2009—allots $1.1 billion over 10 years to improve the quality and efficiency of U.S. health care through CER.2 The law also created a Federal Coordinating Council on CER and directed the Institute of Medicine (IOM) to develop a list of high-priority topics for this research. The IOM prioritization committee sought input from a broad array of stakeholders, including medical researchers, clinicians, professional and advocacy organizations, and the public and, in June, issued a report that lists the top 100 research priorities (see The priorities are divided into four quartiles, each comprising 25 research topics; within each quartile, topics are not ranked in order of importance.

The following are examples of the goals of research programs in the first quartile—the highest-priority group:

* Compare the effectiveness of primary prevention methods, such as exercise and balance training, with clinical treatments in preventing falls in older adults at varying degrees of risk.

* Compare the effectiveness of various screening, prophylactic, and treatment interventions in eradicating methicillin-resistant Staphylococcus aureus (MRSA) in communities, institutions, and hospitals.

* Compare the effectiveness of comprehensive care coordination programs, such as the medical home, with usual care in managing children and adults with severe chronic disease, especially in populations with known health disparities.

The committee also said that "The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels."

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But why is it necessary, you may ask, for the government to encourage this kind of research? After all, aren't most medical treatments based on evidence and an understanding of the cost-effectiveness of different approaches?

Not really. Here's how Princeton economics professor Uwe E. Reinhardt, whose Economix blog appears on the Web site of the New York Times, puts it: "In principle, the clinical practice guidelines promulgated by medical specialty societies to help physicians with their daily treatment decisions should be based on this type of carefully structured comparative effectiveness research. Alas, in practice most of the currently promulgated guidelines lack that kind of rigorous scientific foundation."3

For example, a study published in JAMA found that almost 90% of more than 2,700 recommendations contained in cardiovascular practice guidelines jointly issued by the American College of Cardiologists and the American Heart Association were not supported by high-quality scientific evidence.4

Writing in the Annals of Internal Medicine, Harold C. Sox, MD, and Sheldon Greenfield, MD, cochairmen of the CER prioritization committee, noted that2

Somehow, and in concert with national professional organizations and local opinion leaders, a national program should promote the professional ethos that places the interests of patients and the larger community above all other considerations.

Studying research methods. One of the main aims of CER is to improve the applicability of research to real-world situations. To do this, it's important to begin a renewed examination of the methodology of clinical research. Sox and Greenfield note that, although the two main forms of clinical research—observational research and randomized trials—have great strengths, they also have limitations.2 For example, observational studies that rely on large databases are constrained by the kinds of data available; characteristics of the patient that may influence outcomes as much as treatment may be missing. Likewise, randomized trials often exclude classes of patients that must be treated in the real world and compare the treatment against placebo, rather than testing it against another treatment.

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Despite the clear need for CER and its obvious benefits, it has recently become a matter of heated debate within the struggle over health care reform. Just as some opponents of current reform plans have misrepresented a proposal to fund optional end-of-life counseling for Medicare patients as "death panels" that would "pull the plug on Grandma," they have also tried to raise fears that CER could be used to deny specific treatments to deserving patients and to "ration" health care. Senators Jon Kyl (R-AZ), Mitch McConnell (R-KY), and Pat Roberts (R-KS) introduced the "Preserving Access to Targeted, Individualized, and Effective New Treatments and Services (PATIENTS) Act of 2009," a bill that would prohibit Medicare and Medicaid from using CER to "deny coverage."5, 6 Also, medical device and pharmaceutical manufacturers, who lobbied against the inclusion of funding for CER in the stimulus bill, have been big contributors to these senators' election campaigns.

In an e-mail interview, AJN asked Patricia A. Grady, PhD, RN, FAAN, director of the National Institute for Nursing Research, whether she thought there was any real danger that CER could be misused to deny health care to Americans. "Comparative effectiveness research has garnered increased attention as an area of science that can have a significant impact upon the future health care of the American people," Grady replied. "The purpose of such research is to determine appropriate health care, not to deny it."

James M. Stubenrauch

AJN senior editor

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1. Sisko A, et al. Health spending projections through 2018: recession effects add uncertainty to the outlook. Health Aff (Millwood) 2009;28(2):w346–w57.

2. Sox HC, Greenfield S. Comparative effectiveness research: a report from the institute of medicine. Ann Intern Med 2009;151(3):203–5.

3. Reinhardt UE. 'Cost-effectiveness analysis' and U.S. health care. New York: The New York Times 2009.

4. Tricoci P, et al. Scientific evidence underlying the ACC/AHA clinical practice guidelines. JAMA 2009;301(8):831–41.

5. Krugman P. Taking the hypocritical oath. New York: The New York Times 2009.

6. Volsky I. GOP introduces bill to derail comparative effectiveness research. Washington, DC: Center for American Progress Action Fund 2009.

© 2009 Lippincott Williams & Wilkins, Inc.