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

Despite spending more on care than any other industrialized nation--$2.4 trillion in 2008--the United States lags behind other countries on health measures, such as chronic disease burden. CER weighs the benefits and harms of 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.

An independent IOM committee developed a list of priority topics as part of a $1.1 billion effort to improve the quality and efficiency of healthcare through CER outlined in the American Recovery and Reinvestment Act (ARRA) of 2009. The committee's report provides guidance--informed by public input--to Congress and the secretary of the Health and Human Services (HHS) on how to spend $400 million on research to compare health services and approaches to care.

Within a three-week period, the committee received more than 2,606 nominations from 1,758 individual responders. Using a three-step voting process, the committee identified 100 high-priority topics.

"Healthcare decisions too often area matter of guesswork because we lack good evidence to inform them," said committee co-chair Harold C. Sox, editor of the Annals of Internal Medicine. "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."

Some of the committee's recommendations to establish the infrastructure for a successful CER initiative include the following:

• The HHS Secretary should establish a mechanism--such as a coordinating advisory body--with the authority to strategize, organize, monitor, evaluate and report on the implementation and impact of the CER Program.
• The CER Program should involve consumers, patients and their caregivers in aspects of CER, including strategic planning, priority setting, research proposal development, peer review and dissemination.
• The CER Program should devote resources to research and innovation in the methods of CER, including the development of methodological guidance for CER study design, such as the appropriate use of observational data and more informative, practical and efficient clinical trials.
• The CER Program should help to develop large-scale, clinical and administrative data networks.
• The CER Program should develop and support the workforce for CER to ensure U.S. capacity to carry out the CER mission. Next steps include: the development of a strategic plan for research workforce development; long-term funding for career development, including grants for graduate and postgraduate training opportunities in comparative effectiveness methods, as well as career development grants and mid-career merit awards.
• The CER Program should promote adoption of CER findings and conduct research to identify the effective strategies for disseminating new and existing CER findings to healthcare professionals, consumers and patients.

"Whether the CER initiative can realize its potential to improve health care depends on other features of healthcare reform legislation, the profession's ability to mobilize the best in its members, and the ability of individual patients to engage their physicians in a dialogue about their own care," Sox said. "We believe a national initiative that engages the medical profession and patients justifies a large public investment in CER."

In a corresponding perspective piece, Bryan R. Luce, PhD, MBA, and colleagues discussed approaches that could improve the government's CER initiative. They suggested methods for efficiently generating valid, generalizable evidence from randomized clinical trials (RCTs), as this kind of research is crucial to the health care decision making process.

According to the perspective authors, RCTs offer reliable information and must have a prominent place in the CER agenda. They recommended improving approaches to conducting RCTs so they are better suited to meet the needs of CER.

Study authors wrote that without changes in how researchers conceive, design, conduct, and analyze RCTs, the United States risks spending large sums of money inefficiently to answer the wrong questions through CER.