Health Care Reform Center

John K. Iglehart

Directed by Congress to rapidly develop a list of broad-based priorities for the Department of Health and Human Services (DHHS) to consider as it implements a new agenda for comparative-effectiveness research (CER), the Institute of Medicine (IOM) released a report recommending a portfolio of 100 study topics related to a range of diseases, research methods, and care models that are important to the health of the U.S. population.

The IOM report,¹ released June 30, also recommends research studies on rare diseases that disproportionately affect certain subgroups of the population, such as women, racial or ethnic minorities, and particular age groups. The panel concluded that “the most important priority of all should be the building of a broad and supportive infrastructure to carry out a sustainable national CER strategy” and that Congress and the secretary of health and human services “must take concerted steps to establish a robust CER enterprise.”

The IOM Committee on Comparative Effectiveness Research Prioritization selected its 100 topics after obtaining extensive input from professional organizations and the public, as required by the American Recovery and Reinvestment Act of 2009 (ARRA). Given the public criticism the research initiative has received, the IOM report noted that “Engaging consumers in CER . . . could help improve the public’s trust in the U.S. research enterprise.” The committee began with 1268 CER topics that were nominated by stakeholders and the public and winnowed them down to 82; the other 18 topics were recommended by the committee to fill gaps in the portfolio.

The ARRA, which is the $787 billion economic stimulus package that President Barack Obama signed into law on February 17, 2009, included $1.1 billion for CER. The research priorities developed by the IOM committee — delivered as Congress requested only 19 weeks after Obama signed the measure — must be taken into account by the DHHS as it allocates $400 million in support of CER projects over the next 2 years. (A Federal Coordinating Council for Comparative Effectiveness Research, a new advisory group created by the ARRA, is also providing input to the DHHS [ http://hhs.gov/recovery/programs/cer/cerannualrpt.pdf ].) Of the $700 million appropriated in the law for CER, Congress allocated $400 million to the National Institutes of Health(NIH) and $300 million to the Agency for Healthcare Research and Quality (AHRQ).

The IOM committee placed particular emphasis on leading questions regarding the clinical effectiveness of care. Half the 100 recommended primary research areas compare some aspect of the health care delivery system (see graph). Explaining this emphasis, the report says: “Research topics categorized in this group focus on comparinghow or where services are provided, rather than which services are provided. The prominence of health care delivery systems in the portfolio primarily reflects the interest of the public. . . as well as the committee’s belief that an early investment in CER should focus on learning how to make services more effective.”

Distribution of the IOM’s Recommended CER Priorities.

The total number of research areas within each research topic is shown at the top of each bar; this total is the sum of the primary and secondary research areas. Although each topic has been categorized in only one primary research area, many topics address more than one secondary research area; therefore, there are a total of 100 primary research areas and 193 secondary research areas. The most frequent research area was initially described as “Safety and Quality of Health Care,” but the IOM committee later relabeled it as “Health Care Delivery System,” which it considered more accurate.

Nearly a third of the other primary research priorities address racial and ethnic disparities, and nearly a fifth address patients’functional limitations and disabilities. Other key priority areas are cardiovascular disease, geriatrics, psychiatric disorders, neurologic disorders, and pediatrics. The committee also recommends supporting CER related to patients’ decision making, unhealthy behaviors such as smoking, and determining the most effective dissemination methods to ensure translation of CER results into best practices.

Cardiovascular and peripheral vascular disease ranks second as primary research areas, after the health care delivery system, in terms of the number of recommended research projects. Diseases of the heart were the leading causes of deaths in the United States in 2006, and such conditions are associated with multiple coexisting conditions, such as diabetes and obesity, that are becoming increasingly prevalent. The CER priority list includes eight topics related to ischemic heart disease, heart failure, and cardiac arrhythmias and two topics focused on the treatment and management of peripheral vascular disorders.

The third most frequent primary research area is psychiatric disorders: the committee has recommended CER studies on the location of mental health care, provider training, various pharmacologic treatments, depression, premature death related to mental disorders, and suicide. The priority list also includes six topics related to neurologic disorders, three of which are the following: imaging used for diagnosing such conditions; treatment of headaches, multiple sclerosis, and epilepsy; and the detection, treatment, and management of Alzheimer’s disease and other dementias.

Cancer, the second leading cause of deaths in the United States and one of the most costly diseases to treat, is the focus of six recommended primary CER topics, including screening technologies for colorectal and breast cancers and the use of imaging technologies for diagnosing, staging, and monitoring all cancers. The committee’s emphasis on exploring the increased use of advanced imaging studies reflects concern that has already led Congress to take steps to reduce the rapid growth in the use of such tests under Medicare²; private insurers have made similar efforts to control imaging use.

Although the ARRA legitimized the accelerated pursuit of government-funded CER, it did not provide for funding after 2 years or call for the creation of an entity that would oversee such research. The law did mobilize an array of interest groups that oppose, but have been unable to stop, these new activities.³ The Partnership to Improve Patient Care, a coalition of some 36 organizations, has asserted that CER may fail to consider differences among individual patients and may thereby “stymie progress in personalized medicine.”⁴ Other concerns expressed by opponents — particularly many Republicans — are that CER findings could lead to rationing and government intrusion into the doctor–patient relationship.

As Congress considers legislation to create a more permanent CER structure and to authorize sustained federal funding, opponents will undoubtedly raise similar concerns. The IOM report steers clear of recommending the creation of a new entity to oversee tax-funded CER activities — or of indicating which existing organization should do so — but it urges Congress and the DHHS to “establish a robust CER enterprise” that would closely coordinate these activities. Two bills introduced before the ARRA was enacted and a proposal put forward by health economist Gail Wilensky,⁵ who advised Senator John McCain (R-AZ) during his presidential bid, all envisioned a CER center that would provide objective, credible information on the probable clinical outcomes of various strategies for treating a given medical condition but that would not make coverage or reimbursement decisions.

The Comparative Effectiveness Research Act was introduced by Senators Max Baucus (D-MT) and Kent Conrad (D-ND) in 2008, and its essence is certain to be incorporated into any health care reform measure introduced by the Senate Finance Committee (Baucus chairs the committee, and Conrad is one of its most senior members).It would establish a new nonprofit corporation outside of government as the locus of CER, financed through a contribution from Medicare of $1 per beneficiary, with annual increases tied to medical inflation; private plans would be required to contribute $1 per covered life beginning in 2013. By contrast, the Children’s Health and Medicare Protection Act, which the House passed in 2007, would situate a new CER center within the AHRQ.

Over the next 20 months, the DHHS, the AHRQ, and the NIH will allocate the ARRA’s CER funding. The IOM report contains the first set of priorities for maximizing the value of this research. Because the committee’s work was requested by Congress and the resulting portfolio is so broad in scope, the recommendations may be more influential than they might otherwise have been, but the report is unlikely to quell the controversy surrounding CER.

Source Information

Mr. Iglehart is a national correspondent for the Journal.

This article (10.1056/NEJMp0904133) was published on June 30, 2009, at NEJM.org.

References

1. Institute of Medicine. Initial national priorities for comparative effectiveness research. Washington, DC: Institute of Medicine, 2009.
2. Iglehart JK. Health insurers and medical-imaging policy — a work in progress. N Engl J Med 2009;360:1030-1037. [Free Full Text]
3. Avorn J. Debate about funding comparative-effectiveness research. N Engl J Med 2009;360:1927-1929. [Free Full Text]
4. Garber AM, Tunis SR. Does comparative-effectiveness research threaten personalized medicine? N Engl J Med 2009;360:1925-1927. [Free Full Text]
5. Wilensky GR. Developing a center for comparative effectiveness information. Health Aff (Millwood) 2006;25:w572-w585. [Free Full Text]

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