IOM lists top 100 comparative effectiveness research topics

The panel recommends examining treatment outside clinical settings and encourages strong public awareness and involvement in such research.

By — Posted July 13, 2009

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Federal funding for examining the effectiveness of different treatments for the same conditions also should support work on how best to translate research results into practice, according to the Institute of Medicine. An IOM panel recently prioritized the comparative effectiveness research it says deserves federal support.

The IOM committee stressed the importance of continuously updating research priorities and looking at methods for addressing patients' behavior outside hospitals and physicians' offices, according to its June 30 report, "Initial National Priorities for Comparative Effectiveness Research" (link).

The recent federal stimulus act signed by President Obama on Feb. 17 includes $1.1 billion for comparative effectiveness research, including $400 million for the Dept. of Health and Human Services. The act called for the IOM to provide HHS with a list of high-priority research topics to help lay the groundwork for a national research effort. The panel also offered several recommendations for sustaining the research and for ensuring that it improves the health system.

The committee trimmed the list of initial research topics to 100 from 1,268 submitted by the public. Panel members narrowed the priorities list by considering the total burden of illness linked to each topic, perceived gaps in clinical decision-making and the potential for comparative effectiveness research to change practices, said Harold Sox, MD, committee co-chair and a former editor of the Annals of Internal Medicine. The committee also held a March 20 public forum attended by representatives from 54 organizations, including medical societies, researchers, consumer advocates, health plans, drug and medical device manufacturers, and others.

The 100 priorities are not ranked individually. Instead, committee members grouped the research areas into quartiles.

The most frequently nominated research area involved methods for implementing research findings into patient care, Dr. Sox said. Next was research examining racial disparities in health care, followed by disabilities research. He said committee members added several topics to fill gaps in the publicly generated list -- such as studying management strategies for noninvasive breast cancer, a top 25 topic.

The IOM committee's top 25 priorities include several research questions addressing physician and hospital care, such as comparing the effectiveness of management strategies for localized prostate cancer. But the panelists also called for a more holistic approach, including examining the effectiveness of physical education, meal programs and other school-based interventions in treating and preventing obesity in children. Finding better approaches to preventing disease also is important, Dr. Sox said.

"That often will occur outside the office setting and more in a community-based setting, such as schools," he said.

Reaction to the IOM report was generally positive. American Medical Association Immediate Past President Nancy H. Nielsen, MD, PhD, applauded the IOM panel for prioritizing prevention and health disparities topics. "Both are key issues the AMA has identified as needing more study and research."

The vast majority of health status is determined by the things people do outside of hospitals and physician offices, said Ellen-Marie Whelan, PhD, associate director of health policy at the Center for American Progress, a liberal think tank.

Funding and cost issues

The IOM committee also recommended that HHS:

  • Create a coordinating or advisory body to help implement a national comparative effectiveness research program.
  • Write a plan to expand and sustain the number of workers who conduct the research.
  • Develop large-scale clinical and data networks allowing researchers to access existing and new data from health insurers, hospital and physician networks, and other sources.
  • Regularly re-examine and update the priorities list to address existing knowledge gaps.

The stimulus act's $1.1 billion in federal funding will vastly increase work in the field. But the speed and scope of improvements to the health care system will depend on the types of studies proposed and funded, Dr. Sox said.

IOM committee member Mark B. McClellan, MD, PhD, a former Medicare administrator, said quick improvements in databases and research infrastructure could speed the translation of existing research into new practice standards. But the IOM panel recommended randomized, controlled trials for 49 of the 100 research topics. Such studies can cost tens of millions of dollars, said Gail Wilensky, PhD, a former Medicare administrator and now a senior fellow at Project Hope, an international health advocacy organization.

Many Republicans in Congress have expressed concern that HHS will use the results of comparative effectiveness research to restrict Medicare coverage of treatments -- especially more costly ones. But Wilensky said Medicare is prohibited from considering costs when making coverage determinations.

Wilensky agrees that cost should not be one of the criteria in comparing the effectiveness of treatments. But once experts understand which treatments and interventions work best, price will become a factor. "It's not irrelevant to ask: 'What do we know about the cost differences?' "

Dr. Sox said the research should educate the public about health care treatment options. "Hopefully, armed with that information, more patients will feel that they can speak up and really assert themselves in talking to their doctor to be sure that their preferences are reflected in whatever testing and treatment is decided on."

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The IOM's research priorities

An Institute of Medicine panel on June 30 identified 100 initial research priorities for using federal stimulus money to compare the effectiveness of various treatments on the same conditions. Each recommended priority breaks down into one of 29 categories based on its primary focus. The same priorities are then assigned to additional, multiple categories based on secondary focus elements.

Category Primary research items Secondary research items
Alcoholism, drug dependency and overdose 2 4
Birth and developmental disorders 3 1
Cardiovascular and peripheral vascular disease 8 13
Complementary and alternative medicine 3 0
Endocrinology and metabolism disorders 2 12
Eyes, ears, nose and throat disorders 2 1
Functional limitations and disabilities 2 20
Gastrointestinal system disorders 1 1
Genetics and disease 0 3
Geriatrics 2 19
Health care delivery systems 23 27
Immune system, connective tissue and joint disorders 1 3
Infectious diseases 3 2
Joint disorders 1 3
Kidney and urinary tract disorders 2 1
Liver and biliary tract disorders 1 1
Musculoskeletal disorders 5 7
Neurologic disorders 6 11
Nutrition and obesity 3 1
Oncology and hematology 6 5
Oral health 2 1
Palliative and end-of-life care 2 0
Pediatrics 1 15
Psychiatric disorders 7 10
Racial and ethnic disparities 3 26
Respiratory disease 1 2
Sexual function and reproductive disorders 0 2
Skin disorders 3 1
Trauma, emergency medicine and critical care medicine 1 2
Women's health 5 2

Source: Institute of Medicine, June (link)

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