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The Federal Coordinating Council for Comparative Effectiveness Research Report to the President and The Congress opens with the following questions: “What is the best pain management regimen for disabling arthritis in an elderly African-American woman with heart disease? For neurologically impaired children with special health care needs, what care coordination approach is most effective at preventing hospital readmissions? What treatments are most beneficial for patients with depression who have other medical illnesses? Can physicians tailor therapy to specific groups of patients using their history or special diagnostic tests? What interventions work best to prevent obesity or tobacco use?”

Unfortunately, the authors continue, the answer to such comparative, patient-centered questions is often, “We don’t really know.” To address this fundamental lack of understanding, the American Recovery and Reinvestment Act of 2009 (ARRA) – known as the “stimulus package” – allocates $1.1 billion to support “the development and dissemination of research assessing the comparative effectiveness of health care treatments and strategies.” These funds will be distributed through the Department of Health and Human Services (HHS), the National Institutes of Health (NIH), and the Agency for Healthcare Research and Quality (AHRQ) over the next two years. Priority research areas recommended by the Institute of Medicine (IOM) include the health delivery system, disparities, cardiovascular health, geriatrics, women’s health, and end-of-life care.

The federal prioritization of comparative effectiveness research (CER) represents a massive investment in research to impact clinical decision-making and care delivery processes directly. As the IOM puts it, “Comparative effectiveness research is a way to identify what works for which patients under what circumstances.”

To gain such actionable findings requires comparative research with “real world” populations to assess the efficacy of alternative devices or treatments. In this way, CER goes beyond randomized controlled trials (RCTs) such as those for drug approval by the FDA, in which treatments are tested in ideal settings and compared with a placebo.

The authors of a recent opinion piece in the New England Journal of Medicine (Comparative Effectiveness – Thinking beyond Medication A versus Medication B,” NEJM, Volume 361:331-333, July 23, 2009, Number 4) take this one step further: “If CER’s full potential for improving the population’s health is to be realized, such comparisons must go beyond those between medication A and medication B or device A and device B: we must also assess medications or devices in comparison with behavioral interventions, either alone or in conjunction with other approaches.” This is common sense. For instance, medication-based approaches to treating diabetes should be compared with the effects of behavioral approaches to weight reduction. Federal CER funding aims to stimulate such practical interdisciplinary research, research that has historically been hindered by academic silos.

Proponents of CER maintain that this research is inherently patient-centered: by studying patients in typical day-to-day care, researchers will identify approaches that meet the needs of individual patients. Armed with such specific, actionable intelligence, clinicians will be enabled to more effectively guide patient care.

Given the nature of the research process, many CER projects will yield marginally interesting results with little direct applicability. And tens to hundreds of millions of dollars will be spent on developing research infrastructure – not on research itself. Nevertheless, with a real-world, patient-centered focus, it is likely that some CER projects will succeed in shedding light on the true drivers of health outcomes. Whether these successes justify the substantial federal investment will depend upon the extent to which they transform care delivery.