Commentary: Epidemiology, Comparative Effectiveness Research, and the National Institutes of Health

In December, 2007, the Congressional Budget Office (CBO) released a report entitled “Research on the Comparative Effectiveness of Treatments.”1 The CBO noted that for many conditions high-quality evidence that can direct clinical care is lacking. The report called for expanded federal efforts in comparativeness effectiveness research (CER), defined as “a rigorous evaluation of the impact of different options that are available for treating a given medical condition for a particular set of patients.” Subsequent to the CBO report, two major pieces of Congressional legislation have stimulated public interest in CER. The American Recovery and Reinvestment Act of 2009 allocated $1.1 Billion to the National Institutes of Health (NIH), the Agency for Health Research and Quality (AHRQ), and the Office of the Secretary for Health and Human Services to fund comparative effectiveness research, and to generate major reports on national priorities in CER. The Affordable Care Act of 2010 established a non-profit nongovernmental “Patient Centered Outcomes Research Institute”2 that will establish and oversee a national research program and attempt to establish methodological standards for CER. While the CBO report and the two Congressional Acts have drawn much attention to CER, such research is not new.3 The NIH and the Veterans Administration (VA) have supported comparative effectiveness research for decades. However, the new national focus on CER has stimulated a number of discussions—and, at times, debates4—on topics such as the need for more high-quality clinical evidence, the attitudes of physicians towards science and evidence-based decision-making,5 the role of economics research in health care,6 and (among the most contentious issues) the complementary roles of observational and experimental research in informing clinical care.7,8 With the advent of the informatics revolution and advances in biostatistical methods, some are asking whether population-based and clinical epidemiology may be in a position to push aside the “gold standard” status traditionally given to randomized trials. Others are more cautious, noting that observational findings have often led to dangerously wrong conclusions.