Charting a Path From Comparative Effectiveness Funding to Improved Patient-Centered Health Care

Abstract

PATIENTS AND PHYSICIANS ARE FORTUNATE TO CONfront expanded options for diagnosis, treatment, and monitoring. While physicians strive to tailor recommendations to the unique characteristics and circumstances of each patient, many decisions today must be made without reliable comparative information. That information gap is frustrating for patients, who too often undergo trial and error medicine, as well as for physicians. Moreover, few health care organizations are designed to harness the best clinical insights for widespread dissemination and learning. It is precisely this chasm that a focus on comparative effectiveness (CE) research, also known as patientcentered health research, is intended to address. The concept of CE research is not new; indeed the establishment of the Agency for Healthcare Research and Quality (AHRQ) 20 years ago represented an important investment in supporting science that helps identify which options are most effective for which patients. In addition, the more applied aspects of clinical research often address comparative benefits and harms of therapeutic choices. What is new is the recognition of and substantial public support for research that is essential for delivering care that is consistently patient centered and an important accelerator for achieving the promise of personalized medicine. However, the dimensions of the scientific foundation from which to advance this enterprise are not clearly defined. For example, the Institute of Medicine (IOM) committee commissioned by the US Congress to identify priorities for CE research relied on broad stakeholder input and expert judgment regarding important clinical topics, but had no clear inventory of published studies or research in progress to inform their deliberations. Moreover, the context for increasing interest in comparative information implicitly includes assessment of whether the resulting information is useful to clinicians and patients—and is used. In this issue of JAMA, the article by Hochman and McCormick represents an important step in articulating criteria for published studies that assess the CE of medications. The authors conducted an analysis of CE research studies from the 6 general and internal medicine journals with the highest impact factors. Their study highlights several issues that warrant further discussion. First, the authors report that 87% (90 of 104) of the CE research studies published in these journals relied on noncommercial funding. The American Recovery and Reinvestment Act appropriated $1.1 billion for CE research and the US Department of Health and Human Services is now awarding grants and contracts based on these funds. The Federal Coordinating Council for Comparative Effectiveness Research report outlined a vision for how this funding could lay the foundation for an enterprise that improves the nation’s health and the performance of the US health care system. The budget for fiscal year 2011 issued by President Obama recommends $286 million for the AHRQ portfolio dedicated to this type of research—a $261 million increase from the pre–American Recovery and Reinvestment Act baseline. Given the importance of noncommercial funding to CE research, funding from AHRQ, the National Institutes of Health, the US Department of Veterans Affairs, and others will be essential to continue to the momentum of American Recovery and Reinvestment Act funding. Second, the authors identified only 11 studies (11%) that compared medications with nonpharmacologic interventions and 32 studies (31%) that compared different pharmacologic strategies (31%); both are essential to helping clinicians and patients make fundamental therapeutic decisions. Similarly, both the IOM and the Federal Coordinating Council for Comparative Effectiveness Research included a broad definition of interventions within the spectrum of CE research including procedures, medical and assistive devices, diagnostic testing, behavioral change, and delivery system interventions. Future CE research inventories should capture and categorize the full spectrum of these interventions. Third, the authors note that only 32% (n=104) of medication studies published in these 6 journals met their criteria for CE research. Since many clinical trials, including those supported by the National Institutes of Health and the US Department of Veterans Affairs address benefits and harms of treatments, this finding raises important questions re-