Health care providers are frequently confronted by patients or situations that demand immediate attention, yet the information upon which we base our decision-making is imperfect.

A literature search on PubMed can help narrow the options for a given condition, but because much of the existing evidence has been derived from efficacy trials conducted under idealized circumstances with restrictive recruitment, it is difficult to know the best course of action for a specific patient encounter.

Accordingly, there is increasing demand for data generated by studies that are inclusive enough to be generalizable yet sufficiently refined to be germane to an individual episode of care.

Enter comparative effectiveness research (CER) – a systematic approach to the generation and synthesis of evidence “that compares the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions in ‘real world’ settings.”¹

Also referred to as patient-centered outcome research, the primary purpose of CER is to improve health care by defining the right treatment for a specific patient at a given point in time and disseminating such information to relevant stakeholders (such as consumers, clinicians, purchasers, and policymakers).²

The promise of more applicable, user-friendly evidence that truly enhances decision-making at the individual patient level formulates the core of the CER agenda. By employing advanced methodology such as pragmatic trial design (which attempts to reproduce the clinical circumstances where an intervention will ultimately be used) and Bayesian adaptive randomization (which enables iterative protocol adjustment with elimination of subgroups that appear unlikely to respond), CER will yield insight into often overlooked outcome variations within a study population – an occurrence known as “treatment response heterogeneity.”^3,4

While CER is fundamentally about improving clinical outcomes, it also offers the potential to eliminate waste and shift practice away from low-yield, high-cost interventions. Such potential prompted lawmakers to include CER as a one of eight funded components directed to the Department of Health and Human Services (DHHS) as part of the American Recovery and Reinvestment Act (ARRA) of 2009.

The law allocates $400 million for discretionary use by the Office of the Secretary (OS) of DHHS, $400 million to the National Institutes of Health (NIH), and $300 million to the Agency for Healthcare Research and Quality (AHRQ). In addition to this capital outlay of $1.1 billion, ARRA established the 15-member Federal Coordinating Council (FCC) for Comparative Effectiveness Research (see www.hhs.gov/recovery/programs/os/cerbios.html for a listing of council members) to oversee the government’s CER enterprise.

To help ensure that funds were directed to areas of clinical need, ARRA further mandated that the Institute of Medicine (IOM) establish a list of national priorities for CER. The final IOM report, released in June 2009, identified 29 broad “research areas” within which 100 primary and 193 secondary research priority topics were defined (more than a quarter of which pertained to health care delivery systems or racial and ethnic disparities).⁵

ARRA funds have since been fully disbursed by the three parent organizations, with each having a slightly different focus.

The OS-DHHS concentrated largely on strengthening the infrastructure for CER data compilation and dissemination, and the NIH centered on specific research projects (88 of which were on the IOM’s list of 100 primary priority topics). The AHRQ engaged in a more broad-based effort to:

• Work within preexisting CER programs such as the Developing Evidence to Inform Decisions about Effectiveness (DEcIDE) network.
• Support evidence synthesis through the creation of Evidence-based Practice Centers.
• Derive new evidence through funding of Clinical and Health Outcomes Initiative in Comparative Effectiveness (CHOICE) grants.
• Invest in disease or service specific registries.
• Expand the available workforce of CER researchers through career development awards.
• Devise and test strategies to incorporate CER findings into clinical practice.
• Promote citizen engagement and uptake of CER.³

Because the vast majority of funds were obligated in fiscal year 2010 (see www.hhs.gov/recovery/programs/#Comparative for complete details), it is not yet possible to know the relative value of the ARRA CER initiative.

Though ARRA was clearly an important mechanism to initiate organized funding of CER, the program was, by design, finite in scale. As ARRA funding winds down, however, a new phase of CER funding will ramp up in the form of the Patient-Centered Outcome Research Institute (PCORI), a novel public-private partnership that was established by the Patient Protection and Affordable Care Act of 2010.

Starting in 2013, it will be permanently funded (to the tune of $500 million a year) by a trust derived from a nominal tax on Medicare and every private health insurance company for each life they insure. A primary function of PCORI will be to establish a CER portfolio and commission related projects within the confines of a fixed budget – a task that, according to leading figures in the FCC and the IOM, will likely require that PCORI define not only the research questions but also the methodology necessary to adequately address them.^3,6

The latter will be facilitated by, as stipulated in the health care law, appointment of a standing Methodology Committee. The IOM recently completed solicitation for “Tier 1” pilot project ideas and a request for applications will be forthcoming this fall (see www.pcori.org/images/Tier_1_Topics_Input_Release.pdf for more details).

To capitalize on the CER agenda, emergency physicians will need to push for research that is relevant to the specialty and ensure that new evidence, as it evolves, gets translated into practice.⁷

This will require an investment in human and scientific capital (at the institutional level and beyond) to both create the next generation of emergency medicine researchers and foster a culture that espouses individualized (and perhaps less profitable) decision-making.

A shared data infrastructure is but one example of how the emergency medicine community could work together to better understand, in a longitudinal fashion, how variations in outcome may relate to differential practice patterns and disparate patient substrates.

The commitment to CER will be lasting – all we need to do is seize the day.

References

1. Federal Coordinating Council for Comparative Effectiveness Research. (Accessed Sept. 4, 2011, at www.hhs.gov/recovery/programs/cer/cerannualrpt.pdf.)
2. Conway PH, Clancy C. Transformation of health care at the front line. JAMA 2009;301:763-5.
3. Sox HC. Comparative effectiveness research: a progress report. Ann. Intern. Med. 2010;153:469-72.
4. Sullivan P, Goldmann D. The promise of comparative effectiveness research. JAMA 2011;305:400-1.
5. Iglehart JK. Prioritizing comparative-effectiveness research – IOM recommendations. N. Engl. J. Med. 2009;361:325-8.
6. VanLare JM, Conway PH, Sox HC. Five next steps for a new national program for comparative-effectiveness research. N. Engl. J. Med. 2010;362:970-3.
7. Conway PH, Clancy C. Com­parative-effectiveness research – implications of the Federal Coordinating Council’s report. N. Engl. J. Med. 2009;361:328-30.

Dr. Levy is an Associate Professor and Associate Director of Clinical Research in the Department of Emergency Medicine at Wayne State University. He is also a member of ACEP’s Research Committee.