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.