Decisions made by patients and physicians about the care that the patient will receive frequently involve choices among available therapies. Commonly, there are few research studies that compare alternative treatments and provide evidence to guide a particular decision. Even less plentiful are studies that compare treatment options in real-world settings. As the realization of this need for enhanced comparative information to achieve best outcomes has grown, attention has focused on comparative eﬀectiveness research. The Institute of Medicine deﬁnes this Comparative Eﬀectiveness Research (CER) as:
“...the generation and synthesis of evidence that compares the beneﬁts and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians, purchasers and policymakers to make informed decisions that will improve health care at both the individual and population levels.”
With the current national focus on improving our health care system, CER has received substantial attention. In addition, in early 2009, the American Recovery and Reinvestment Act allocated $1.1 billion in new funds to increase the number of new CER studies. Three important types of comparative eﬀectiveness research include: 1) Randomized Controlled Trials (RCTs), 2) Meta-Analysis, and 3) Observational Studies. If conducted and interpreted correctly, these types of research can help to inform health care decision-making. If, however, such studies are conducted or interpreted incorrectly, the comparative answers from these studies may be inaccurate, or worse, misleading. This white paper will review the three CER study types, discuss their proper methodologic approaches, and provide tools for CER consumers to use when they review them. Although additional types of biomedical research are very useful in understanding disease and developing new drugs, the focus of this paper is solely on the most common types of research used for CER.