Evidence and Education
About 60 years ago a major, powerful methodology was added to the clinical investigator's toolbox: the double-blind randomized controlled trial (RCT). As a result, therapeutic decisions in cardiovascular medicine are increasingly evidence-based. The expanding array of treatment options, a decline in event rates, and a desire for increased efficiency have stimulated several major shifts in contemporary RCTs: the use of a composite endpoint, a noninferiority hypothesis, and an adaptive design.
To facilitate the comparison of the benefits and risks of various treatment options in "real world" settings, comparative effectiveness research (CER) uses methodologies such as pragmatic RCTs, meta-analysis, decision models, and observational studies. Because of the increasing possibility of the play of chance, selection bias, and confounding, there is a gradient in the level of rigor by which evidence is currently acquired and analyzed as one moves from the homogeneous populations studied in RCTs to the more diverse populations studied in CER. Greater use of sensitivity analyses, propensity scores, and multiple control groups are needed in CER to properly interpret statistical associations when considering cause-and-effect relationships.
Research and education in the optimal methods for aligning the efforts of groups of investigators conducting RCTs and those performing CER are high priorities. This is important to restrain appropriately the costs of healthcare (without compromising patient outcomes), while accelerating the delivery of advances in clinical/ translational science to the bedside. Such efforts are needed to facilitate achieving the AHA's 2020 strategic goal of improving the cardiovascular health of all Americans by 20% while reducing deaths from cardiovascular disease and stroke by 20%.
- © 2010 by American Heart Association, Inc.