Importance of Clinical Trials and International Cooperation
As physicians, we are fundamentally biologists with the objective of utilizing this knowledge to limit disease burden. As medical practitioners and investigators, we strive to meaningfully contribute to this growing body of knowledge to improve the understanding of physiology and pathophysiology in an attempt to develop better therapeutic approaches for patients. Preliminary data from multiple sources are used to develop mechanistic constructs which can be focused into a testable hypothesis. In clinical investigation, the randomized controlled clinical trial (RCT) has emerged as the most definitive method of determining whether or not a novel therapy alters the clinical course of a cohort of patients with a defined disease. Although the skillful practice of medicine remains an art of using, at best incomplete data, the RCT provides the most quantitative assessments of both the benefits as well as the risks of a therapeutic strategy. However, RCTs that can provide such data are massive undertakings that require long-term dedication from large numbers of patients, investigators, and staff. As such, these large international cooperative ventures demand huge financial and personal commitments. As a consequence, smaller studies with more intermediate so-called surrogate outcomes are often conducted providing less definitive data that can only be extrapolated to presumed clinical outcomes. This presentation will highlight the advances and challenges of designing and conducting international clinical outcome trials with examples from cardiovascular medicine looking backwards as well as forward.
- © 2013 by American Heart Association, Inc.