Managing patient expectations

Abstract

The wealth of research that is currently being conducted in cystic fibrosis (CF) may bring to fruition new treatments that may ultimately have a significant impact on progression of this disease. However, the research requirements involved in bringing these agents to the market are numerous and complex, patient expectations are high, and the general sense of optimism must be balanced against the knowledge that positive research study results may not necessarily materialize into clinically meaningful advances. This discussion explores the current research challenges that investigators face in meeting patient enrollment requirements, evaluating impact of therapy on critical patient outcomes (eg, survival), and extrapolating the available evidence. Also included are 2 brief case studies with audience discussion that illustrate the various issues that arise among patients participating in clinical research.