Clinical Significance

• Published in: Methods and Applications of Statistics in Clinical Trials pp. 170 - 190
• Main Authors: Kamath, Celia C, Sloan, Jeffrey A, Cappelleri, Joseph C
• Format: Book Chapter
• Language: English
• Published: Hoboken, NJ, USA John Wiley & Sons, Inc 29.08.2014
• Subjects: clinical significance; clinical studies; health‐related quality of life (QOL); patient‐reported outcomes
• ISBN: 1118595920; 9781118595923
• DOI: 10.1002/9781118596005.ch15

The field of patient‐reported outcomes, particularly health‐related quality of life (QOL), has burgeoned in the last few years. Why is clinical significance of QOL scores important? It aids in the design of studies by helping to determine sample size calculations. This chapter provides an overview on the clinical significance of QOL measures. The section on Design and Methodology covers the different perspectives and existing methods to determine clinical significance. Two common approaches used to establish the interpretability of QOL measures are termed :anchor‐based: and :distribution based:. The characteristics of each approach are described in detail. The section on Examples illustrates trials in which researchers attempted to define the concept on specific QOL measures. It also presents general advice for defining and interpreting clinical significance in clinical studies. Then, the section on Recent Developments highlights new methods to determine clinical significance. Finally, the chapter discusses some future directions for research.