Clinical Significance

Celia C. Kamath, Jeffrey A. Sloan, Joseph C. Cappelleri

Research output: Chapter in Book/Report/Conference proceedingChapter

4 Scopus citations


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.

Original languageEnglish (US)
Title of host publicationConcepts, Principles, Trials, and Designs
Number of pages21
ISBN (Electronic)9781118596005
ISBN (Print)9781118595923
StatePublished - Sep 26 2014


  • Clinical significance
  • Clinical studies
  • Health-related quality of life (QOL)
  • Patient-reported outcomes

ASJC Scopus subject areas

  • General Medicine


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