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Assessment of clinical relevance by considering point estimates and associated confidence intervals
Author(s) -
Kieser Meinhard,
Hauschke Dieter
Publication year - 2005
Publication title -
pharmaceutical statistics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.421
H-Index - 38
eISSN - 1539-1612
pISSN - 1539-1604
DOI - 10.1002/pst.161
Subject(s) - relevance (law) , confidence interval , clinical significance , point estimation , clinical trial , point (geometry) , medicine , focus (optics) , medical physics , placebo , statistics , econometrics , intensive care medicine , alternative medicine , mathematics , political science , pathology , geometry , law , physics , optics
For the proof of efficacy of a new drug in a placebo‐controlled clinical trial it is not sufficient merely to demonstrate a statistically significant treatment difference. In recent years, regulatory authorities have strongly recommended assessing additionally whether the observed effect size is also of clinical relevance. This opinion is reflected in various guidelines which are of the utmost importance for the successful approval of a new drug. Clinical relevance can be investigated by responder analyses or by considering the point estimates on the original scale together with the associated confidence intervals. In this paper, we focus on the latter approach and discuss the suitability of different criteria which are commonly applied in medical research. Copyright © 2005 John Wiley & Sons, Ltd.