Primary Endpoints for Irritable Bowel Syndrome Trials: A Review of Performance of Endpoints

Michael Camilleri, Allen W. Mangel, Sheri E. Fehnel, Douglas A. Drossman, Emeran A. Mayer, Nicholas J. Talley

Research output: Contribution to journalArticle

68 Citations (Scopus)

Abstract

The choice of primary endpoint for a clinical trial is one of the most important determinants of the ability of a clinical trial to demonstrate efficacy of therapeutic agents. Although there are still no clear, universally accepted guidelines on the definition of clinical benefit for irritable bowel syndrome (IBS), consensus guidelines stress the importance of using validated endpoints. This article reviews the evidence available in the literature on the psychometric validation and performance of the 3 endpoints recommended by the Rome III Committee for use as primary endpoints in treatment trials of IBS. The Rome III Committee recommends 2 types of measures: binary endpoints addressing the construct of relief (that is, adequate relief and satisfactory relief) and an integrative symptom questionnaire that addresses the change in severity of a representative group of symptoms of IBS (that is, the IBS Severity Scale). The current evidence suggests that at present, adequate relief should be recognized by regulatory authorities as an acceptable primary endpoint in clinical trials. This analysis also suggests that data from individual clinical trials should be pooled and undergo meta-analysis, and that prospective studies should be considered to further characterize the performance of available endpoints as outcome measures in pharmacotherapeutic trials in IBS.

Original languageEnglish (US)
Pages (from-to)534-540
Number of pages7
JournalClinical Gastroenterology and Hepatology
Volume5
Issue number5
DOIs
StatePublished - May 2007

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Irritable Bowel Syndrome
Clinical Trials
Guidelines
Aptitude
Psychometrics
Meta-Analysis
Consensus
Outcome Assessment (Health Care)
Prospective Studies

ASJC Scopus subject areas

  • Gastroenterology

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Primary Endpoints for Irritable Bowel Syndrome Trials : A Review of Performance of Endpoints. / Camilleri, Michael; Mangel, Allen W.; Fehnel, Sheri E.; Drossman, Douglas A.; Mayer, Emeran A.; Talley, Nicholas J.

In: Clinical Gastroenterology and Hepatology, Vol. 5, No. 5, 05.2007, p. 534-540.

Research output: Contribution to journalArticle

Camilleri, Michael ; Mangel, Allen W. ; Fehnel, Sheri E. ; Drossman, Douglas A. ; Mayer, Emeran A. ; Talley, Nicholas J. / Primary Endpoints for Irritable Bowel Syndrome Trials : A Review of Performance of Endpoints. In: Clinical Gastroenterology and Hepatology. 2007 ; Vol. 5, No. 5. pp. 534-540.
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