Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
3292056 | Gastroenterology | 2016 | 13 Pages |
Abstract
This article summarizes recent progress and regulatory guidance on design of trials to assess the efficacy of new therapies for functional gastrointestinal disorders (FGIDs). The double-masked, placebo-controlled, parallel-group design remains the accepted standard for evaluating treatment efficacy. A control group is essential, and a detailed description of the randomization process and concealed allocation method must be included in the study report. The control will most often be placebo, but for therapeutic procedures and for behavioral treatment trials, respectively, a sham procedure and control intervention with similar expectation of benefit, but lacking the treatment principle, are recommended. Investigators should be aware of, and attempt to minimize, expectancy effects (placebo, nocebo, precebo). The primary analysis should be based on the proportion of patients in each treatment arm who satisfy a treatment responder definition or a prespecified clinically meaningful change in a patient-reported outcome measure. Data analysis should use the intention-to-treat principle. Reporting of results should follow the Consolidated Standards for Reporting Trials guidelines and include secondary outcome measures to support or explain the primary outcome and an analysis of harms data. Trials should be registered in a public location before initiation and results should be published regardless of outcome.
Keywords
IBS-MEMAFDAIBSPragmatic clinical trialsIBS-DIBS-CRCTHRQOLBSFSPCTFGIDMCIDRandomized controlled trialEuropean Medicines agencyFunctional gastrointestinal disorderFunctional gastrointestinal disordersUS Food and Drug Administrationirritable bowel syndrome with diarrheairritable bowel syndrome with constipationirritable bowel syndromeintention to treatControlled trialpatient-reported outcome measureBristol Stool Form Scalepatient-reported outcomePROCONSORTHealth-related quality of life
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Authors
E. Jan Irvine, Jan Tack, Michael D. Crowell, Kok Ann Gwee, Meiyun Ke, Max J. Schmulson, William E. Whitehead, Brennan Spiegel,