Interpreting Registrational Clinical Trials of Biological Therapies in Adults with Inflammatory Bowel Diseases

Ghosh S1, Sandborn WJ, Colombel JF, Feagan BG, Panaccione R, Hanauer S, Schreiber S, Peyrin-Biroulet L, Vermeire S, Eichner S, Huang B, Robinson AM, Pappalardo B. Inflamm Bowel Dis. 2016 Aug 31. [Epub ahead of print]
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1*Department of Medicine, University of Calgary, Calgary, Alberta, Canada; †Division of Gastroenterology, University of California San Diego, La Jolla, California; ‡Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York, New York; §Robarts Clinical Trials, Robarts Research Institute, Department of Medicine, University of Western Ontario, London, Ontario, Canada; ‖Department of Medicine, Division of Gastroenterology and Hepatology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois; ¶Department of Medicine, University Hospital Schleswig-Holstein, University of Kiel, Kiel, Germany; **INSERM Unité 954, Department of Gastroenterology, University of Lorraine, Nancy, France; ††Department of Gastroenterology, University Hospital Leuven, Leuven, Belgium; and ‡‡AbbVie Inc., North Chicago, Illinois.


BACKGROUND: The use of biologics to treat inflammatory bowel disease is supported by robust randomized controlled trials in both ulcerative colitis and Crohn's disease. Nonetheless, an understanding of the principles of clinical trial design is necessary to extrapolate study findings to clinical practice.

METHODS: We conducted a review of inflammatory bowel disease registrational clinical trials of biologics to determine how differences in trial design potentially influence results and interpretation.

RESULTS: Registrational trials of biological agents have used diverse patient populations, outcome measures, and designs, which makes comparisons of results among studies difficult. Key differences among trials include patient populations, choice of symptom-based measures or objective outcomes as endpoints, and overall trial design. Additional factors, including analytical methods, can also influence the interpretation of outcomes.

CONCLUSIONS: The most robust evidence is derived from comparative effectiveness trials. In the absence of these, clinicians should be aware of the various methodological issues which could impact interpretation of efficacy and safety outcomes, including differences in patient population, study design, and analytic methodology.This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially.

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