Use of Placebo in Pediatric Inflammatory Bowel Diseases: A Position Paper from ESPGHAN, ECCO, PIBDnet and the Canadian Children IBD Network

Turner D1, Koletzko S, Griffiths AM, Hyams J, Dubinsky M, de Ridder L, Escher J, Lionetti P, Cucchiara S, Lentze MJ, Koletzko B, van Rheenen P, Russell RK, Mack D, Veereman G, Vermeire S, Ruemmele F. J Pediatr Gastroenterol Nutr. 2015 Nov 3. [Epub ahead of print]
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1*The European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) †European Crohn's and Colitis Organization (PECCO) ‡Global pediatric IBD network (PIBDnet) §Canadian Children IBD Network: a joint partnership of the Canadian Institutes of Health Research and the CH.I.L.D. Foundation ||Institute of Pediatric Gastroenterology, Shaare Zedek Medical Center, The Hebrew University of Jerusalem, Israel ¶Ludwig-Maximilians-University of Munich, Dr. von Hauner Children's Hospital, University of Munich Medical Center - Klinikum der Universität München, München, Germany #The Hospital for Sick Children, University of Toronto, Canada **Connecticut Children's Medical Center, Hartford, Connecticut, USA ††Department of Pediatrics, Feinstein IBD Center, Icahn School of Medicine, Mount Sinai, New York, USA ‡‡Department of Pediatric Gastroenterology, Erasmus MC-Sophia Children's Hospital, Rotterdam, The Netherlands §§Department Neurofarba, University of Florence - Meyer Hospital, Florence, Italy |||| Sapienza University of Rome, Italy ¶¶Department Pediatrics, Children's Hospital Medical Center, University Hospitals, Bonn, Germany ##University of Groningen, University Medical Centre Groningen, Netherlands ***The Royal Hospital for Children, Glasgow, UK †††Children Hospital of Eastern Ontario, University of Ottawa, Canada ‡‡‡Children's University Hospital, Brussels, Belgium §§§University Hospitals Gasthuisberg, Belgium ||||||Hôpital Necker-Enfants Malades, Paris, France.

Abstract Performing well designed and ethical trials in pediatric inflammatory bowel diseases (IBD) is a priority to support optimal therapy and to reduce the unacceptable long lag between adult and pediatric drug approval. Recently, clinical trials in children have been incorporating placebo arms into their protocols under conditions that created controversy. Therefore, four organizations (ESPGHAN, ECCO, the Canadian Children IBD Network and the global pediatric IBD network (PIBDnet)) jointly provide a statement on the role of placebo in pediatric IBD trials. Consensus was achieved by 94/100 (94%) voting committees' members that placebo should only be used if there is genuine equipoise between the active treatment and placebo. For example, this may be considered in trials of drugs with new mechanisms of action without existing adult data, especially when proven effective alternatives do not exist outside the trial. Placebo may also be used in situations where it is an 'add-on" to an effective therapy or to evaluate exit-strategies of maintenance therapy after long-term deep remission. However, it has been agreed that no child enrolled in a trial should receive a known inferior treatment both within and outside the trial. This also includes withholding therapy in children who show clinical response after a short induction therapy. Given the similarity between pediatric and adult IBD in regards to pathophysiology and response to treatments, drugs generally cannot be considered being in genuine equipoise with placebo if it has proven efficacy in adults. Continued collaboration of all stakeholders is needed to facilitate drug development and evaluation in pediatric IBD.

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