1. Use of Placebo in Pediatric Inflammatory Bowel Diseases
- Author
-
Michael J. Lentze, Sibylle Koletzko, Patrick F. van Rheenen, Paolo Lionetti, Johanna C. Escher, Berthold Koletzko, Gigi Veereman, Frank M. Ruemmele, Marla Dubinsky, Anne M. Griffiths, David R. Mack, Severine Vermeire, Salvatore Cucchiara, Lissy de Ridder, Dan Turner, Richard K. Russell, Jeffrey S. Hyams, Center for Liver, Digestive and Metabolic Diseases (CLDM), Pediatrics, Clinical sciences, and Growth and Development
- Subjects
Pathology ,Placebos ,0302 clinical medicine ,Maintenance therapy ,SEVERE CROHNS-DISEASE ,European Medicines Agency ,European Society for Pediatric Gastroenterology ,EPISODIC TREATMENT ,Child ,Clinical Trials as Topic ,Gastroenterology ,European Crohn's and Colitis Organization ,Ulcerative colitis ,Europe ,ULCERATIVE-COLITIS ,Research Design ,030220 oncology & carcinogenesis ,Therapeutic Equipoise ,030211 gastroenterology & hepatology ,TRIAL ,medicine.drug ,medicine.medical_specialty ,Canada ,Consensus ,pediatrics ,Placebo ,gastroenterology ,perinatology and child health ,03 medical and health sciences ,Pediatric Inflammatory Bowel Diseases, Placebo ,INFLIXIMAB ,medicine ,Adalimumab ,Humans ,Intensive care medicine ,Pediatric gastroenterology ,METAANALYSIS ,and Nutrition ,clinical trials ,Hepatology ,business.industry ,Consensus Development Conference ,Drugs, Investigational ,medicine.disease ,Inflammatory Bowel Diseases ,CONSENSUS GUIDELINES ,US Food and Drug Administration ,Infliximab ,Clinical trial ,Human Experimentation ,Pediatric Inflammatory Bowel Disease Network (PIBDnet) ,MAINTENANCE ,Pediatrics, Perinatology and Child Health ,Position paper ,MODERATE ,business ,ADALIMUMAB - Abstract
Performing well-designed and ethical trials in pediatric inflammatory bowel diseases (IBD) is a priority to support optimal therapy and 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, 4 organizations (the European Society for Pediatric Gastroenterology, Hepatology, and Nutrition; European Crohn's and Colitis Organization; the Canadian Children IBD Network; and the Global Pediatric IBD Network) jointly provide a statement on the role of placebo in pediatric IBD trials. Consensus was achieved by 94 of 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. It has been, however, 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 regarding 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.
- Published
- 2016