Are We Ready to Abandon Placebo in Randomised Clinical Trials for Inflammatory Bowel Disease? Pros and Cons.
The role of placebo in clinical trials for drug development in inflammatory bowel disease [IBD] was the topic of a panel discussion held during the 10th Congress of the European Crohn's and Colitis Organisation [ECCO], in Barcelona, Spain, in 2015. Panellists discussed a number of issues around placebo-controlled trials in IBD, noting issues such as difficulties with recruitment, leading to less then representative patient populations in clinical studies. It was noted that, whereas the easiest answer may be to drop placebo, it is much more complicated than that. The relevance of placebo is affected by a number of factors, including the phase of the trial, as well as the nature of the drug. In most cases where placebo has been left out in drug development, it has been for trials involving a new formulation, a new dosing schedule, or a biosimilar, for example. The panel agreed that placebo-controlled trials are of particular importance early in the development programme, perhaps not so much in phase III, although placebo is important for monitoring safety. The current trial paradigm, in which patients remain on a plethora of, likely ineffective and toxic, background medication, was also questioned. The applicability of placebo in the paediatric population was also discussed. The overall consensus from this panel discussion was that placebo is still necessary in clinical trials in inflammatory bowel disease, but there remain questions as to how and when placebo should be used.
Humanitas University, IBD Center, Humanitas Clinical and Research Hospital, Milan, Italy sdanese@hotmail.com.
Silvio
S
Danese
Federal Institute for Drugs and Medical Devices, Bonn, Germany.
Elmer
E
Schabel
Ferring, St. Prex, Switzerland.
Johan
J
Masure
Janssen Research & Development, Spring House, PA, USA.
Scott
S
Plevy
University of Kiel, Department of Medicine I, Kiel, Germany.
Stefan
S
Schreiber
D032921:Consensus; D004341:Drug Evaluation / Q000941:ethics / Q000379:methods; D005765:Gastrointestinal Agents / Q000494:pharmacology*; D006801:Humans; D015212:Inflammatory Bowel Diseases / Q000188:drug therapy*; D010919:Placebos; D016032:Randomized Controlled Trials as Topic / Q000379:methods*
D005765:Gastrointestinal Agents; D010919:Placebos
Inflammatory bowel disease; drug development; placebo