Designing clinical trials in paediatric inflammatory bowel diseases: a PIBDnet commentary.

The optimal trial design for assessing novel therapies in paediatric IBD (PIBD) is a subject of intense ongoing global discussions and debate among the different stakeholders. However, there is a consensus that the current situation in which most medications used in children with IBD are prescribed as off-label without sufficient paediatric data is unacceptable. Shortening the time lag between adult and paediatric approval of drugs is of the upmost importance. In this position paper we aimed to provide guidance from the global clinical research network (Pediatric Inflammatory Bowel Disease Network, PIBDnet) for designing clinical trials in PIBD in order to facilitate drug approval for children. A writing group has been established by PIBDnet and topics were assigned to different members. After an iterative process of revisions among the writing group and one face-to-face meeting, all statements have reached consensus of >80% as defined a priori. Next, all core members of PIBDnet voted on the statements, reaching consensus of >80% on all statements. Comments from the members were incorporated in the text. The commentary includes 18 statements for guiding data extrapolation from adults, eligibility criteria to PIBD trials, use of placebo, dosing, endpoints and recommendations for feasible trials. Controversial issues have been highlighted in the text. The viewpoints expressed in this paper could assist planning clinical trials in PIBD which are both of high quality and ethical, while remaining pragmatic.

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Competing interests: DT received consultation fees, research grant, royalties, or honorarium from Janssen, Pfizer, Hospital for Sick Children, Ferring, AstraZeneca, AbbVie, Takeda, Boehringer Ingelheim, Biogen, Atlantic Health, Shire, Celgene and Lilly for the last 3 years. AMG received consultation fees, research grant or honorarium from AbbVie, Celgene, Ferring, Gilead, Janssen, Lilly, Pfizer, Shire and Takeda for the last 3 years. DW received consultation fees, honoraria, research support or meeting expenses from AbbVie, Takeda, Ferring, Falk, Napp, Predictimmune and Roche for the last 3 years. LdR received consultation fees, honoraria or meeting expenses from AbbVie, Celltrion, Shire, Pfizer, Mallinckrodt and Nestlé for the last 3 years. DRM is president of a consulting company that provides service to the pharmaceutical industry. RKR received speaker fees and travel support and participated in medical board meetings with AbbVie, Janssen, Shire, Celltrion, NAPP and Nestle. MBH received research grant support from Genentech, AbbVie, Janssen, Takeda, Sucampo and Shire; is a member of the advisory board for Gilead and Genentech; received education grant from Mallinckrodt. FMR received speaker fees, consultation fees, research grants or honorarium from Janssen, AbbVie, Centocor, MSD France, Nestlé Nutrition Institute, Nestlé Health Science, Danone, Mead Johnson; Takeda, Celgene, Biogen, Shire, Pfizer, Therakos and ARKOPHARMA for the last 3 years. LC received honoraria and travel fees from Janssen for the last 3 years; is an employee of ReveraGen BioPharma and owns stock options in ReveraGen. JH is a member of the advisory board for Janssen and AbbVie; is a consultant of Pfizer, Roche, Lilly, Receptos and Boehringer Ingelheim. AB received research support from Prometheus, Janssen, AbbVie, Takeda and Buhlmann; consulting fees from Shire, Takeda and Best Doctors; and honoraria/royalties from Up to Date, Boston University and Nutricia. RNB is a member of the pediatric advisory board for AbbVie, Janssen, Pfizer and Celgene. MD received consultant fees or research support from Janssen, Abbvie, Roche, UCB, Pfizer, Takeda, Arena, Prometheus, Celgene and Lilly for the last three years. JMdC received consultation fees, honoraria or meeting expenses from AbbVie, Celltrion, MSD Spain, Dr Falk, Abbott, Lactalis, Otsuka, Roche, Celgene and Nestlé for the last 3 years. SK received consultant fees or research support from AbbVie, Berlin-Chemie, Biocodex, BioGaia, Danone, Mead Johnson, Nestle Nutrition, Shire and Thermo Fisher for the last 3 years.