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Outcome measures for clinical trials in paediatric IBD: an evidence-based, expert-driven practical statement paper of
Frank M Ruemmele1, Jeffrey S Hyams2, Anthony Otley3
1Université Paris Descartes, Sorbonne Paris Cité, Paris, France INSERM U989, Institut IMAGINE, Paris, France APHP, Hôpital Necker Enfants Malades, Service de Gastroentérologie pédiatrique, Paris, France.
Insights
Choosing appropriate outcome measures is vital for paediatric inflammatory bowel disease (IBD) trials. Standardized measures facilitate drug approval and reduce off-label medication use in children.
Area of Science:
- Pediatric Gastroenterology
- Clinical Trial Design
- Inflammatory Bowel Disease (IBD) Research
Background:
- Paediatric-onset inflammatory bowel disease (IBD) is increasingly common, yet approved medications for children are limited.
- Clinical trials in children face unique challenges, necessitating optimized outcome measures for successful drug development and approval.
Purpose of the Study:
- To establish key factors and address paediatric-specific issues for conducting optimal clinical trials in paediatric IBD.
- To define appropriate outcome measures that can facilitate patient recruitment and expedite drug approval for paediatric IBD.
Main Methods:
- An international expert panel convened by the Paediatric European Crohn's and Colitis Organisation (ECCO) committee.
- A literature search followed by a modified Delphi process to determine consensus on outcome measures.
- Recommendations required endorsement by at least 80% of the expert panel.
Main Results:
- Steroid-free mucosal healing (MH) is recommended as the primary outcome for new drug categories, with one to two endoscopies.
- Paediatric-specific disease activity indices (e.g., Paediatric Crohn's Disease Activity Index, Paediatric Ulcerative Colitis Activity Index) can serve as primary outcomes if MH is already established.
- Secondary outcomes include safety, MR enterography scores, faecal calprotectin, quality of life, and patient-reported outcomes.
Conclusions:
- Early paediatric trials for new IBD drugs are essential to minimize off-label prescribing in children.
- Selecting feasible and standardized outcome measures is critical for advancing paediatric IBD drug development.
Objective:
Although paediatric-onset IBD is becoming more common, few medications have a registered paediatric indication. There are multiple hurdles to performing clinical trials in children, emphasising the importance of choosing an appropriate outcome measure, which can facilitate enrolment, and thereby also drug approval. The aim of this consensus statement is to highlight paediatric specific issues and key factors critical for the optimal conduct of paediatric IBD trials.
Design:
The Paediatric European Crohn's and Colitis Organisation (ECCO) committee has established an international expert panel to determine the best outcome measures in paediatric IBD, following a literature search and a modified Delphi process. All recommendations were endorsed by at least 80% agreement.
Results:
Recognising the importance of mucosal healing (MH), the panel defined steroid-free MH as primary outcome measure for all drugs of new category with one or two postintervention endoscopies per trial (at 8-12 weeks and/or 54 weeks). Since endoscopic evaluation is a barrier for recruitment in children, trials with medications already shown to induce MH in children or adults, could use paediatric-specific disease activity scores as primary outcome, including a modified Paediatric Crohn's Disease Activity Index in Crohn's disease and the Paediatric Ulcerative Colitis Activity Index in UC. Secondary outcomes should include safety issues, MR enterography-based damage and inflammatory scores (in Crohn's disease), faecal calprotectin, quality of life scales, and a patient-reported outcome.
Conclusions:
It is crucial to perform paediatric trials early in the development of new drugs in order to reduce off-label use of IBD medication in children. The thoughtful choice of feasible and standardised outcome measures can help move us towards this goal.
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