P0842 Paediatric Inflammatory Bowel Diseases: novel agents on the therapeutic horizon
Bibliographic record
Abstract
Abstract Background The therapeutic landscape for inflammatory bowel disease (IBD) has advanced considerably over the past two decades with the development of monoclonal antibodies and advanced small molecules. However, only one advanced therapy class is approved for children with IBD. Long delays exist following adult approval, with a median delay of over seven years to paediatric approval (Figure 1).1 With the recent circulation of draft guidance for industry on drug development in paediatric IBD (pIBD) by the US Food and Drug Administration (FDA),2 an opportunity exists to improve clinical trial processes for drug approval in pIBD. The aim of this study is to summarize the landscape of pIBD clinical trials through a review of trial registries. Methods We conducted a cross-sectional review of publicly accessible data from Clinicaltrials.gov and ClinicalTrialsRegister.eu to identify investigational therapeutic agents as well as approved therapeutic agents for the treatment of pIBD. All interventional pIBD studies (<18 years and/or included a paediatric population) listed from the database’s inception to May 13, 2024, were considered for inclusion. Results A total of 3,493 records were identified from Clinicaltrials.gov (2,758) and ClinicalTrialsRegister.eu (735). One-hundred and sixteen completed trials were included in the review. Of those completed trials, 34 studies focused on biologic agents (29%). Novel small molecule agents (spingosine-1-phosphate agonists and Janus kinase inhibitors) were examined in 7 studies (6%). A further 118 IBD trials are actively recruiting paediatric patients. Sixty-five studies are randomised controlled trials and 53 are open-label studies. With regards to biologic agents, 17 trials are exploring the use of approved biologic agents in children and 34 trials are focusing on novel biologic agents. These include etrolizumab, golimumab, guselkumab, mirikizumab, risankizumab, ustekinumab, and vedolizumab. Sixteen trials are recruiting, examining small molecule therapies in pediatrics, to include upadacitinib, tofacitinib, etrasimod, and ozanimod. Conclusion Efforts to hasten the approvals of novel agents in pIBD is paramount to ensure timely access to effective medications. Whilst there is increasing trial activity in the pIBD landscape, approval by regulatory bodies continue to pose barriers. Consideration for novel trial designs and collaboration between research networks could reduce the delay in paediatric marketing approvals. Continued engagement with regulatory bodies and the international pIBD community offers a critical opportunity to advance drug approvals in children with IBD. References 1.Crowley E, Ma C, Andic M, Feagan BG, Griffiths AM, Jairath V. Impact of Drug Approval Pathways for Paediatric Inflammatory Bowel Disease. J Crohns Colitis. 2022;16(2):331-5 2.U.S Food and Drug Administration. Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/pediatric-inflammatory-bowel-disease-developing-drugs-treatment 3.Created in BioRender. Suthar, N. (2024) BioRender.com/l43j981
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.005 | 0.014 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.001 |
| Bibliometrics | 0.005 | 0.006 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.003 | 0.003 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.016 | 0.003 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".