P0842 Paediatric Inflammatory Bowel Diseases: novel agents on the therapeutic horizon
Notice bibliographique
Résumé
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approval, with a median delay of over seven years to paediatric approval (Figure 1).1 With the recent circulation of draft guidance for industry on drug development in paediatric IBD (pIBD) by the US Food and Drug Administration (FDA),2 an opportunity exists to improve clinical trial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
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,005 | 0,014 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,002 | 0,001 |
| Bibliométrie | 0,005 | 0,006 |
| Études des sciences et des technologies | 0,000 | 0,001 |
| Communication savante | 0,003 | 0,003 |
| Science ouverte | 0,001 | 0,001 |
| Intégrité de la recherche | 0,001 | 0,002 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,016 | 0,003 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».