Title :

Comparison of treatment effect between phase 2 and phase 3 trials in patients with inflammatory bowel disease.

Author(s) :

Wils, Pauline [Auteur]
Hôpital Claude Huriez [Lille]
Institute for Translational Research in Inflammation - U 1286 [INFINITE]
Jairath, Vipul [Auteur]
University of Western Ontario [UWO]
Sands, Bruce E. [Auteur]
Icahn School of Medicine at Mount Sinai [New York] [MSSM]
Magro, Fernando [Auteur]
Hospital de São João [Porto]
Reinisch, Walter [Auteur]
Medizinische Universität Wien = Medical University of Vienna
Rubin, David [Auteur]
The University of Chicago Medicine [Chicago]
Danese, Silvio [Auteur]
IRCCS Ospedale San Raffaele [Milan, Italy]
Baumann, Cédric [Auteur]
Centre Hospitalier Régional Universitaire de Nancy [CHRU Nancy]
Peyrin-Biroulet, Laurent [Auteur]
Centre Hospitalier Universitaire de Nancy [CHU Nancy]

Journal title :

United European Gastroenterology Journal

Abbreviated title :

United European Gastroenterol J

Publication date :

2023-09-11

ISSN :

2050-6414

English keyword(s) :

biologics
Crohn's disease
inflammatory bowel disease
randomized controlled trial
ulcerative colitis

English abstract : [en]

Background and Aims The accumulation of multiple randomized controlled trials in the field of inflammatory bowel diseases provides an opportunity to compare treatment effects between phase 2 and 3 trials. We aimed to ...
Show more >Background and Aims The accumulation of multiple randomized controlled trials in the field of inflammatory bowel diseases provides an opportunity to compare treatment effects between phase 2 and 3 trials. We aimed to determine whether treatment effects observed in phase 3 investigating biologics and small molecule drugs differed from those in their preceding phase 2 trial. Methods We first performed a review of phase 2 and phase 3 trials enrolling ulcerative colitis (UC) or Crohn's disease (CD) patients. We compared the percent overall success for key endpoints between phases (several phase 3 could be matched to a single phase 2 trial). Then, we compared the percent overall success in the matched phase 2 and 3 trials (ratio 1:1), and performed sensitivity analysis. Results We identified 14 phase 2 (8 CD; 6 UC) and 24 phase 3 (13 CD; 11 UC) trials. In CD, the different analyses suggest that the percentage of overall success of clinical remission and clinical response was significantly higher in phase 2 than in phase 3 trials. In UC, the analyses suggest collectively that the percent of treatment effect seemed similar for clinical remission, clinical response and histologic outcomes between phases but with a lower percentage of overall success in phase 2 than in phase 3 trials for endoscopic endpoints. Conclusions In UC, we observed a similar percentage of treatment effect for clinical and histologic outcomes between phase 2 and 3 trials but not for endoscopic outcomes. Whereas in CD, we showed a failure to reproduce similar results between phases. These results may help sponsors in the design of future drug development programs.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Submission date :

2024-02-01T22:03:57Z
2024-03-11T14:32:54Z