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. 2024 Aug 14;18(8):1222-1231.
doi: 10.1093/ecco-jcc/jjae026.

Pragmatic Trial Design to Compare Real-world Effectiveness of Different Treatments for Inflammatory Bowel Diseases: The PRACTICE-IBD European Consensus

Massimo Claudio Fantini  1 , Gionata Fiorino  2 , Agostino Colli  3 , David Laharie  4 , Alessandro Armuzzi  5   6 , Flavio Andrea Caprioli  7   8 , Javier P Gisbert  9 , Julien Kirchgesner  10 , Fabio Salvatore Macaluso  11 , Fernando Magro  12 , Subrata Ghosh  13 ; PRACTICE-IBD study group
Collaborators, Affiliations

Pragmatic Trial Design to Compare Real-world Effectiveness of Different Treatments for Inflammatory Bowel Diseases: The PRACTICE-IBD European Consensus

Massimo Claudio Fantini et al. J Crohns Colitis. .

Abstract

Background and aims: Pragmatic studies designed to test interventions in everyday clinical settings can successfully complement the evidence from registration and explanatory clinical trials. The European consensus project PRACTICE-IBD was developed to identify essential criteria and address key methodological issues needed to design valid, comparative, pragmatic studies in inflammatory bowel diseases [BDs].

Methods: Statements were issued by a panel of 11 European experts in IBD management and trial methodology, on four main topics: [I] study design; [II] eligibility, recruitment and organisation, flexibility; [III] outcomes; [IV] analysis. The consensus process followed a modified Delphi approach, involving two rounds of assessment and rating of the level of agreement [1 to 9; cut-off ≥7 for approval] with the statements by 18 additional European experts in IBD.

Results: At the first voting round, 25 out of the 26 statements reached a mean score ≥7. Following the discussion that preceded the second round of voting, it was decided to eliminate two statements and to split one into two. At the second voting round, 25 final statements were approved: seven for study design; six for eligibility, recruitment and organisation, flexibility; eight for outcomes; and four for analysis.

Conclusions: Pragmatic, randomised, clinical trials can address important questions in IBD clinical practice, and may provide complementary, high-level evidence, as long as they follow a methodologically rigorous approach. These 25 statements intend to offer practical guidance in the design of high-quality, pragmatic, clinical trials that can aid decision making in choosing a management strategy for IBDs.

Keywords: Pragmatic clinical trials; consensus procedure; inflammatory bowel diseases.

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Conflict of interest statement

None declared by all the authors with respect to the content of this paper.

Figures

Figure 1
Figure 1
Graphic representation of steps and outputs leading to the development of consensus statements. SC, Steering Commitee [N = 5]; SB, Scientific Board [N = 6]; VP, Voting Panel [N = 18].
See this image and copyright information in PMC

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