Core Outcome Set for Real-World Observational Data in Inflammatory Bowel Disease
Peter Bossuyt, ClinCom Member
Multiple collaborations exist between ECCO Members in the collection of clinical data in a real-world setting. These observational data are an elegant addition to data from randomised controlled trials, as they reflect the efficacy and safety of clinical interventions in a more heterogeneous population who may not be included in randomised controlled trails and take into account the specific hurdles and challenges of everyday care for patients with IBD. The utility of real-world data, however, is dependent on the quality and homogeneity of the reported data. Data collection standardisation may enhance the quality of real-world evidence.
In a joint effort co-ordinated by EpiCom and ClinCom, we aimed to develop a core outcome set for real-world studies in adult patients with IBD. This multi-step process was initiated in 2020. First, we performed a systematic review of the available real-world observational studies (Wong C, van Oostrom J, Bossuyt P, et al. A narrative systematic review and categorisation of outcomes in Inflammatory Bowel Disease to inform a Core Outcome Set for real-world evidence. J Crohns Colitis. 2022 May 5:jjac057. doi: 10.1093/ecco-jcc/jjac057. Online ahead of print). In total, 315 studies were included in this systematic review. We concluded that a high variability exists in outcomes and in instruments used in measuring these outcomes. This systematic review was the starting point for the next step, a Delphi consensus project on the core outcome set for real-world observational studies in adult patients with IBD. For this we categorised the outcomes in 12 categories. Following an open call to ECCO Members, a balanced group of clinicians, including epidemiologists, gastroenterologists, nurses, pathologists and surgeons, was assembled for the Delphi consensus to develop the core outcome sets. An additional two patient representatives from the European Federation of Crohn’s and Colitis Associations (EFCCA) were included, along with regulatory input from the European Medicine Agency (EMA). The panellists restructured the data from the systematic review prior to the start of the Delphi voting rounds. After two Delphi voting rounds, a final core outcome set was discussed at the consensus meeting, where the core outcome set was further refined prior to final voting. Major conclusions of the Delphi process are the fact that real-world observational studies should include biomarker and endoscopic data in addition to clinical indices that reflect adequately the symptoms of the patient. Suggestions were made for the Short Inflammatory Bowel Disease Questionnaire (SIBDQ) as an elegant patient-reported outcome measurement instrument. While treatment targets are evolving, with radiological and histological outcomes being adopted in randomised controlled trials, it was agreed in the consensus that currently these outcomes are too early to be included in a core outcome set for observational real-world studies.
The final paper on this core outcome set was submitted early September for peer-review publication. The adoption of this core outcome set will reduce heterogeneity of reporting in real-world studies and facilitate synthesis in the generation of real-world evidence.