A Core Outcome Set for Inflammatory Bowel Diseases: Development and Recommendations for Implementation in Clinical Practice Through an International Multi-stakeholder Consensus Process

Abstract

Quality of life; Socioeconomical and psychological end points

Qualitat de vida; Aspectes socioeconòmics i psicològics

Calidad de vida; Aspectos socioeconómicos y psicológicos

Background and Aims Standardising health outcome measurements supports delivery of care and enables data-driven learning systems and secondary data use for research. As part of the Health Outcomes Observatory [H2O] initiative, and building on existing knowledge, a core outcome set [COS] for inflammatory bowel diseases [IBD] was defined through an international modified Delphi method. Methods Stakeholders rated 90 variables on a 9-point importance scale twice, allowing score modification based on feedback displayed per stakeholder group. Two consecutive consensus meetings were held to discuss results and formulate recommendations for measurement in clinical practice. Variables scoring 7 or higher by ≥80% of the participants, or based on consensus meeting agreement, were included in the final set. Results In total, 136 stakeholders (45 IBD patients [advocates], 74 health care professionals/researchers, 13 industry representatives, and four regulators) from 20 different countries participated. The final set includes 18 case-mix variables, three biomarkers [haemoglobin to detect anaemia, C-reactive protein and faecal calprotectin to detect inflammation] for completeness, and 28 outcomes (including 16 patient-reported outcomes [PROs] and one patient-reported experience). The PRO-2 and IBD-Control questionnaires were recommended to collect disease-specific PROs at every contact with an IBD practitioner, and the Subjective Health Experience model questionnaire, PROMIS Global Health and Self-Efficacy short form, to collect generic PROs annually. Conclusions A COS for IBD, including a recommendation for use in clinical practice, was defined. Implementation of this set will start in Vienna, Berlin, Barcelona, Leuven, and Rotterdam, empowering patients to better manage their care. Additional centres will follow worldwide.

This work was supported by the Innovative Medicines Initiative [IMI] 2 Joint Undertaking [945345-2]. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme, the European Federation of Pharmaceutical Industries and Associations [EFPIA], Trial Nation and the Juvenile Diabetes Research Foundation [JDRF]. The public grant funding is matched with in-kind contributions of EFPIA partners. About IMI: the Innovative Medicines Initiative is a partnership between the European Union and the European pharmaceutical industry, represented by EFPIA. It is working to improve health by speeding up the next generation of medicines, particularly in areas with unmet medical or social needs. It works by facilitating collaboration between the key players involved in health research, including universities, research centres, pharmaceutical and other industries, small and medium-sized enterprises [SMEs], patient organisations, and medicines regulators. IMI is the world’s most extensive public-private partnership [PPP] in the life sciences. This manuscript reflects only the authors’ views. The European Union and IMI are not responsible for any use that may be made of the information it contains.