Development of a Core Outcome Set for Clinical Trials in Non-infectious Uveitis of the Posterior Segment

Abstract Purpose To develop an agreed set of outcomes known as a core outcome set (COS) for Non-Infectious Uveitis of the Posterior Segment (NIU-PS) clinical trials. Design Mixed-methods study design comprising a systematic review and qualitative study followed by a two round Delphi exercise and face-to-face consensus meeting. Participants Key stakeholders including: patients diagnosed with NIU-PS; their caregivers; healthcare professionals involved in decision-making for patients with NIU-PS including ophthalmologists, nurse practitioners and policymakers/commissioners. Methods A long list of outcomes was developed based on the results of (1) a systematic review of clinical trials of NIU-PS and (2) a qualitative study of key stakeholders including focus groups and interviews. The long list was used to generate a two-round Delphi exercise of stakeholders rating the importance of outcomes on a nine-point Likert scale. The proportion of respondents rating each item was calculated, leading to recommendations of ‘include’, ‘exclude’ or ‘for discussion’ that were taken forward to a face-to-face consensus meeting of key stakeholders at which the final COS was agreed. Main outcome measure Items recommended for inclusion in the COS for NIU-PS Results A total of 57 outcomes grouped in 11 outcome domains were presented for evaluation in the Delphi exercise, resulting in 9 outcomes directly qualifying for inclusion and 15 outcomes being carried forward to the consensus meeting of which 7/15 were agreed for inclusion. The final COS contained 16 outcomes organized into 4 outcome domains comprising visual function, Health Related Quality of Life (HRQoL), treatment side effects and disease control. Conclusion This study builds on international work across the clinical trials community and our qualitative research to construct the world’s first COS for NIU-PS. The COS provide a list of outcomes that represent the priorities of key stakeholders and provides a minimum set of outcomes for use in all future NIU-PS clinical trials. Adoption of this COS can improve the value of future uveitis clinical trials and reduce non-informative research. Some of the outcomes identified do not yet have internationally agreed methods for measurement and should be the subject of future international consensus development. Trial Registration The study was registered with COMET ( http://comet-initiative.org/studies/details/640 )