OMERACT (The Outcome Measures in Rheumatology initiative) has endorsed core outcome domains that should be reported in gout trials (serum urate, gout flares, tophus burden, pain, patient global assessment, HR-QOL and activity limitation). Many of the domains also have endorsed measurement tools. However there are no recommendations on how or when these outcomes should be reported.
Our aim is to develop recommendations for how and when these core outcome domains should reported in gout urate-lowering therapy trials.
We will be running an online delphi survey (3 rounds of questionnaires) for patients, clinicians, researchers, and other healthcare professionals and stakeholders with an interest or expertise in gout worldwide. Patient participants will be recruited via participating clinics, as well as via international email lists held by 2 organisations; OMERACT and G-CAN. Non-patient participants will also be invited via these email lists. We will ask participants to rate possible ways of reporting the domains, as well as possible timepoints, on a scale of 1-9. Participants will also be able to suggest additional items to be included in subsequent survey rounds. This survey will be run via Qualtrics, which conforms with data privacy laws. The survey is partially de-identified - we need to collect email address of those who participate to do the secondary rounds. Participant background (e.g. patient, clinician, other health professional etc), age bracket, gender, country of origin and email for further survey rounds.
Using the data from this survey, we will develop a set of recommendations on how and when the OMERACT
core outcome domains should be reported in urate-lowering therapy trials.
Ellen Prendergast- University of Otago, Christchurch
Lisa Stamp- University of Otago, Christchurch
Nicola Dalbeth- University of Auckland
Robin Christensen- 1. Section for Biostatistics and Evidence-Based Research, the Parker Institute, Bispebjerg and Frederiksberg Hospital, Copenhagen, Denmark. 2. Research Unit of Rheumatology, Department of Clinical Research, University of Southern Denmark, Odense University Hospital, Denmark.
Disease Category: Rheumatology
Disease Name: Gout
Age Range: 18 - 120
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (patients)
- Methodologists
- Patient/ support group representatives
- Pharmaceutical industry representatives
- Policy makers
- Regulatory agency representatives
- Researchers
- Service commissioners
- Service providers
- Service users
- Recommendations for outcome measures (measurement/how)
- Delphi process
We will undertake a delphi survey. For each core domain there will be 2 questions; one asking how the domain should be measured and one question asking when the domain should be measured and reported. The lists of possible options for each were developed using the ways that the core domains are currently measured and reported in the literature. The options for questions of ‘how’ the core domains should be reported were collated using the findings from Morillon et al. (4). To develop options for time points for measuring and reporting the core domains (the ‘when’), studies included in Morillon et al from 2005 onwards were reviewed. Studies published prior to 2005 were excluded from analysis as this was prior to the requirement of registration with a clinical trials registry. Data was manually extracted from these trials by EP. Trial characteristics including the phase and the length of trial and year of publication were collected. Each trial was reviewed for use of the OMERACT core outcomes. Data was extracted on the core domains including whether it was a primary/secondary or other endpoint, the number of times it was measured and frequency it was measured. Data was also be collected on whether the measurements are reported. From this, common measurement frequencies for each of the core domains was identified.
For the delphi survey we will be recruiting two groups of participants; a patient group, which will include patients over the age of 18 years old with a diagnosis of gout, and a group of other stakeholders (e.g. clinicians, researchers) with gout expertise. We will aim to internationally recruit 200 participants (100 in each group), as per the OMERACT guidelines (2). These participants will be invited via email to undertake an online survey. Patient participants will be recruited via public advertising and also via an email list held by OMERACT. Non-patient participants will be invited using email lists held by OMERACT and G-CAN. Authors may also nominate other clinicians or researchers. Members of the working group will also be invited to participate. The final list of participants will be kept confidential, known only to the project team. Answers to the surveys will de-identified. Ethics will be applied for via University of Otago Human Ethics Committee.
The Delphi procedure will be run in 2025. There will be 3 rounds of surveys, with each round lasting 2-4 weeks. All participants notified of each round via an email to the email address provided on registration. The survey will be carried out on the Qualtrics platform. The survey will ask participants to score the importance of each item when undertaking or reviewing a gout trial. There will be questions asking both ‘how’ and ‘when’ each core domain should be reported. Where applicable, information will be given regarding what is currently being done in the literature. During each survey, participants will be asked to score each item on a scale of 1-9, with 1 labeled “not important” and 9 labeled “critical” (2,8). In round 1 participants are invited to make comments on items presented and suggest additional items to be included in round 2. Round 2 will include all round 1 items, as well as the additional items suggested by participants in round 1 and agreed upon by the working group. Round 3 will be a ranking exercise where participants are invited to rank the options presented.