Background Measurement of treatment effects in systemic lupus erythematosus (SLE) randomised controlled trials (RCTs) remains challenging. Current RCT endpoints are not based on contemporary outcome assessment methodology. We report the current status of a global academic-industry-patient partnership aiming to develop a novel, patient-centred clinical outcome assessment (COA) for SLE RCTs: Treatment Response Measure for SLE (TRM-SLE).
Methods We convened a global Taskforce of clinical-academic, metrology and regulatory experts, patient representatives and experts from ten pharmaceutical companies. A study protocol outlining a structured process for COA development was drafted and refined by TRM-SLE Taskforce members (figure 1), with reference to updated regulatory guidance. We next defined the high-level measurement goals for TRM-SLE using formal consensus methods, first by generating a conceptual definition using the PICO-C framework then establishing the context of use. Taskforce members participated in moderated discussion and real-time voting via virtual platforms with a pre-defined consensus threshold of 70% agreement.
Results A panel of 45 Taskforce members representing key stakeholder groups formulated the conceptual definition and context of use for TRM-SLE. Moderated discussion and multiple rounds of voting resulted in high agreement (81–100%, table 1). The current project phase (Aim 1.3) will establish consensus on the domain-level concepts to be measured by TRM-SLE. To date, TRM-SLE Taskforce members, including patients, have nominated 64 concepts, which have been grouped into a core list of 34 candidate domains.
Conclusions A global academic-industry-patient partnership has completed the first steps towards developing a novel SLE COA that is a quantitative measure of treatment response. Subsequent steps will combine consensus techniques with evaluation of supporting evidence, to determine the final set of concepts and associated measurements to be included in TRM-SLE. These measures will be integrated as a multi-domain COA in an SLE RCT endpoint that will be validated in clinical trials.
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