Preparing a clinical outcomes assessment set for nephrotic syndrome [Prepare-NS]

We are proposing a 5-year plan, encompassing the UG3 (2 years) and UH3 (3 years) phases, in which we will develop and establish the psychometric properties of a core set of clinical outcome assessments (COAs) for nephrotic syndrome, with primary focus on hypervolemia. We will use the UG3 phase to convene a diverse team of experts and stakeholders, in cooperation with the FDA, and develop the core set of COAs. UG3 activities will likely include, but not be limited to, systematic literature review, qualitative interviews with patients and clinicians, and identification of the most appropriate instrument instruments to capture the impacts of hypervolemia that are most relevant to patients and that are most relevant as therapeutic targets for hypervolemia treatments. The UH3 will focus on testing and implementing the core set of COAs identified in the UG3 phase. The research conducted in the UH3 phase will also allow us to refine the hypervolemia COAs. The final activities will be focused on making our final core set of COAs widely available to the public for use in trials. generate a core set of highly-relevant COAs that will advance patient-focused drug development in nephrotic syndrome.