Preparation and Rationale for a Patient-Centered Clinical Outcome Assessment Set of Fluid Overload for Drug Development in Nephrotic Syndrome.

Introduction: Fluid overload is a source of substantial morbidity for adults and children with nephrotic syndrome (NS). Preparation and Rationale for a Fluid Overload in Nephrotic Syndrome Clinical Outcomes Assessment Set for Drug Development (Prepare-NS, 5UG3FD007308) was funded by the US Food and Drug Administration to develop a core set of patient-reported and observer-reported (for young children) outcome measures of fluid overload for use in pharmaceutical trials across the lifespan.

Methods: The Prepare-NS study team developed the proposed context of use with input from stakeholders.