Development of Standard Core Clinical Outcomes Assessments (COAs) and Endpoints (UG3/UH3 Clinical Trial Optional)

The purpose of this Funding Opportunity Announcement (FOA) is to solicit applications for UG3/UH3 cooperative agreements to support the development of a publicly available core set(s) of COAs and their related endpoints for specific disease indications or for disease impacts that span multiple rare diseases of similar phenotypes.

The standard core set(s) can include different types of COAs (i.e. patient-reported outcome [PRO], clinician-reported outcome [ClinRO], observer-reported outcome [ObsRO], performance outcome [PerfO] instruments) and their related endpoints that assess a minimum list of impacts that matter most to patients, are likely to demonstrate change (including differences in trial arms related to disease burden, treatment burden, and if applicable, physical function) and should be reported in a clinical trial. A standard core set might be relevant across several disease populations or subgroups or be focused on attributes of a specific disease (a disease-specific or generic instrument that can adapted and applied more broadly across disease areas or across age groups).

The UG3/UH3 Phase Innovation Award Cooperative Agreement involves 2 phases. The UG3 phase will provide funding for 1 to 2 years to conduct planning activities. The UH3 phase will provide funding for 3 to 4 years to projects that successfully complete the planning activities and reach the projected milestones set in the UG3 phase.