Research Gaps and Priorities for aHUS in the Gulf Cooperation Countries (GCC): Findings from a Payer-Focused Expert Meeting

Abstract: Atypical hemolytic uremic syndrome (aHUS) is a rare, life-threatening complement-mediated thrombotic microangiopathy (TMA) associated with high morbidity, mortality, and substantial healthcare resource utilization (HCRU). Although complement inhibitors such as eculizumab and ravulizumab have dramatically improved outcomes, aHUS continues to impose significant economic and operational burden on healthcare systems. In the Gulf region, payers and health systems face increasing pressures driven by rising costs, delayed diagnosis, fragmented access, and limited real-world evidence necessary for informed resource allocation decisions. This manuscript summarizes the findings of a payer-focused regional expert meeting, which included three presentations: (1) the role and value of HCRU studies for payers, (2) the clinical and economic burden of aHUS, and (3) existing global evidence on HCRU in aHUS and gaps relevant to the Gulf. The meeting concluded with an expert panel discussion and a survey assessing feasibility and priorities for a collaborative real-world study. Consensus highlighted urgent needs to establish regional incidence and prevalence, quantify utilization and cost burden, evaluate treatment patterns and outcomes, and design a standardized multicenter study. Priority research directions include retrospective chart review with prospective follow-up and unified data collection across Gulf institutions. These findings provide a payer-informed roadmap for building evidence that can support sustainable, equitable, and value-based decision making for aHUS care in the region. Keywords: rare diseases, reimbursement, health technology assessment, formulary, orphan drugs, aHUS, research gaps, Gulf