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

Laila Carolina Abu Esba,1– 3 Ghada Hussain AlMardawi,1,3 Abdulaziz Alhenaidi,4 Mohamed Ahmed,5 Abdullah O AlShehry,6 Waiel Al Naeem,7 Qasim Alshara,8 Omar Alkandari,4 Hussain Abdulrahman Al-Omar9 1Pharmaceutical Care Department, Ministry of National Guard - Health Affairs, Riyadh, Saudi Arabia; 2College of Pharmacy, King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia; 3King Abdullah International Medical Research Centre, Riyadh, Saudi Arabia; 4Department of Planning and Monitoring, Ministry of Health, Kuwait City, Kuwait; 5Formulary Management Department, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia; 6Pharmaceutical Care Department, King Fahd Medical City, Riyadh, Saudi Arabia; 7Pharmaceutical Care Department, Sheikh Khalifa Medical City, Abu Dhabi, United Arab Emirates; 8Pharmacy Department, Salmaniya Medical Complex, Manama, Bahrain; 9Department of Clinical Pharmacy, College of Pharmacy, King Saud University, Riyadh, Saudi ArabiaCorrespondence: Laila Carolina Abu Esba, Pharmaceutical Care Department, Ministry of National Guard - Health Affairs, Riyadh, 11426, Saudi Arabia, Tel +966 1 8011111 Extension No. 12555, Email [email protected]: 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