Presentation (CTI)

OBJECTIVES: Health technology assessment (HTA) systems across Gulf Cooperation Council (GCC) countries are evolving rapidly, yet traditional HTA approaches often fail to capture patient centered outcomes relevant to rare diseases. This study developed the first consensus-derived framework for patient engagement (PE) in rare disease HTA across GCC countries.
METHODS: A multiphase mixed-methods study combined a targeted literature review, stakeholder mapping, and a one-day consensus workshop using a modified Nominal Group Technique with anonymous digital voting. Stakeholders with leading HTA roles across GCC countries were purposively recruited, including regulators, policymakers, payers, HTA experts, clinicians, and researchers. Participants evaluated PE mechanisms across key HTA phases: topic identification and scoping, evidence generation, assessment and appraisal, and dissemination and monitoring. Contextual implementation factors were considered across all stages. Consensus was predefined as ≥70% agreement in the highest rating categories.
RESULTS: Fifteen stakeholders from four GCC countries (Saudi Arabia, the United Arab Emirates, Qatar, and Bahrain) participated. Strong consensus supported integrating patient-reported outcome measures (100%), caregiver-reported outcomes (86%), and patient-reported experience measures (71%) into evidence generation for rare disease HTA. High agreement was also reached for patient involvement in post-adoption monitoring (87%) and co-development of dissemination materials (77%). Lower consensus for patient membership on HTA committees (47%), review of draft HTA reports (13%), and independent patient submissions (13%) highlighted variability in institutional readiness and feasibility across GCC systems. Contextual factors, including GCC organizational and policy environments, cultural and societal factors (language barriers, confidentiality concerns, etc.) and nascent patient advocacy groups, were considered important determinants of PE feasibility. The resulting framework consolidated stakeholder-prioritized PE elements across all key HTA phases.
CONCLUSIONS: This study provides the first GCC consensus framework for integrating PE into rare disease HTA, supporting HTA bodies in more patient-centered assessments across the region. Broader validation, including Kuwait and Oman, would further strengthen regional alignment and implementation readiness.