Presentation (CTI)

OBJECTIVES: To examine the policy, funding, infrastructure, and governance requirements necessary to support equitable and sustainable access to gene therapies and orphan drugs across MENA health systems.
METHODS: A structured qualitative expert-consensus study was conducted following COREQ guidelines, involving a purposive sample of 15 experts, including regulators, payers, clinicians, health economists, and patient representatives. Consensus was established via iterative polling (≥85% agreement), and findings were synthesized using directed content analysis across domains including genomic infrastructure, financing, patient engagement, and real-world evidence (RWE) generation.
RESULTS: Foundations: 100% of experts identified early diagnosis via genomic testing, newborn screening, and integrated data systems as foundational for sustainability. Adaptive HTA: 95% of participants agreed that conventional HTA is insufficient for rare disease therapies; they endorsed adaptive frameworks incorporating lifecycle evidence and RWE generation. Financing: 95% of experts prioritized outcome-based reimbursement, managed entry agreements, and risk-sharing arrangements to balance affordability and system sustainability. Governance & Engagement: Formal patient participation in HTA was endorsed by 90% of experts. Additionally, 100% of participants agreed that rare disease registries, regional data-sharing, and coordinated governance are critical for evidence-informed decision-making.
CONCLUSIONS: Sustainable access to rare therapies rests on adaptive HTA using lifecycle and real-world evidence for faster, smarter decisions; innovative risk-sharing financing that balances uncertainty, value, and affordability; and coordinated governance driven by patient engagement and regional collaboration. Investment in genomic infrastructure and integrated data ecosystems is the critical enabler for scalable, equitable access and durable health system resilience.