Presentation (CTI)

OBJECTIVES: High-cost rare disease medicines challenge health systems in LATAM, as small populations, high prices and evidence uncertainty can delay funded access. We compared how key markets finance these therapies and assessed the maturity of innovative funding and access mechanisms.
METHODS: A targeted cross-country review covered Argentina, Brazil, Colombia, Costa Rica, Mexico, Panama, Peru and Uruguay. Official legislation, HTA and reimbursement guidance, payer policies, procurement records, reimbursement lists, peer-reviewed literature and grey literature were reviewed. A predefined framework extracted dedicated funding, high-cost payer routes, procurement and negotiation tools, managed entry/risk-sharing agreements, exceptional access, litigation, monitoring and real-world evidence requirements. Findings were synthesized narratively by mechanism type and maturity.
RESULTS: A maturity gradient was identified in how systems organize financing and access for high-cost rare disease medicines. No standalone public funds dedicated exclusively to rare disease medicines were identified; access was generally financed through broader high-cost medicine budgets, social security arrangements or payer pathways. Countries with centralized financing or procurement architecture showed clearer levers for innovation. Uruguay and Peru had established high-cost financing routes, while Panama and Colombia showed emerging centralized procurement approaches for selected high-cost or orphan-disease medicines. These mechanisms created opportunities for price negotiation, eligibility control and budget management. More fragmented systems relied on multiple payer routes, institutional listing, exceptions or case-by-case access. Managed entry/risk-sharing agreement use was uneven: Argentina, Brazil and Uruguay showed case-based precedents involving confidential discounts, staged payments or risk-sharing approaches; Mexico and Costa Rica had less visible routine orphan-specific arrangements. Exceptional access and litigation remained important fallback routes, indicating formal pathways do not consistently deliver timely reimbursement.
CONCLUSIONS: Rare disease financing in LATAM is moving toward structured high-cost medicine management, but innovation access remains uneven and is more often procurement- or discount-based than outcomes-based. Strengthening capacity for managed access, monitoring/registries and pooled procurement remains a priority for equitable access.