Multi-Stakeholder Approach to Rare Disease Care in Latin America

OBJECTIVES: This study aimed to understand the rare disease (RD) care approach in Argentina, Brazil, Chile, Colombia, Mexico, Peru and Uruguay. Specifically, to determine patient access to diagnosis and treatments from a multi-stakeholder perspective, identifying cross-regional best practices translatable to RD care strategies among countries.

METHODS: A task force was convened of independent panels comprising 5-6 experts per country including physicians, reimbursement and policy experts, and patient advocates. A briefing pack of global, regional, and country-specific materials was prepared from a targeted literature search. Questions regarding access to RD medicines were developed to guide the task force discussion. Results were compiled into country-level reports. A scorecard was developed to rank countries in terms of access to RD medicines, impact of medical societies on access, and impact of patient organizations on access.

RESULTS: A strong relationship was found between multi-stakeholder collaboration and improved access to RD medicines. The main challenges identified were fragmented healthcare systems, lack of knowledge of RDs at the primary care level, unclear treatment incorporation processes, and budget constraints. Key opportunities were RD awareness, adapted HTA processes, and innovative financing mechanisms. Experts scored Argentina, Brazil and Colombia as medium-high in terms of access, while Mexico and Peru had low access. The impact of medical societies on access in most countries was medium, except in Brazil and Uruguay (high), and Chile (low). The impact of patient organizations on access in most countries was high, except in Chile and Mexico (medium).

CONCLUSIONS: Robust regulatory frameworks, patient advocacy, multi-stakeholder collaboration, and innovative financing are associated with more effective adoption of high-cost RD treatments. Persistent systemic barriers in some countries highlight the uneven progress within the region. Greater engagement by authorities of the expertise of PAOs and medical societies is suggested in the development of effective diagnosis and treatment access pathways and collaborative policy development.