ASSESSING ACCESS TO INNOVATIVE ORPHAN MEDICINES IN LATIN AMERICA: A CROSS-COUNTRY VALUE AND ACCESS ANALYSIS
Author(s)
OSCAR COURTNEY, BSc1, Francisca Rodriguez, MD2, Silvana Lay Ma, Eng., MBA3, Diego F. Guarin, MPH, MSc, MD4.
1IQVIA, Mexico, Mexico, 2cif chile, santiago, Chile, 3ACCESS, FIFARMA, MEXICO, Mexico, 4FIFARMA, MEXICO, Mexico.
1IQVIA, Mexico, Mexico, 2cif chile, santiago, Chile, 3ACCESS, FIFARMA, MEXICO, Mexico, 4FIFARMA, MEXICO, Mexico.
Presentation Documents
OBJECTIVES: To assess the availability and time to access of innovative orphan medicines across Latin America and identify system-level barriers affecting equitable access for rare disease patients.
METHODS: A total of 271 orphan medicines approved globally between 2014 and 2024 were analyzed across ten Latin American countries. Availability and time-to-access definitions were standardized to allow cross-country comparison. Extended availability was defined as the combined presence of full, limited, and private access. Data were collected through national pharmaceutical associations, research-based companies, and publicly available sources. Descriptive analyses were conducted.
RESULTS: Average extended availability of orphan medicines was 3%. Argentina and Colombia showed the highest availability (6%), while Ecuador, Costa Rica, the Dominican Republic, and Panama presented the lowest (1%). Time to availability ranged from 21 to 49 months. Benchmarking against European datasets highlights substantially higher availability levels outside the region.
CONCLUSIONS: Access to orphan medicines remains extremely limited across Latin America, reflecting structural constraints in regulation, financing, and HTA processes. Strengthening rare-disease-specific policies and coordinated multi-stakeholder action is essential to improve value-based decision-making and patient outcomes.
METHODS: A total of 271 orphan medicines approved globally between 2014 and 2024 were analyzed across ten Latin American countries. Availability and time-to-access definitions were standardized to allow cross-country comparison. Extended availability was defined as the combined presence of full, limited, and private access. Data were collected through national pharmaceutical associations, research-based companies, and publicly available sources. Descriptive analyses were conducted.
RESULTS: Average extended availability of orphan medicines was 3%. Argentina and Colombia showed the highest availability (6%), while Ecuador, Costa Rica, the Dominican Republic, and Panama presented the lowest (1%). Time to availability ranged from 21 to 49 months. Benchmarking against European datasets highlights substantially higher availability levels outside the region.
CONCLUSIONS: Access to orphan medicines remains extremely limited across Latin America, reflecting structural constraints in regulation, financing, and HTA processes. Strengthening rare-disease-specific policies and coordinated multi-stakeholder action is essential to improve value-based decision-making and patient outcomes.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR87
Topic
Health Policy & Regulatory
Topic Subcategory
Health Disparities & Equity, Reimbursement & Access Policy
Disease
SDC: Rare & Orphan Diseases