REGULATORY AND HEALTH TECHNOLOGY ASSESSMENT (HTA) ADAPTATIONS FOR HIGH-COST RARE-DISEASE MEDICINES IN LATIN AMERICA (LATAM): A CROSS-COUNTRY POLICY REVIEW
Author(s)
Sanowar Sayeed, M.Pharm1, Geetika Sharma, Masters of Science(MS)2, Monica Verma, MPH3, Anand Jha, MBA4.
1Ansea Consultants Pte Ltd, Greater Noida, India, 2Ansea Consultants Pte Ltd, Pune, India, 3Associate Director, Ansea Consultants Pte Ltd, Singapore, Singapore, 4Ansea Consultants Pte Ltd, Singapore, Singapore.
1Ansea Consultants Pte Ltd, Greater Noida, India, 2Ansea Consultants Pte Ltd, Pune, India, 3Associate Director, Ansea Consultants Pte Ltd, Singapore, Singapore, 4Ansea Consultants Pte Ltd, Singapore, Singapore.
OBJECTIVES: Rare-disease policies, regulatory routes and HTA evidence expectations are evolving across LATAM, creating diverse pathways for high-cost rare-disease medicines. We compared policy, regulatory and HTA/evidence adaptations across key LATAM countries.
METHODS: A targeted cross-country policy review covered Argentina, Brazil, Colombia, Costa Rica, Mexico, Panama, Peru and Uruguay. Scientific literature, government and regulatory portals, HTA agency reports, reimbursement documents and grey literature were reviewed, with emphasis on recent policy updates. A structured framework captured rare-disease policy, regulatory pathways, HTA evidence adaptations, multicriteria assessment, post-access evidence generation and access agreements. Findings were synthesized thematically to identify cross-country patterns and gaps.
RESULTS: Rare-disease policy recognition was broader than dedicated access infrastructure. Most countries had rare-disease laws, national policies, registries or formal definitions. Argentina, Brazil, Mexico, Panama and Peru had specific or facilitated regulatory routes, including special registration, orphan recognition, reliance, prioritized/accelerated regulatory review or reduced documentation. Colombia, Costa Rica and Uruguay mainly relied on standard medicine registration pathways, with exceptional import, reliance or urgent procedures available in selected circumstances. HTA adaptations were heterogeneous and often embedded within standard processes. Common adaptations included rare-disease prioritization for technical assessment, acceptance of non-randomized or real-world evidence, broader value considerations, multicriteria decision analysis, budget-impact emphasis, patient or societal input and post-access monitoring. Dedicated rare-disease HTA or reimbursement fast tracks were uncommon, creating a gap between policy recognition, adaptive evidence approaches and funded access.
CONCLUSIONS: LATAM health systems are moving from rare-disease policy recognition toward more adaptive regulatory and HTA approaches, but implementation remains uneven. These findings highlight the need to link accelerated regulatory pathways with explicit evidence standards, post-access data systems, and reimbursement implementation mechanisms.
METHODS: A targeted cross-country policy review covered Argentina, Brazil, Colombia, Costa Rica, Mexico, Panama, Peru and Uruguay. Scientific literature, government and regulatory portals, HTA agency reports, reimbursement documents and grey literature were reviewed, with emphasis on recent policy updates. A structured framework captured rare-disease policy, regulatory pathways, HTA evidence adaptations, multicriteria assessment, post-access evidence generation and access agreements. Findings were synthesized thematically to identify cross-country patterns and gaps.
RESULTS: Rare-disease policy recognition was broader than dedicated access infrastructure. Most countries had rare-disease laws, national policies, registries or formal definitions. Argentina, Brazil, Mexico, Panama and Peru had specific or facilitated regulatory routes, including special registration, orphan recognition, reliance, prioritized/accelerated regulatory review or reduced documentation. Colombia, Costa Rica and Uruguay mainly relied on standard medicine registration pathways, with exceptional import, reliance or urgent procedures available in selected circumstances. HTA adaptations were heterogeneous and often embedded within standard processes. Common adaptations included rare-disease prioritization for technical assessment, acceptance of non-randomized or real-world evidence, broader value considerations, multicriteria decision analysis, budget-impact emphasis, patient or societal input and post-access monitoring. Dedicated rare-disease HTA or reimbursement fast tracks were uncommon, creating a gap between policy recognition, adaptive evidence approaches and funded access.
CONCLUSIONS: LATAM health systems are moving from rare-disease policy recognition toward more adaptive regulatory and HTA approaches, but implementation remains uneven. These findings highlight the need to link accelerated regulatory pathways with explicit evidence standards, post-access data systems, and reimbursement implementation mechanisms.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA361
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Systems & Structure
Disease
Rare & Orphan Diseases