Stakeholder Mapping and Policy Assessment of Rare Diseases in Southeast Asia
Author(s)
Anand Jha, MBA1, Monica Verma, MPH2, Vinita Devjani, MBA3.
1Ansea Consultants Pte Ltd, Basel, Switzerland, 2Ansea Consultants Pte Ltd, Singapore, Singapore, 3Ansea Consultants Pte Ltd, Ahmedabad, India.
1Ansea Consultants Pte Ltd, Basel, Switzerland, 2Ansea Consultants Pte Ltd, Singapore, Singapore, 3Ansea Consultants Pte Ltd, Ahmedabad, India.
OBJECTIVES: Despite affecting over 300 million people globally, rare diseases remain under-prioritized, especially in low- and middle-income countries. In Southeast Asia, patients face delayed diagnoses, limited access, and fragmented care. This study examined six countries in Southeast Asia including, Indonesia, Malaysia, Philippines, Singapore, Thailand and Vietnam to identify access gaps, map key stakeholders, and define key priorities for patient-centered rare disease care.
METHODS: A comprehensive review of literature of peer-reviewed journal articles, policy documents, technical reports was conducted across six Southeast Asian countries. Six semi-structured interviews with key stakeholders were conducted to augment the data gaps of secondary research . A standardized typology was developed to guide the data collection, which included five key domains: policy landscape, funding analysis, advocacy activities, diagnostics solutions, and campaign and engagement. National rare disease policies, treatment and testing guidelines and access frameworks were reviewed. The findings from primary and secondary research were triangulated and benchmarked against global best practices to develop evidence-informed recommendations for the way forward.
RESULTS: Southeast Asia shares key barriers in rare disease care: fragmented policies, inconsistent reimbursement, low awareness, weak advocacy, and regulatory policies that deter access to orphan drugs. Access to genetic testing and specialist care is limited. Still, early policy initiatives and stakeholder collaborations are emerging. Stakeholders called for more funding, unified policies, and inclusive decision-making. Five strategic priorities were identified: national policy, stakeholder engagement, funding and access, genetic services, and capacity building.
CONCLUSIONS: This research underscores the need for regional collaboration and patient-centered approaches to rare disease policy in Southeast Asia, aiming to overcome systemic barriers and improve equitable access to diagnosis, care, and treatment.
METHODS: A comprehensive review of literature of peer-reviewed journal articles, policy documents, technical reports was conducted across six Southeast Asian countries. Six semi-structured interviews with key stakeholders were conducted to augment the data gaps of secondary research . A standardized typology was developed to guide the data collection, which included five key domains: policy landscape, funding analysis, advocacy activities, diagnostics solutions, and campaign and engagement. National rare disease policies, treatment and testing guidelines and access frameworks were reviewed. The findings from primary and secondary research were triangulated and benchmarked against global best practices to develop evidence-informed recommendations for the way forward.
RESULTS: Southeast Asia shares key barriers in rare disease care: fragmented policies, inconsistent reimbursement, low awareness, weak advocacy, and regulatory policies that deter access to orphan drugs. Access to genetic testing and specialist care is limited. Still, early policy initiatives and stakeholder collaborations are emerging. Stakeholders called for more funding, unified policies, and inclusive decision-making. Five strategic priorities were identified: national policy, stakeholder engagement, funding and access, genetic services, and capacity building.
CONCLUSIONS: This research underscores the need for regional collaboration and patient-centered approaches to rare disease policy in Southeast Asia, aiming to overcome systemic barriers and improve equitable access to diagnosis, care, and treatment.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR189
Topic
Health Policy & Regulatory, Health Service Delivery & Process of Care, Medical Technologies
Topic Subcategory
Health Disparities & Equity, Public Spending & National Health Expenditures, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases