Unlocking Access to Medicines for Rare Diseases in Southeast Asia

This white paper presents findings from the Southeast Asia Rare Disease Summit 2024, which convened to discuss the challenges faced by patients with rare diseases in the region. It outlines the burden of rare diseases, affecting approximately 45 million people in Southeast Asia, and the obstacles in accessing effective treatments. The document details the patient journey, highlighting the importance of early diagnosis through newborn screening and the role of advocacy groups in shaping healthcare policy. Specific case studies from countries like Singapore and Thailand illustrate successful strategies to improve access to medicines and streamline referral processes for patients. The paper emphasizes the need for healthcare systems strengthening initiatives and patient education to enhance the quality of care. It serves as a resource for stakeholders looking to implement effective solutions for rare disease management in Southeast Asia, showcasing examples of local initiatives that can be adapted across different healthcare contexts.