Rare disease management in APAC faces challenges from inconsistent definitions, lack of epidemiological data, limited HCP awareness, delayed diagnoses, few treatment options, and high costs. Patients often wait years for diagnosis, with only 5% of rare diseases having therapies. Countries are responding with registries, newborn screening, education programs, innovative funding models, and managed entry agreements. Advocacy groups are expanding roles, while AI, genetic testing, and data platforms advance drug discovery and personalized care. Progress depends on collaboration, affordability, and patient empowerment to improve access and outcomes across the region.

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