According to the Resolution on Rare Diseases adopted by the 78th World Health Assembly, there are currently more than 7,000 rare diseases recorded, affecting over 300 million people worldwide. About 70% of rare diseases originate in children, mostly due to genetic causes, chronic progression, complexity and requiring lifelong treatment and care.
According to data from the Ministry of Health, 6 million Vietnamese people live with about 100 rare diseases, of which 58% are children, and many children die due to late detection. Not only is it difficult to diagnose, rare diseases also pose a very large financial burden for patients and their families.
Common and characteristic rare diseases in Vietnam such as hemophilia: Patients lack blood clotting factors, are prone to bleeding.
Thalassemia: A common genetic disease, causing chronic anemia.
Lysosomal Storage Disorders: Including Pompe, Gaucher, Fabry, MPS diseases... affecting physical and intellectual development.
Xeroderma Pigmentosum: A strange disease that causes skin ulcers, common in some small areas.
Other diseases: Wilson's disease (copper metabolism disorder), multiple sclerosis, myasthenia gravis, lupus erythematosus, and rare cancers in children
According to Dr. Nguyen Trong Khoa - Deputy Director of the Department of Medical Examination and Treatment Management (Ministry of Health), in Vietnam, the average diagnosis journey lasts up to 7 years. These figures pose an urgent requirement: Rare diseases must be seen as a priority in national health policy planning, not just a separate story of each specialty. Treating and solving the problem of rare diseases requires a solid legal framework and close coordination between stakeholders.
One of the biggest bottlenecks today is treatment costs. Rare drugs are often very expensive, exceeding the affordability of many families. Currently, experts propose 4 key groups of solutions: legal completion; expansion of health insurance funds; application of risk sharing mechanisms; and strengthening socialization of resources.
Over the past time, the legal corridor for rare diseases has been gradually completed. The 2016 Law on Pharmacy has built a list of rare drugs; Circular 01. 2025 guiding the Health Insurance Law stipulates that people with dangerous diseases and rare diseases are not required to carry out referral procedures.
In 2025, the Ministry of Health issued the National Action Plan and established the Steering Committee for Strengthening Management of Rare Diseases in Vietnam. However, diagnostic capacity at the lower level is still limited; in-depth tests are not uniform; financial burdens still weigh heavily on patients' families.
February 28, 2026 was chosen by the European Organization for Rare Diseases (EURORDIS - Rare Diseaes Europe) as the International Rare Diseases Day because this is the rarest month of the year - the only month without 30 or 31 days, with the message "You can do more than you can imagine".
The long-term goal of the "Day of Rare Diseases" campaign is to achieve equal access to diagnosis, treatment, and health care for people affected by a rare disease.
