Notably, 58% of rare diseases occur in children because most of them are of genetic origin, and 30% of children with rare diseases die before the age of 5 due to not being treated promptly. Most of the rare diseases today do not have treatment. Many patients have to rely on supportive therapies to control symptoms to improve their quality of life.
For many years, rare diseases have always been a medical challenge for families of children with rare diseases, because the treatment burden is 5-10 times higher than that of common diseases. For example, Pompe's disease has serious health consequences and requires a specific treatment regimen. The incidence rate ranges from 1/40,000 - 1/140,000, possibly 1/200,000 (depending on the region).
Pompe is a rare genetic disease due to a deficiency of alpha glucosidase enzyme, causing neurological disorders that gradually lead to disability, even death due to early-onset heart failure in the body. Children with this disease need to be given medicine for life, every 1-2 weeks to maintain their health, the total cost is up to billions of VND per year. In particular, many pediatric patients in the province, when detected with the disease, need to travel weekly/monthly to the National Children's Hospital (Hanoi), Children's Hospital 1 (Ho Chi Minh City) ... to check their health and access treatment drugs.
However, not every child with rare diseases is lucky enough to be diagnosed with the disease correctly and promptly to have the opportunity to live healthy. Many families have children with rare diseases in very difficult circumstances, while the cost of rare disease therapies is too high and requires long-term treatment, making it difficult to access treatment drugs. In addition, people's awareness of the risk of disease and disease is still limited. Therefore, any positive changes in policies to improve diagnosis, treatment and management of rare diseases bring hope to children, reduce pain as well as share the burden of payment with their families.
From 2025, Vietnam will record many systematic positive signals, as rare diseases begin to be put at the center of the national health strategy. One of the most obvious changes comes from the milestone of establishing the " Steering Committee for Strengthening Rare Disease Management", according to Decision 337/QD-BYT dated January 25, 2025 of the Minister of Health. The " Steering Committee for Strengthening Rare Disease Management" will play a role in advising, coordinating and supervising the implementation of the plan for the Ministry of Health, ensuring that rare disease management policies are implemented effectively and promptly.
Along with efforts to perfect the personnel for this field, the health sector is also gradually perfecting policies related to rare diseases. Recently, the Ministry of Health and the Vietnam Medical Association coordinated to organize a scientific seminar to deploy the "National Action Plan on Rare Disease Management 2025 - 2026" on September 16, 2025.
The "National Action Plan on Rare Disease Management 2025-2026" approved under Decision 2885/QD-BYT dated September 12, 2025 of the Ministry of Health shows that the health sector is committed to accompanying domestic and foreign organizations, all joining hands for people with rare diseases according to the recommendations of the World Health Organization (WHO), so that no one is left behind. These signals show the deep concern and commitment to synchronous support of the health sector for rare disease patients.
Therefore, with policies to support and facilitate the diagnosis and treatment of rare diseases in Vietnam, we believe that with the cooperation of relevant agencies, health experts, and the business community, we can build a fair, humane and inclusive healthcare system, thereby adding hope and improving the quality of life for rare disease patients.
