This information was provided by experts at the Scientific Symposium on Implementing the National Action Plan for Rare Disease Management 2025-2026, organized by the Vietnam Medical Association and the Ministry of Health (MOH) on September 16.

Globally, about 300 million patients suffer from approximately 6,000 types of rare diseases, accounting for 3.5 to 5.9 percent of the world’s population. In Vietnam, one in every 15 people is affected, equivalent to 6 million individuals.

Globally, there are approximately 300 million patients suffering from around 6,000 types of rare diseases, accounting for 3.5-5.9 percent of the world population. In Vietnam, 1 in every 15 people is affected, equivalent to 6 million individuals.

According to Ha Anh Duc, director of the Department of Medical Services Administration, Vietnam currently records about 100 rare diseases, with 58 percent of cases occurring in children. About 30 percent of children with rare diseases do not survive past the age of 5 due to limited access to effective treatment methods.

This reflects major challenges for the healthcare system, especially in the context of increasingly complex infectious and non-communicable diseases, coupled with limited healthcare resources. Therefore, a more comprehensive and effective strategy for rare disease management is urgently needed.

Vu Chi Dung, director of the Center for Endocrinology, Metabolism, Genetics, and Molecular Therapy at the National Children's Hospital, said diagnosing and treating rare diseases is extremely challenging due to the lack of information, limited number of specialists, and, most notably, a shortage of effective treatment methods.

It is estimated that around 40 percent of rare diseases are misdiagnosed, leading to delayed diagnosis, improper treatment, and high treatment costs. This puts patients with rare diseases at a significant disadvantage, both in terms of health and financial burden.

For example, at the National Children's Hospital, one patient underwent treatment for spinal muscular atrophy with a drug costing VND50 billion per dose. The medication, developed by a Swiss pharmaceutical group, was priced at $2.1 million per dose at launch, the most expensive drug in the world at the time, intended for children under the age of 2.

Nguyen Khanh Phuong, director of the Institute for Health Strategy and Policy, emphasized that rare diseases often lack standard treatment protocols, and treatment costs are typically very high. Medications are either scarce or expensive, making them inaccessible to many patients. This remains one of the biggest barriers to managing rare diseases in Vietnam.

One notable suggestion to address the issue is the creation of a "rare drug reserve" that healthcare facilities could collectively access, helping reduce costs and increase medication accessibility for patients.

Vo Thu