In Vietnam, an estimated 6 million people live with rare diseases, about 58% of whom are children, while nearly 30% of affected children die before the age of five due to delayed diagnosis or limited access to appropriate treatment.
Medical workers tend to a patient. (Illustrative photo: VNA)
The Medical Services Administration under the Ministry of Health on February 28 held a national event marking the International Rare Disease Day and a workshop on building a national rare disease list and financial solutions for orphan drugs in Vietnam.
The event, organised for the first time at the national scale, was attended by Deputy Minister of Health Tran Van Thuan, who is also Chairman of the National Medical Council and head of the Steering Committee for Strengthening Rare Disease Management in Vietnam, along with leading experts, central hospital representatives, support organisations and patient groups.
Laying policy foundation for specialised field
Addressing the event, Thuan stressed that the event is not merely a communication activity but a clear commitment by Vietnam’s health sector to a highly challenging field requiring systematic, long-term and humanitarian solutions.
Since its launch in 2008, the International Rare Disease Day, held on the last day of February, has been observed in more than 100 countries and territories. According to a 2025 resolution adopted by the World Health Assembly, over 7,000 rare diseases have been identified globally, affecting more than 300 million people. Most are genetic, around 70% begin in childhood, and many require lifelong treatment.
In Vietnam, an estimated 6 million people live with rare diseases, about 58% of whom are children. Nearly 30% of affected children die before the age of five due to delayed diagnosis or limited access to appropriate treatment.
These figures underscore that rare diseases must be recognised as a national health policy priority, not isolated cases within individual specialties, he said.
In recent years, he noted, the Ministry of Health has improved the legal framework. The 2016 Pharmacy Law and related regulations established a list of orphan drugs, while Circular 01/2025 guiding the enforcement of the Health Insurance Law allows patients with severe or rare diseases to access care without referral procedures.
In 2025, the Steering Committee for Strengthening Rare Disease Management was consolidated, and an action plan for 2025–2026 was issued, targeting the promulgation of a national rare disease list this year and the development of an appropriate financial mechanism for orphan drugs.
Tackling legal, financial bottlenecks
The subsequent workshop focused on two major bottlenecks – legal and financial frameworks.
Thuan emphasised that without a standardised, evidence-based and context-appropriate national list, it will be difficult to design reimbursement policies, allocate resources or ensure equitable access to treatment.
He called for discussions around three pillars: finalising the national rare disease list based on scientific criteria and international experience tailored to Vietnam’s epidemiological and resource conditions; establishing sustainable financing mechanisms for orphan drugs, including risk-sharing models, price negotiations and closer coordination between the Ministry of Health and Vietnam Social Security; and developing a specialised network with reference centres, expanded genetic testing, standardised treatment protocols, continuous training and a national rare disease database.
Representatives from relevant departments, research institutes and leading hospitals joined the dialogue, proposing solutions to expand health insurance coverage and mobilise social resources transparently to improve patient access to advanced therapies at reasonable costs.
Nguyen Trong Khoa, Deputy Director of the Medical Services Administration, affirmed that completing the draft national rare disease list based on scientific and practical criteria will provide the cornerstone for standard treatment protocols and more effective support for patients nationwide./.
VNA