Strengthening comprehensive rare disease management

A team of doctors performed a corneal transplant on a patient at Hai Phong Eye Hospital.

A team of doctors performed a corneal transplant for a patient at Hai Phong Eye Hospital.

According to statistics from the World Health Organization, there are currently more than 7,000 rare diseases worldwide, affecting about 300 million people. In Vietnam, it is estimated that there are about 100 rare diseases with 6 million people suffering and affected, of which 58% are children.

Notably, about 30% of children with rare diseases die before the age of 5 due to difficulty in accessing treatment. This number requires the Ministry of Health to have a strategy to strengthen the management of rare diseases more comprehensively and effectively in the context of increasingly complex infectious and non-communicable diseases, when health resources are still limited.

Currently, the diagnosis and treatment of rare diseases is difficult due to lack of information, lack of experts and effective treatment methods. Many cases of the disease are diagnosed late, treated incorrectly and at high cost.

Therefore, it is necessary to develop a national plan on rare diseases, including urgently developing and promulgating a list of rare diseases in Vietnam; establishing a national database and registration system for rare diseases.

Therefore, in early 2025, the Ministry of Health decided to establish a Steering Committee to strengthen and manage rare diseases as an advisory body as well as promote comprehensive improvement in rare disease management.

On September 12, the Ministry also approved the "Action Plan for Rare Disease Management 2025 - 2026", setting out key contents and tasks, including perfecting the institution in the field of rare disease management, such as: Issuing a list of rare diseases, updating standard guidelines on diagnosis, treatment and management of some rare diseases; amending the Circular to update the list of rare drugs; communicating to raise public awareness of rare diseases; promoting scientific research and international cooperation to support and improve capacity for management and treatment of rare diseases.

At the scientific seminar on implementing the Action Plan on rare disease management in 2025 - 2026, Director of the Department of Medical Examination and Treatment Management (Ministry of Health) Ha Anh Duc assessed: The seminar is not only an opportunity for leading experts to share experiences and knowledge, but also an opportunity to discuss and propose practical solutions to improve the quality of management, diagnosis and treatment of rare diseases in Vietnam.

Doctor Nguyen Trong Khoa, Deputy Director of the Department of Medical Examination and Treatment Management, said: The goal from now until the end of 2025 is to accept and include 30-40 rare diseases in the list and by 2026, issue a list of rare diseases in Vietnam.

To address the issue of rare diseases and improve the effectiveness of rare disease management, we need the participation of the entire political system and the social community. Building a policy requires vision and scientific evidence so that the 2025-2026 Action Plan on Rare Disease Management can be practically effective; at the same time, research on payment support mechanisms and policies to help patients access advanced treatments.

Associate Professor, Dr. Vu Chi Dung, Director of the Center for Endocrinology, Metabolism, Genetics and Molecular Therapy (National Children's Hospital) said that it is necessary to continue expanding diagnostic and treatment guidelines for rare diseases (currently there are about 40 guidelines); strengthen screening, diagnosis, treatment and management services with a long history and increasingly improving quality; urgently issue a temporary list of rare diseases; increase investment in diagnostic and treatment resources and training of human resources for the field of rare diseases.
PV (synthesis)

Source: https://baohaiphong.vn/tang-cuong-quan-ly-benh-hiem-toan-dien-521076.html