Many Vietnamese children are given a drug that costs up to 50 billion VND per dose.

(Dan Tri) - There are currently about 6,000 rare diseases in the world, which are diseases that cannot be treated. Participating in clinical trials of new drugs is also an opportunity for children with these diseases.

At the seminar on Building a research and development ecosystem in Vietnam held on June 6 in Hanoi , Associate Professor, Dr. Vu Tri Dung, Director of the Center for Endocrinology - Metabolism - Genetics and Molecular Therapy, National Children's Hospital shared his own experiences when participating in clinical trials.

Rare diseases or as the world uses the term is a disease that cannot be treated. Currently in the world there are about 6,000 rare diseases.

“We are heartbroken to have to answer patients and their families that “there is currently no cure in the world”. Only about 5% of these diseases have cures, other support is care, the result is that patients die or are disabled for life”, Associate Professor Dung shared.

Thanks to participating in clinical trials, many Vietnamese children with rare diseases have access to new treatments (Photo of a drug that costs up to 50 billion VND per dose: National Children's Hospital).

For most rare diseases, the time from when the first case is confirmed to when a cure is available can be up to 90 years, some diseases can take 125 years to have a cure, and many diseases take longer.

Participating in clinical trials of new drugs is also an opportunity for children with rare diseases. To participate in this activity, the first thing hospitals need is a database.

According to Associate Professor Dung, the National Children's Hospital also participates in many clinical trials such as drugs to treat rare spinal muscular atrophy (for children under 2 years old).

In 2019, the FDA approved this drug for children under 2 years old. This is a gene replacement drug and is one of the most expensive drugs with a one-time treatment cost of up to 51 billion VND.

To date, 32 children at the National Children's Hospital and 14 children at the Children's Hospital have received sponsorship for this expensive medicine.

In addition, from 2022, the hospital will also participate in a multi-center clinical trial for 2-year-old children. Vietnam is one of 15 participating countries, the study was completed on May 30. Notably, out of 127 children participating in this trial, 20 are Vietnamese.

Vietnam is facing the opportunity to become the leading clinical trial center in ASEAN, but many challenges such as unsynchronized infrastructure, shortage of specialized human resources...

Strongly and comprehensively promote digital transformation in healthcare

The discussion was part of the Forum "Innovation and digital transformation to promote comprehensive and sustainable healthcare, meeting Vietnam's development requirements in the new era" taking place on June 6 in Hanoi.

Speaking at the forum, Deputy Prime Minister Le Thanh Long said this is an important event in the context of Vietnam promoting digital transformation in all areas, including health care and protection. Health is the most valuable asset of human beings.

Currently, the healthcare system is strongly and deeply affected by the 4th industrial revolution, digital transformation and innovation. This is an inevitable trend and a mandatory requirement for the healthcare sector to develop sustainably, fairly, qualitatively, effectively and internationally.

Science and technology are profoundly changing approaches to medical examination and treatment, disease prevention, scientific research, medical management training and health management.

In recent times, digital transformation in healthcare has made significant progress. 100 medical facilities have deployed hospital information systems, many medical facilities have deployed electronic health records on VneID, remote medical examination and treatment, electronic prescription applications, artificial intelligence to support treatment, and the use of robots in surgery.

Deputy Prime Minister Le Thanh Long (Photo: Tran Minh).

“However, digital transformation in healthcare still faces many difficulties and challenges, the infrastructure of information security and network security systems is not yet synchronized. Digital transformation is not uniform among sectors, and the lack of high-quality healthcare human resources does not meet practical requirements.

In addition, the Covid-19 pandemic has exposed many shortcomings and limitations, especially in preventive medicine, primary health care, drug supply capacity, medical equipment, etc.," the Deputy Prime Minister emphasized.

Therefore, according to the Deputy Prime Minister, the above issues pose an urgent need for innovation in leadership, direction, and strong and comprehensive implementation of people's health care in the new era, and innovation in thinking from medical examination and treatment to focusing on disease prevention and health improvement.

Specifically, strongly and comprehensively promote digital transformation of healthcare, ensuring efficiency and savings in the context of limited resources; effectively deploy electronic health books, electronic medical records, electronic prescriptions; connect data in the fields of healthcare and health insurance.

Units need to strengthen research, development, and application of science and technology, especially artificial intelligence, blockchain, big data, internet of things... in providing medical services.

In addition, the health sector needs to promote scientific research, improve the capacity of research centers, testing, high technology, laboratories and scientific research facilities; strongly promote the domestic pharmaceutical industry, focus on research and production of new drugs, invented drugs, high-tech drugs, herbal medicines, vaccines and biological products.

Vietnam is facing the challenge of an increasing aging population, expected to reach 25% by 2040. The rate of non-communicable diseases accounts for 77%. The burden of disease and disparities in access to health care between regions not only reduce the quality of life but also put a burden on the health system and the state budget.

Therefore, the urgent issue now is that Vietnam needs to create a modern, effective, equitable and sustainable medical - pharmaceutical ecosystem.

The Healthcare Innovation Forum 2025 aims to remove institutional bottlenecks and create breakthroughs in promoting innovative solutions to realize the national vision in the new era.

Source: https://dantri.com.vn/suc-khoe/nhieu-tre-viet-duoc-su-dung-loai-thuoc-co-gia-den-50-ty-dong-mot-lieu-20250606153420092.htm