Removing concerns about rare drugs

Báo Sài Gòn Giải phóngBáo Sài Gòn Giải phóng26/05/2023


SGGP

6 bottles of Botulism Antitoxin Heptavalent have just arrived in Ho Chi Minh City under the emergency aid of the World Health Organization (WHO), but many patients could not wait! The anxiety of doctors and relatives of patients raises the question: why is the antidote not available?

Botulism is caused by the anaerobic bacterium Clostridium botulinum. This is a type of bacteria that can only survive and grow in an environment that is completely devoid of air. This bacterium produces a toxic protein, which can be considered the most toxic. This toxin is absorbed through the intestines of the infected person, enters the blood and attacks by binding to nerve cells, paralyzing motor nerves, leading to muscle weakness and paralysis, respiratory arrest and death.

The strains of botulinum bacteria that cause disease in humans are currently divided into 7 types (subtypes A, B, C, D, E, F, G), so there are also 7 different types of toxins. The strains that cause disease in children are usually caused by 2 types A and E, while in adults it can be caused by all 7 types above. When there was no antidote, the recorded mortality rate in the US was 60%, now that there is an antidote, the mortality rate has dropped to below 7%.

Botulinum antitoxin was first created in 1970 at the US Military Medical Research Institute. It was not until 2013 that a mixture of seven types of botulinum antitoxin was licensed for mass production. The production principle of botulinum antitoxin is similar to that of snake venom antitoxin. But why is it rare?

The problem is that the number of people infected with botulinum in the world is not much. According to statistics in the US, in the last century, there were less than 200 people/year infected with botulinum and now it only fluctuates in the units or dozens. Therefore, producing antidotes in large quantities is not necessary, only large-scale production when there is an order.

In developed countries, each country's emergency medical service has a certain amount of stock at most major airports. When needed, they can be put on a plane and transported immediately to the place of need. The best treatment is within 2 days of detecting symptoms. The antidote can only combine with the toxins that are still free in the blood and decompose them, but if the toxins have attached to the cells of the nervous system, they cannot be detoxified, only waiting for the body to regenerate other cells.

The risk of poisoning always exists and can happen at any time. The health sector needs to purchase a certain amount of this antidote. Botulism Antitoxin Heptavalent can be stored at -20 ° C for 4 years, so purchasing the drug for the whole country is necessary and within the capacity of the health sector. Not only botulinum, the Ministry of Health has recently been assigned by the Government to research and develop a mechanism to ensure rare drugs and drugs with limited supply.

Therefore, it is necessary to soon have a specific financial mechanism for medical facilities to proactively reserve rare drugs; have a mechanism for medical facilities to purchase and reserve some antidotes and accept to destroy them if there are no patients, leading to the drugs expiring. Importantly, the Ministry must have a policy to encourage domestic pharmaceutical enterprises to deploy the production of rare drugs to proactively supply domestic supplies. Do not let the sad story called "Drug shortage" continue!



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