Addressing concerns about rare drugs.

SGGP 26/05/2023 06:32

Six vials of Botulism Antitoxin Heptavalent have just arrived in Ho Chi Minh City as emergency aid from the World Health Organization (WHO), but many patients couldn't wait any longer! The anguish of doctors and patients' relatives raises a question for us: why isn't the antidote readily available?

Botulinum poisoning is caused by the anaerobic bacterium Clostridium botulinum. This bacterium can only survive and thrive in an environment completely devoid of air. It produces a highly toxic protein, arguably the most potent of its kind. This toxin is absorbed through the intestines of infected individuals, enters the bloodstream, and attacks nerve cells by binding to them, paralyzing motor nerves. This leads to muscle weakness and paralysis, respiratory arrest, and death.

The botulinum bacteria that cause disease in humans are currently classified into 7 types (subtypes A, B, C, D, E, F, G), and therefore the toxin also has 7 different types. The bacteria that cause disease in children are usually types A and E, while in adults it can be caused by all 7 types. Before an antidote was available, the mortality rate in the US was recorded as 60%; now that an antidote is available, the mortality rate has decreased to below 7%.

The first botulinum antidote was created in 1970 at the U.S. Army Medical Research Institute. It wasn't until 2013 that a new, mixed botulinum antidote containing seven different compounds was approved for mass production. The manufacturing principle for botulinum antidotes is similar to that of snake venom antidotes. But why are they so rare?

The problem is that the number of people infected with botulinum toxin worldwide is not large. According to statistics in the US, in the last century, there were fewer than 200 botulinum infections per year, and now it only fluctuates in the tens or thousands. Therefore, producing the antidote in large quantities is unnecessary; large-scale production is only necessary when there is a specific order.

In developed countries, each country's emergency medical services maintain a certain reserve at most major airports. When needed, they can load the antidote onto planes and transport it immediately to the affected area. The best time for treatment is within two days of symptom onset. The antidote only binds to free toxins in the blood and breaks them down; if the toxins have already bound to nerve cells, it cannot be neutralized, and the body will have to regenerate new cells.

The risk of poisoning is ever-present and can occur at any time. The health sector needs to stockpile a certain amount of this antidote. Botulism Antitoxin Heptavalent can be stored at -20 ° C for 4 years, so purchasing it to prepare for the whole country is necessary and within the health sector's capabilities. Not only botulinum, the Ministry of Health has recently been tasked by the Government to research and develop mechanisms to ensure the availability of rare and limited-supply drugs.

Therefore, a specific financial mechanism is urgently needed to enable healthcare facilities to proactively stockpile rare medications; a mechanism should also be in place for healthcare facilities to purchase and stockpile certain anti-poison drugs and accept their disposal if they expire due to lack of patients. Crucially, the ministry must have policies to encourage domestic pharmaceutical companies to produce rare medications to ensure a reliable domestic supply. Let's not allow the sad story of "drug shortages" to continue!

Source