For rare diseases that are not rare, how to ensure the availability of drugs?

At present, the main sources of domestic rare disease drugs include imported original research drugs, localization of imported drugs and imitation, etc.

In recent years, for rare disease drugs, various departments have taken a number of measures to increase the flexibility of drug imports, shorten the time for market approval as much as possible under the premise of weighing risks and benefits, and strive to allow rare disease patients to obtain therapeutic drugs
as soon as possible.

In June this year, in order to allow domestic rare disease patients to use a small number of specific clinical urgently needed drugs that have been listed abroad but have not yet been approved in China, the National Health Commission and the State Joint Food and Drug Administration jointly issued the "Temporary Import Work Plan for Clinically Urgent Drugs"
.

In June, the official website of the National Health Commission released the "Temporary Import Work Plan for Chlor Bajan", which clarified that 50 top three hospitals in the country led by Peking Union Medical College Hospital will be able to purchase imported Chlor Bachan
.

The prevalence of epilepsy in China is 4 to 7 thousandths, of which 30 to 40% have repeated seizures after long-term drug treatment, which is called refractory epilepsy
.

On September 22, the former Yanke Chlorbazhan, who was temporarily imported from drugs in urgent clinical need, issued the country's first prescription at Peking Union Medical College Hospital, so that this long-awaited "life-saving drug" for parents of children with rare epilepsy could finally be officially purchased
through the hospital's formal channels.

Domestic chlorbachan is already on the way

In 2017, Chlorbachan was elected as "Chinese mainland Children's Drugs that Have Not Been Registered and Listed in China and Are Urgently Needed Clinically", and was included in the second batch of recommended lists for encouraging research and development to declare children's drugs; In 2018, Renfu Pharmaceutical's application for the research and development of the drug was approved by
the State Food and Drug Administration.

According to the approval and listing information of the CPM new drug research and development monitoring data tablet database of China Pharmaceutical Industry Information Center, on September 14, 2022, the chlorobachan tablets produced by Yichang Renfu Pharmaceutical Co.

The managers of Renfu Pharmaceutical said that the audience of rare disease drugs is small and the research and development difficulty is high, so there are not many
domestic companies that produce such drugs.

Medication for rare diseases has a long way to go

Behind the scenery, some rare disease drugs are quietly withdrawing from the Chinese market
.

In June, Saproteropterin hydrochloride, a drug for hyperphenylalaninemia (HPA), was announced that it would no longer be sold
in China.

This drug has no generic drug approved for listing in China, and the original research pharmaceutical factory is Baimarin Pharmaceutical
of the United States.

The development of rare diseases is expensive for pharmaceutical companies to develop and develop, and due to the limited number of users of drugs, future sales profits can hardly offset the cost of
research and development.

epilogue

Rare diseases are not uncommon, although the number of patients is small, but for the patient's family, once the onset of the disease, rare disease drugs are life-saving drugs
.