I can extract side effects.

Chapter 110 Orphan Drug Generic Program

"Is Sanqing interested in making a generic drug of clobazam?"

When Wei Kang heard this question, he couldn't help being taken aback. He tapped his fingers on the table and said in a deep thought: "Generic drugs can be made, but whether to do it or not is a very complicated problem. Sanqing always tends to solve problems in essence. achieve a therapeutic effect."

"This is related to the company's strategic decision. I can't answer you right away. But I can say that if Sanqing is going to make a generic drug, it's not just clobazam, but a series of rare disease drugs it represents."

Jian Lianyun was surprised and delighted, not knowing what to say: "Wei Kang, it would be great if this is true. I thank you on behalf of the patients!"

Wei Kang asked her to wait for the news and hung up the phone.

He searched for news about this matter, and now the discussion on the Internet has been in full swing. More than 2 family members of the children jointly published an article "How to keep our children alive?" "'s joint letter, hoping to legally buy clobazam, instantly went viral on the Internet, and Chen Ping's jumping incident moved countless netizens.

The joint letter wrote: "At the end of "I'm Not the God of Medicine", the protagonist Cheng Yong was released with a commutation of his sentence. After he came out, he learned the news that the drugs that made him go to jail for buying drugs have been included in the medical insurance, and he will never be released again. No one will face the problem of not being able to take medicine. I hope that in the near future, our children can also safely take clobazam from regular channels!"

This is simply another version of "I'm Not the God of Medicine", but it's about another drug - clobazam.

Clobazam is a broad-spectrum anti-seizure drug that has been used to treat rare diseases since the beginning of the century. It is very popular in the treatment of pediatric epilepsy because its side effects are relatively small among all similar drugs.

Epilepsy occurs when excitatory neurotransmitters in the human brain increase, or inhibitory neurotransmitters decrease.And clobazam belongs to benzodiazepine drugs, after taking it, it can increase the inhibitory neurotransmitter in the brain, make it balance with the excitatory neurotransmitter, and avoid seizures.

The continuous seizures of epilepsy not only cause permanent damage to the patient's brain, but also lead to the risk of suffocation and death of the patient.Once the drug is stopped, each attack will cause harm to the child, and it will cause permanent brain damage, causing the child to die prematurely.

In fact, the State Food and Drug Administration was promoting the research and development of rare disease drugs such as clobazam a few years ago, but domestic pharmaceutical companies are not very active in the research and development of rare disease drugs.

This is not an isolated case. For a long time, the field of rare diseases in China has been stagnant due to the development of the entire pharmaceutical industry.Statistics show that the number of rare disease populations in China is about 1680 million, and many patients are untreated and in a state where no medicine is available.

Rare diseases, also known as "orphan diseases", refer to those diseases with extremely low incidence.According to the definition of the World Health Organization (WHO), a rare disease is a disease whose prevalence accounts for 0.65‰-1‰ of the total population.

Common rare diseases include albinism, multiple sclerosis, acromegaly, idiopathic pulmonary hypertension, phenylketonuria, mitochondrial disease, etc.Due to the small number of patients with rare diseases and the high cost of research and development, pharmaceutical companies are not active in the research and development of rare disease drugs based on the consideration of economic benefits.

Taking Eagle Country as an example, there were only 83 kinds of orphan drugs listed before 38. In order to encourage pharmaceutical companies and support the development and application of drugs for rare diseases, the government of Eagle Country promulgated the "Orphan Drug Act" that year, which was also the world's first orphan drug act. .

After obtaining the orphan drug qualification, pharmaceutical companies will receive a series of preferential policies, including government support for R&D funds, exemption of new drug application fees, drug marketing application fees, fast-track approval, priority for scientific research fund applications, tax incentives and 7-year market exclusivity.At the same time, an application for orphan drug qualification can be submitted during the animal experiment stage.

After the promulgation of the Orphan Drug Act, in more than 30 years, the FDA issued a total of 5219 orphan drug qualifications, and finally approved 843 drugs.

In view of the huge impetus of the Orphan Drug Act, countries around the world have successively enacted similar bills to encourage the innovation of orphan drugs.

With the development of science and technology, the emergence of new treatment methods such as gene therapy, siRNA, and immunotherapy have also played a huge role in promoting the research and development of rare disease drugs. At present, there are about 560 rare disease drug projects in progress around the world.In the past five years, the FDA has approved a total of 15 orphan drugs, and there have been new breakthroughs in the treatment of some refractory and rare diseases.

In 19, China also passed fast approval, priority approval or conditional marketing authorization, and approved 8 kinds of orphan drugs for marketing.At the same time, with the continuous improvement of the level of the pharmaceutical industry, some local companies have begun to set foot in the field of rare diseases and entered the early stage of exploration.

However, the research and development of drugs for rare diseases in China is still difficult. Not only is the research and development and clinical application difficult, but the most important reason is that the drug prices for rare diseases are high and the audience is small.

Due to the small number of patients with rare diseases, even if the company aims to fill the gap in clinical needs rather than profit, in order to recover the cost, the price of the drug must be high.

For example, Novartis’ fingolimod, a drug for treating multiple sclerosis, is priced at 10500 yuan per box in China. Although this price is not high globally, it is still unaffordable for many families.

In 19, Zolgensma's gene therapy drug for spinal muscular atrophy was approved for marketing in Eagle Country, and the price was 210 million US dollars (about 1469 million yuan). It is the most expensive drug in history, and it is beyond the reach of ordinary people.

Due to patent reasons, these expensive original drugs cannot be imitated in China, but the patents of many drugs for rare diseases have already expired, but there are no domestic companies to apply for imitation.

There are many reasons behind the reluctance of pharmaceutical companies to imitate rare disease drugs, nothing more than technical difficulties, high costs, and a small market. In other words, the returns are very unsatisfactory.

For example, Deraros Dispersible Tablets, the manufacturer of the original drug is Novartis. In foreign countries, this drug is recommended as the first-line drug for patients with iron overload in thalassemia over 6 years old.However, the annual sales of the original research drug in domestic sample public hospitals is only 874 million yuan.

And like clobazam, there are only tens of thousands of patients in urgent need in China, and the purchase price per box is only 400 yuan. If it is replaced by domestic pharmaceutical companies, it is no wonder that it does not lose money. No wonder pharmaceutical companies have no incentive to imitate.

However, isn't there a sanqing?

Sanqing has the technology and funds, and the difficulties encountered by other pharmaceutical companies are not a problem for Weikang at all. As long as he wants to do it, he will definitely be able to do it.

Let’s start with generic drugs for rare diseases, which are easy to use. The first one is clobazam, which can just solve the urgent needs of parents of children with children.

Wait until the generic drugs are ready, and then develop original drugs for other rare diseases.

He called Li Zhong, the former subordinate, who had been working hard all the time, so it was perfect for him to start a low-tech matter like generic drugs.

"You first collect information, make a global list of rare diseases and therapeutic drugs, and mark the patents of the original drugs that have expired. I am useful."

Li Zhong still looked diligent and honest, adjusted his glasses, and nodded in agreement.

Wei Kang thought for a while, and added a few words: "Sanqing is going to launch a generic plan for rare disease drugs, the first one is clobazam, and then prioritize all children's drugs and make an urgent list. "

"You start to prepare the materials first, and apply for the project approval of clobazam generic drugs. As soon as possible!"

Watching Li Zhong leave, Wei Kang sent a message to Jian Jianyun of his decision, which was regarded as his reply to her.

The matter was then posted online.

"The recent clobazam incident touched me a lot. When Sanqing conquered cancer and Alzheimer's disease, there were still many patients with rare diseases fighting against the disease in order to survive."

"This should not be the case. All diseases in the world should be equal. You should not stop or even give up because there are fewer patients with a certain disease and more money is needed for research and development. Especially many drugs are not troubled by patents. Generics can save lives."

"At this time, Sanqing, as a member of the pharmaceutical industry, should shoulder its due responsibilities and provide domestic patients with rare diseases with cheap and high-quality generic drugs."

"Sanqing has launched a marketing plan for generic drugs for rare diseases, and will invest resources in generic drugs for orphan drugs. The first one starts with clobazam, and the project has already started. We will do our best to get on the market as soon as possible, so that all patients You can buy the medicines you need through regular channels.”

"This is Sanqing's philosophy: pharmaceuticals for social needs."

　Thanks to Mr. Wang Yao for the 3000 tip, I am the police flower for the 100 tip, and the sloppy Z for the 100 tip.

　　
　
(End of this chapter)