“Is Sanqing interested in making a generic version of clobazam?”
When Wei Kang heard this question, he couldn't help but was stunned. He tapped his fingers on the table and muttered: "Generic drugs can be used, but whether they can be used or not is a very complicated issue. Sanqing has always tended to solve the problem essentially. Achieve therapeutic effects.”
"This is related to the company's strategic decision-making. I can't answer you right away. But I can say that if Sanqing wants to make generic drugs, it will not only be clobazam, but a series of rare disease drugs it represents."
Jian Jian Yun was surprised and happy, and didn't know what to say: "Wei Kang, if this is true, it would be great. I want to 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 there is a heated discussion on the Internet. More than 20,000 family members of the children have united to publish a letter "How to keep our children alive?" "The joint letter, hoping to legally purchase clobazam, instantly went viral on the Internet. Combined with Chen Ping's jumping off building incident, it moved countless netizens.
The joint letter reads: "At the end of "I'm Not the God of Medicine," the protagonist Cheng Yong was released from prison with a reduced sentence. After he came out, he learned the news that the drugs that sent him 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 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-epileptic 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 it has minimal side effects among all similar drugs.
Epilepsy occurs when the excitatory neurotransmitters in the human brain increase or the inhibitory neurotransmitters decrease. Clobazam, a benzodiazepine, can increase inhibitory neurotransmitters in the brain and balance them with excitatory neurotransmitters to prevent epileptic seizures.
Continuous seizures of epilepsy not only cause permanent damage to the patient's brain, but also lead to the risk of death from suffocation. Once the medication is stopped, each attack will harm the child, causing permanent brain damage and causing the child to die prematurely.
In fact, the State Food and Drug Administration has been promoting the research and development of rare disease drugs such as clobazam a few years ago, but domestic pharmaceutical companies have not had much enthusiasm for the research and development of rare disease drugs.
This is not an isolated case. For a long time, the domestic field of rare diseases has been restricted by the development of the entire pharmaceutical industry and has been in a state of stagnation. Statistics show that there are about 16.8 million people with rare diseases in China, and many patients are untreated and have no available drugs.
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), rare diseases are diseases that affect 0.65‰-1‰ of the total population.
Common rare diseases include albinism, multiple sclerosis, acromegaly, idiopathic pulmonary hypertension, phenylketonuria, mitochondrial diseases, etc. Due to the small number of rare disease patients and high research and development costs, pharmaceutical companies are not active in the research and development of rare disease drugs based on economic considerations.
Taking Eagle Country as an example, there were only 38 orphan drug varieties on the market before 1983. In order to encourage pharmaceutical companies and support the development and application of rare disease drugs, the Eagle Country government promulgated the Orphan Drug Act that year, which was also the world's first orphan drug act. .
After pharmaceutical companies obtain orphan drug status, they will receive a series of benefits, including government support for R&D funding, exemption from new drug application fees, drug marketing application fees, fast-track approval, priority in scientific research fund applications, tax incentives and 7-year market exclusivity. At the same time, an application for orphan drug status can be submitted during the animal experiment stage.
After the promulgation of the Orphan Drug Act, more than 30 years ago, the FDA issued a total of 5,219 orphan drug qualifications, and the final number of approved drugs was 843.
In view of the huge promotion effect 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 has also played a huge role in promoting the research and development of rare disease drugs. Currently, 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 difficult-to-treat rare diseases.
In 2019, China also passed rapid approval, priority approval or conditional marketing authorization, and approved the marketing of 8 orphan drugs. At the same time, as the level of the pharmaceutical industry continues to improve, some local companies have begun to get involved in the field of rare diseases and entered the early exploration stage.
However, the research and development of drugs for rare diseases in China is still full of difficulties. Not only are R&D and clinical applications difficult, but the most important reason is that drugs for rare diseases are expensive and have a small audience.
Due to the small number of patients with rare diseases, even if companies aim to fill gaps in clinical needs rather than making a profit, drugs must be expensive in order to recover costs.
For example, Novartis's fingolimod, a drug that treats multiple sclerosis, is priced at 10,500 yuan per box in China. Although this price is not high globally, it is still unaffordable for many families.
In 2019, zolgensma, a gene therapy drug for spinal muscular atrophy, was approved for sale in Eagle Country, priced at US$2.1 million (approximately 14.69 million yuan), making it the most expensive drug in history and beyond the reach of ordinary people.
These expensive original drugs cannot be copied in China due to patent reasons. However, the patents of many rare disease drugs have long expired, but currently no domestic companies have applied for imitations.
There are many reasons behind the reluctance of pharmaceutical companies to imitate rare disease drugs. They are simply that the technology is difficult, the cost is high, and the market is not large. In other words, the returns are very unsatisfactory.
For example, defelaros dispersible tablets, the original drug manufacturer is Novartis. Abroad, this drug is recommended as a first-line drug for patients with thalassemia and iron overload over 6 years old. However, the annual sales of this original drug in domestic sample public hospitals were only 8.74 million yuan.
As for clobazam, there are only tens of thousands of patients in urgent need in the country, and the purchasing price per box is only three to four hundred yuan. It would be strange if a domestic pharmaceutical company would do it without losing money. No wonder pharmaceutical companies have no incentive to copy it.
However, isn’t this the Three Purities?
Sanqing has the technology and capital. The difficulties encountered by other pharmaceutical companies are not a problem for Weikang at all. As long as he wants to do it, he can definitely do it.
Let’s start with generic drugs for rare diseases, which are easy to get started with. The first one is clobazam, which can solve the urgent need of parents of children with the disease.
After the generic drugs are ready, we can then develop original drugs for other rare diseases.
He called Li Zhong, his former subordinate, who had been working conscientiously. It was perfect for him to start a relatively low-tech thing like generic drugs.
"You first collect information, make a list of global rare diseases and treatment drugs, and mark the patents of the original drugs that have expired. I can help."
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. Then we will prioritize all children's drugs and make an expedited list. "
"You should first prepare the materials and apply for the clobazam generic drug project. As soon as possible!"
After watching Li Zhong leave, Wei Kang sent a message to Jian Jianyun about his decision as a reply to her.
The incident was then posted online.
"The recent clobazam incident touched me a lot. While Sanqing is fighting cancer and Alzheimer's disease, there are still many patients with rare diseases who are fighting for their lives."
"This shouldn't be the case. All diseases in the world should be equal. Just because there are few patients with a certain disease and the money required for research and development is high, we should not stop or even give up. Especially since many drugs are not subject to patents, they only need to Generic drugs can save lives.”
"At this time, as a member of the pharmaceutical industry, Sanqing should shoulder its due responsibilities and provide low-cost and high-quality generic drugs for domestic patients with rare diseases."
"Sanqing has launched a plan to launch a rare disease generic drug and will invest resources in the generic version of orphan drugs. The first one will start with clobazam. The project has already begun. We will do our best to strive to launch it as soon as possible so that all patients can benefit from it. You can buy the medicine you need through formal channels.”
“This is Sanqing’s philosophy: making medicines for social needs.”
