Behind the policy support, what trapped original drugs

 Recently, two posters of the American Society of Clinical Oncology and a lecture report of the American Society of Neuro-Oncology have given Nankai University Professor Chen Yue a mixed blessing.

Fortunately, Chen Yue has devoted 11 years of painstaking efforts to the original drug ACT001, which has conducted 10 clinical trials around the world. The results are gratifying. Among them, the clinical trial for relapsed glioblastoma was treated with ACT001 monotherapy. One patient obtained long-term Complete remission, and single-drug therapy for children with diffuse endogenous pontine glioma has also observed very good curative effects, which indicates that ACT001 is one step closer to the successful launch of the drug.

The worry is that the upcoming national and global multi-center clinical trials will not only require more subsidiaries and businesses in developed countries around the world, but also require a lot of financial support. The company needs to go public as soon as possible to expand its international influence. , And also provide sufficient funding guarantee for global clinical trials.

The proportion of original drugs in the true sense in my country is still very low, most of which are generic drugs

In the eyes of many people, Chen Yue is a "great god"-level figure. His blockbuster originated from the fact that in 2017, the ACT001 anti-glioma drug developed by his team was recognized by the U.S. Food and Drug Administration (FDA) as a rare disease drug and became one of the few in my country. One of several medicines that have obtained this qualification.

Due to the small number of patients with rare diseases, low market demand, and high research and development costs, few pharmaceutical companies have paid attention to the development of this therapeutic drug before, so these drugs are vividly called "orphan drugs."

"There is currently no specific medicine for glioma. The incidence of this malignant tumor is less than 8 per 100,000, but the 5-year survival rate is only 5%. The malignancy is very high, especially for recurrent glioblastoma, with a median survival rate. The period is only 4-6 months." Professor Li Wenbin, director of the Neurotumor Comprehensive Ward of Beijing Tiantan Hospital, Capital Medical University, lamented, because it is difficult to break through the blood-brain barrier and the immune microenvironment of the brain is not the same as the tumor microenvironment of the body. , Brain tumors have become a "forbidden zone" for targeted drugs. At present, temozolomide, the best drug for the treatment of glioma, can only prolong the survival period of patients by about two to three months.

Chen Yue broke into such a "forbidden zone" and also made original drugs.

During his Ph.D., Chen Yue did research on the total synthesis of anti-cancer natural products and medicinal chemistry. After graduating in 2003, he joined a company in the United States that specializes in the development of new drugs using natural drugs as templates. His research team has successively developed a number of new drugs. One of the projects code-named KOS2187 was approved by the FDA to enter global clinical trials, and was eventually acquired by Pfizer for US$250 million.

Having been in the industry for a long time, Chen Yue is well versed in what it means for a country to develop original drugs.

"For patients, original drugs solve the needs of patients who do not yet have therapeutic drugs, real new drugs with great clinical value, and patients in the country are definitely the first beneficiaries." Chen Yue said, on the other hand, there is a good Medicine, patients still have to afford it. Otherwise, like Gleevec in the movie "I am not the god of medicine", this "magic medicine" for the treatment of chronic myelogenous leukemia was first introduced to China in 2001. The price was 23,500 yuan a box, and a box was one month. Taking the dose will cost the patient 280,000 yuan a year.

As Brad Longka, the founder of the biomedical investment fund Loncar Investments, said, original medicine is the area where the biomedical industry brings the greatest value to patients.

Making one's own original drugs also means that in the future, my country will have the right to set prices, and that good original drugs can be exported to the world and can earn foreign exchange for the country. For example, Xiu Mei Le, known as the "Global Medicine King", has a yearly sales volume of 20 billion U.S. dollars. Conversely, importing a large number of original foreign medicines will greatly increase the burden of my country's medical insurance, and there is also the risk of being "stuck" by foreign countries at any time.

Chen Yue's industry experience also coincides with the needs of the country. Since the 18th National Congress of the Communist Party of China, General Secretary Xi Jinping has been concerned about the health of the masses of the people and the safety of medication. He emphasized: We must always put the health of the masses of people in the first place. He pointed out: "It is necessary to closely monitor the shortage of medicines and take effective measures to solve the supply problems of low-priced medicines,'life-saving medicines','orphan medicines' and medicines for children."

In Chen Yue's mind, it can be called (FIC, original drug) if it has obvious novelty in the three aspects of mechanism, use, and structure, or at least the mechanism of action is different.

In addition to original drugs, there are drugs with expired foreign patents, which are generic drugs. In my country, many so-called new drugs should basically be "micro-innovations" with minor revisions. They have the same mechanism targets, and their chemical structures break through the patents of original drugs. At present, the proportion of genuine original drugs in China is still very low, most of which are generic drugs.

The reporter inquired about the data of Yaodu and learned that as of April 2021, excluding compound drugs, there are a total of 1,001 class 1 chemical drugs in China, including 990 NME (New Molecular Entities) and only 4 FIC (First-in-class) chemical drugs. There are 700 category 1 biological drugs in China, including 662 NME and only 2 FIC biological drugs (0.29%).