This year is the fifth year of Yan Shu’s diagnosis of chronic myeloid leukemia. “They all say that five years is a barrier. Many patients have fallen in this year. I don’t know if I can pass. Anyway, my mentality is very good. I’m open to it, ha ha.” Yan Shu told the Huaxia times.
Chronic myeloid leukemia is a rare disease. On January 13, Nike issued the first batch of prescriptions simultaneously in many places across the country and officially began to supply drugs in many provinces and cities. This gives new hope to CML patients with drug resistance, including Yan Shu. Previously, on November 25 last year, Nike, a third-generation new drug jointly developed by Yasheng medicine and Xinda biology, was approved for listing to treat any tyrosine kinase inhibitor (TKI) resistance.
The prototype of Lenin’s “life-saving drug” in the film “I’m not a god of medicine” is the first generation of greavel (imatinib). Imatinib is a tyrosine kinase inhibitor, which was approved by the FDA in 2001 for the treatment of chronic myeloid leukemia (CML) and malignant gastrointestinal stromal tumors. Due to T315I gene mutation, some patients will have drug resistance reaction during medication, so they need to try to change their dressing. Therefore, in the years after imatinib came out, generic drugs of Gleevec (including the first and second generation) have been approved for listing. The continuous search for new alternative drugs has become an obstacle to the long-term survival of CML patients.
Wang Wensheng, director of Hematology Department of the first hospital of Peking University, told the Huaxia times that the difference between the first generation, the second generation and the third generation of greavel lies in its different indications. First generation glipizide (imatinib) is a drug targeting the target of BCR-ABL, which can make 80% of patients survive for a long time. The second-generation Glivec (nilotinib dahina, dasatinib sdasai, etc.) is suitable for patients resistant to the first-generation drugs. The third-generation Gleevec is suitable for patients resistant to the first and second-generation drugs and patients with CML with T315I gene mutation.
the market for advanced rare diseases of drugs is a blue ocean
Chronic myeloid leukemia (CML) is a malignant tumor formed by clonal proliferation of bone marrow hematopoietic stem cells, also known as chronic myeloid leukemia. It is the most common type of chronic leukemia, accounting for 15% of adult leukemia. The incidence of CML in China is younger than that in the West. Epidemiological investigation shows that the median age of onset of CML in China is 45-50 years old, and the median age of onset of CML in western countries is 67 years old.
According to official data, by 2021, there are expected to be more than 300 million patients with rare diseases worldwide, and more than 20 million in China. At present, there are more than 7000 kinds of rare diseases, of which 80% are genetic diseases, such as albinism and hemophilia. 95% of rare diseases are still incurable, and the market scale of rare diseases is still increasing year by year. According to frost Sullivan data, the market size of the global rare disease drug market is expected to increase from US $135.1 billion in 2020 to US $383.3 billion in 2030, with a CAGR of 11%. At the same time, it is expected that China’s rare disease drug market will grow rapidly, from US $1.3 billion in 2020 to US $25.9 billion in 2030, equivalent to more than 160 billion yuan, with a CAGR of 34.5%, while the CAGR of the United States and other regions in the world in the same period are 10.5% and 10% respectively.
In China, the development of rare diseases is affected by the pharmaceutical industry and has not been paid attention to. Patients can only rely on imported drugs to prolong their lives. In 2018, with the continuous fermentation of the case of Lu Yong, a patient with CML, the prototype of the story “I am not a god of medicine”, rare diseases finally attracted extensive attention from the society. In May 2018, the National Health Commission jointly issued China’s first batch of rare diseases catalogue, which included 121 diseases, including albinism, to strengthen the national management of rare diseases. That is, since then, more pharmaceutical enterprises have begun to pay attention to the drug R & D and promotion of rare diseases.
In 2019, the State Drug Administration approved the listing of 8 rare disease drugs; On February 18, 2020, Wuxi Apptec Co.Ltd(603259) invested US $30 million in Beihai Kangcheng to build an ecosystem of rare diseases; In July 2020, Langhua pharmaceutical obtained round a financing of USD 80 million; In February 2021, newforce obtained round B financing of RMB 400 million, with a total financing of RMB 500 million; In October and November 2021, the technical guidelines for clinical research and development of drugs for rare diseases and the statistical guidelines for clinical research of drugs for rare diseases (Draft for comments) were successively issued. The principles provide suggestions and references for drug research and development of rare diseases and scientific trial design, which greatly improves the efficiency of clinical research and development related to rare diseases; In December 2021, Hokkaido Kangcheng officially landed in Hong Kong stocks, becoming the “first stock” in the field of disease pharmacy.
The reason why the rare disease industry is called a blue ocean is not only because of the advantages of differentiated competition, but also because rare diseases have solved the clinical needs of patients on the spot, which has attracted a lot of capital. Enterprises also have Beijing Dynamic Power Co.Ltd(600405) sources to continuously develop and develop the market. According to the comprehensive market research and investment analysis report of China’s rare disease drug industry from 2020 to 2024 issued by xinsijie Industrial Research Center, in order to encourage the R & D and listing of rare disease drugs, China has issued a number of policies to support, attract overseas products to enter the Chinese market, and enhance the attention of local pharmaceutical enterprises to the research of rare disease drugs, In the next five years, drugs for different rare diseases may be listed in the Chinese market.
connect with medical insurance to make every patient have a doctor
As a grass-roots civil servant in Changji, Xinjiang, his work is stable and his family is happy. Yan Shu, 30, lives a life that many people envy. However, it is a routine physical examination that breaks all peace. From suspected to diagnosed, just two days have plunged the family into deep pain. After repeated investigation, Yan Shu finally chose the imported second-generation drug dachina (nilotinib capsule) with the least side effects. “I didn’t think about anything at that time. It seems that money is a number. Dashina is a box of 150mg specification, and the full price is 9120 yuan. After reimbursement, it’s about a change of more than 2000. You can eat it for a month, no matter how expensive it is!”
In the sixth month after taking the medicine, Yan Shu’s chromosomes and genes have all turned negative. Later, he needs to continue taking the medicine and control it for a period of time before he can stop taking the medicine. Recalling his treatment process, Yan Shu said that the past can’t be looked back. “But I really thank my attending doctor, Dr. Chen, who helped me apply for the assistance of the China Charity Federation. After buying 8 boxes of dachina, you can help 5 boxes. In this way, the pressure is much less. And now I have applied for chronic diseases, so I can buy drugs directly in the outpatient department. The reimbursement ratio is the same, which is very convenient.”
Many patients are discouraged in the rare disease industry because of the high cost of obtaining drugs and long treatment cycle. The ultimate goal of the rare disease industry is to explore the entry of rare disease drugs into medical insurance and provide patients with medical treatment. The future vision of the ecosystem in the field of rare diseases is to find and treat early, develop more and enter medical insurance, so that every patient can afford drugs.
On January 5, Yasheng pharmaceutical announced that Nike had been officially included in the list of high-value drugs of “Zheli medical insurance · South Taihu health insurance” commercial supplementary medical insurance in Huzhou City, Zhejiang Province. This is the first urban customized commercial medical insurance included in Nike since its approval on November 25 last year, which will accelerate the accessibility of Nike and benefit more patients with chronic myeloid leukemia (CML). According to the product introduction, after being diagnosed by the clinician, if the insured needs to use the drugs in the high-value drug list of “Zheli medical insurance · South Taihu health insurance” such as Nike, and meets the scope of corresponding drug indications in the list, he can pick up the drugs in the designated pharmacy with the external prescription in the designated hospital. The part above 5000 yuan of the starting line will be reimbursed 50%, and the upper limit will be 1 million yuan.
\u3000\u3000 “We are actively promoting the launch of Nike across the country. At present, the first batch of prescriptions have been issued all over the country. We will also continue to make efforts in commercial cooperation. We look forward to working with more partners to make commercial innovation, open the door of multi-dimensional cooperation, and jointly promote the commercialization process after Nike is approved for listing, so that patients in more regions of the country can use it Innovative drugs. ” Zhu Gang, chief business operation officer of Yasheng pharmaceutical, said.
