Analysis of the opportunities and challenges faced by the drug market of rare diseases in China

[pharmaceutical network market analysis] rare diseases have the characteristics of difficult diagnosis, treatment and expensive treatment costs Moreover, due to the small number of patients, small market demand and high research and development costs, rare disease treatment drugs are seriously lacking According to the definition of the World Health Organization, more than 7000 rare diseases have been found in the world, but less than 10% of them have treatment plans In this context, countries provide preferential policies for the research and development of orphan drugs to treat rare diseases, and the orphan drug market has made great development According to the report "orphan drug report 2019" released by evaluatepharma, the global orphan drug market has a sales volume of US $131 billion in 2018 and is expected to reach US $242 billion by 2024 There are about 16.8 million patients with rare diseases in China who need treatment Some of them cost 12 million yuan a year, which brings serious burden to the patients In recent years, the country has paid more and more attention to the patients with rare diseases, paid attention to the prevention and control of rare diseases, and reduced the burden of patients In May 2018, five national departments released a batch of rare diseases catalogue, including 121 kinds of diseases, among which 83 kinds of drugs are available in 53 kinds of diseases in the mainland, but 21 kinds of diseases are faced with the situation that there are drugs abroad and there are no drugs in China In 2019, a total of 18 diseases and 29 drugs will be listed in the national medical insurance catalogue For patients with rare diseases who need life-long medication, it will reduce the cost of some drugs On December 29, the second batch of national organizations started the centralized purchase and use of drugs According to the work arrangement of centralized procurement, the bid opening will be held on January 17, 2020 to produce the results of the proposed selection, and patients from all over the country will use the second batch of centralized procurement of selected drugs in April next year The second batch of national drug collection organizations selected 33 varieties, covering diabetes, hypertension, anti-tumor and rare diseases and other treatment fields, involving more than 100 pharmaceutical manufacturers According to the data submitted by each alliance region, the base of this purchase volume is 12.4 billion pieces (bags / piece) It is reported that the purpose of this centralized purchase is to reduce the price of drugs, which will also greatly reduce the burden of patients with rare diseases In February this year, the state also established a diagnosis and treatment network for rare diseases 324 hospitals across the country joined in the diagnosis and treatment network for rare diseases Patients with rare diseases can find a hospital suitable for diagnosis and treatment according to their condition It can be seen that the drug market for rare diseases will continue to grow with the support of many It is worth mentioning that the pressure of orphan drug research and development in China is still huge On the one hand, the market is small, the risk is high, the enterprise is unprofitable, and often lacks enthusiasm for research and development; on the other hand, new drug research and development faces many difficulties, including relatively weak basic research, insufficient transformation ability; little research experience in rare diseases; and difficulties in clinical trials In this regard, the industry suggests that China should establish and improve the incentive policies and measures related to orphan drug research and development At the same time, under the current medical insurance system in China, medical insurance funds are limited, and the economic burden of patients with rare diseases needs to continue to be reduced In addition, under the current industry situation, the detailed rules of policies need to protect the actual interests of orphan drug R & D enterprises In order to solve the problem of patients' access to medication, we can use the strategy of fixed-point production of rare drugs for reference As for orphan drug R & D enterprises, they need to actively participate in the establishment and improvement of China's rare disease policy, and find a path suitable for their own development The industry believes that for traditional pharmaceutical enterprises, it is also a way of upgrading and transformation to set foot in the field of rare diseases and provide clinically valuable products in the future.