Since December, the State Drug Administration has successively approved the application for import registration of two rare disease drugs, rituximab for injection and alfaltizumab injection, through the priority review and approval procedure, which is also the first import registration approval of rare disease drugs this year.
Rare disease is a general term for a large class of diseases with extremely low prevalence. It is generally chronic, serious and most of them will lead to death without intervention. At the 2021 China rare diseases conference held in Beijing on December 18, Li Tao, deputy director of the state medical security administration, introduced that since its establishment in 2018, the state medical security administration has dynamically adjusted the list of Medicare drugs once a year for four consecutive years, and a total of 507 new and good drugs have been included in the list. Through the list adjustment, the scope of drug security has been continuously expanded and the structure has been optimized. So far, more than 60 drugs for rare diseases have been approved for listing in China, of which more than 40 have been included in the national medical insurance catalogue, involving 25 diseases.
Through the negotiation on medical insurance access of drugs for rare diseases, the price of drugs for rare diseases has also been greatly reduced. A total of 7 drugs for rare diseases have been successfully negotiated this year, with an average decrease of 65%. In particular, SMA (spinal muscular atrophy) injection has been included in the catalogue at a substantial price reduction, from the sky high price of 700000 yuan per injection to more than 30000 yuan. In addition, it has been included in the expense reimbursement of medical insurance, which has basically solved the drug problem of SMA patients and greatly reduced the economic burden of patients.
In order to encourage pharmaceutical enterprises to develop drugs for the treatment of rare diseases, the guiding principles for clinical research and development of drugs for rare diseases and the guiding principles for statistics of clinical research of drugs for rare diseases (Draft for comments) were successively issued in October and November this year. These policies, combined with the characteristics of rare diseases, provide suggestions and references for drug research and development of rare diseases and scientific trial design, It improves the clinical R & D efficiency related to rare diseases.
Zhang Cuixia, chief investment adviser of Jufeng investment, told the Securities Daily that China has attached great importance to the diagnosis, treatment and R & D of rare diseases in recent years, the relevant policy framework has been improved day by day, and more and more pharmaceutical enterprises have invested in the R & D of new drugs for rare diseases. The market boom in the research and development of new drugs for rare diseases is also increasing, and relevant companies are favored by capital.
Zhang Cuixia said: "in the primary market, Yaoming biology, Ruihua capital and other well-known institutions and funds have become strategic investors in rare disease drug R & D companies, mainly investing in pharmaceutical enterprises with rich R & D pipelines and independent R & D. in the secondary market, kangdini pharmaceutical, Deyi sunshine and Langyu group have actively arranged in the early R & D stage, as well as Yasheng pharmaceutical and Shanghai Junshi Biosciences Co.Ltd(688180) Under the guidance of policies, they have entered the field of drug use for rare diseases. There has always been a shortage of drugs for rare diseases and they rely on imports. With the promotion of policies, this field is expected to become a blue ocean market for pharmaceutical enterprises. "
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 shows that in order to encourage the R & D and listing of rare disease drugs, China has issued a number of policies to support it, 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.
Guosen Securities Co.Ltd(002736) senior researcher Zhang Lichao told the Securities Daily that compared with ordinary drugs, rare disease drugs are not only difficult to develop and costly, but also face problems such as narrow audience and insufficient drug patent protection. Due to the small amount of drugs used in the rare disease market, relevant pharmaceutical enterprises should comprehensively evaluate the R & D cost of new rare disease drugs and the benefits brought to patients, health care system and society, objectively evaluate the whole cycle process of rare disease drugs from R & D to market, and ensure reasonable profits in combination with relevant preferential incentive policies of the state.
"Compared with ordinary drugs, rare disease drugs have the characteristics of higher R & D cost, lower return on investment, longer R & D cycle and higher risk in the R & D process. Therefore, when M & A and expanding the R & D pipeline of rare disease drugs, pharmaceutical enterprises should focus on the R & D cycle, approval time limit, R & D cost, related expenses, corporate profits, social benefits and other indicators, combined with the enterprise The construction of corporate social responsibility should effectively take into account the relationship between corporate profits and social benefits. It is expected that with the government's incentive policy support for the R & D of rare disease drugs and the continuous pursuit of corporate social responsibility, more and more excellent rare disease drug enterprises will stand out and gain enterprise value-added while winning social respect. " Zhang Lichao said.
(Securities Daily)
