"Generic directory" declared war on rare diseases?

Source: Sina Pharmaceutical News 2017-11-27 recently, CFDA released the list of the first batch of drugs with expired, terminated, invalid and no copy application For the purpose of the notice, the document said that it is to guide the research and development of generic drugs and improve the public access to drugs The evaluation center screened the drugs that had been listed in foreign countries with expiration, termination, invalidity of domestic compound patent and no copy application, preliminarily screened 10 drugs with clear clinical value, such as tavarboro, and listed them in the guidance list Table 1 data source of the first batch of drugs with expired, terminated, invalid and no copy application: the author of the national drug examination center roughly analyzed the above recommended directory and found that in addition to tavarboro, oriwanxing phosphate and ranimivir octanoate mainly for the treatment of infectious diseases, the remaining seven drugs are all rare disease treatment drugs What kind of message does the first directory of guiding opinions of drug Audit Center convey? On the one hand, as mentioned in the document, it is not difficult to guide the production of generic drugs in pharmaceutical enterprises and reduce the repeated declaration On the other hand, it is not difficult to find that the catalogue released this time is mainly concentrated in the field of rare disease drug use, which is a profound manifestation of "accessibility", and also reflects the importance of drug review center on the development of rare disease drug use The incidence rate of incidence of disease is less than 1% Eight of them are genetic diseases Although the incidence rate is very low, the number of rare diseases in China is 16 million 800 thousand, which is nearly 100 times that of developed countries In China, due to the lack of corresponding drugs, diseases that could have been cured or alleviated have been delayed, bringing lingering shadows to many families, such as phenylketonuria, Gaucher's disease, Huntington's chorea and so on The "ice bucket challenge" that swept the world in 2014 is intended to arouse the public's attention to the gradually frozen human (amyotrophic lateral sclerosis) Table two Some rare drugs for rare diseases are also known as orphan drugs Because of the low incidence rate and few audience groups, many pharmaceutical companies are unwilling to invest in human and material resources This phenomenon is especially evident in China Due to the perfect medical insurance system, strong scientific research foundation and policy guidance in developed countries, there are many pharmaceutical companies focusing on the research and development of drugs for rare diseases, so new drugs are constantly coming out In China, due to the weak foundation of pharmaceutical research and development, most of the drugs for rare diseases rely on import, so the price is high, especially for some biological agents Because most of the imported drugs for rare diseases are not included in the medical insurance reimbursement catalogue, the treatment of rare diseases is a heavy burden for domestic patients In response to this problem, the government is also actively promoting the formulation of policies related to rare diseases In 2012, Qingdao was the first one to include drugs for rare diseases in the medical insurance catalogue; in 2015, drugs for rare diseases received the priority approval of CFDA; in 2016, the state officially released the rare diseases catalogue Although the government pays more and more attention to rare diseases, the fundamental solution lies in improving the "accessibility" of drug use of the public, guiding domestic pharmaceutical enterprises to participate in the research and development of rare disease drugs and imitation Only in this way can we completely solve this problem Therefore, seven drugs for rare diseases are selected in the list of drugs with expired, terminated, invalid and no copy application in the first batch of patents The purpose is to encourage many domestic pharmaceutical enterprises to carry out the copy work of drugs for rare diseases and alleviate the shortage of drugs for rare diseases in China In view of the large number of patients with rare diseases in China, the development of such drugs has broad prospects In the future, we hope that more and more pharmaceutical companies will participate in the development and imitation of drugs for rare diseases in China, bringing new light to families living in the shadow!