National Medical Products Administration： Accelerate the review and approval of rare disease drugs and fully protect the health rights and interests of patients with rare diseases

Cctv newsOn July 5th, Huang Guo, deputy director of National Medical Products Administration, pointed out at a series of news conferences on the theme of "Opening by Authorities" held by the State Council Press Office that in recent years, National Medical Products Administration has taken the opportunity of deepening the reform of evaluation and approval system to speed up the listing of drugs for rare diseases and make more drugs available to patients with rare diseases. Since 2018, National Medical Products Administration has set up a special channel to queue up independently, encourage declaration and speed up examination and approval for overseas new drugs, including drugs for rare diseases, which are urgently needed in China. According to the above policy, 23 imported new drugs for rare diseases have been approved for listing through special channels. In 2020, we further clarified the priority review procedure, and included new drugs for the prevention and treatment of rare diseases with obvious clinical value into the priority review and approval procedure. At present, among all drug listing applications, the time limit for the review and approval of new drugs with rare diseases is the shortest.

In view of the fact that the incidence rate of rare diseases is extremely low and the difficulty of drug research far exceeds that of common frequently-occurring diseases, National Medical Products Administration has given preferential policies to innovative drugs for treating rare diseases. Drug evaluation institutions have implemented early intervention, research and trial linkage and full-service for new drugs for rare diseases, set up evaluation teams to follow up the research and innovation of rare drugs, allowed enterprises to submit research materials in a rolling way, and established a seamless connection mechanism in key links such as communication, verification and inspection, and comprehensive evaluation. These practices can greatly improve the quality and efficiency of research and development of new drugs for rare diseases.

Through comprehensive measures, in recent years, the number and speed of drugs for rare diseases in China have achieved a "double improvement". Since 2018, China has approved a total of 68 imported and domestically produced drugs for rare diseases. In the next step, National Medical Products Administration will continue to pay close attention to the needs of patients with rare diseases, speed up the review and approval of drugs with rare diseases on the basis of ensuring the safety, effectiveness and controllable quality of drugs on the market, and do its utmost to delay the development of patients with rare diseases and improve their quality of life.