National Medical Products Administration： Accelerate the review and approval of drugs for the treatment of rare diseases

　　China news agency, Beijing, September 29 (Reporter Li Yanan) National Medical Products Administration, China, announced on the 29th that as of May 2019, 69 applications for registration of drugs for treating rare diseases had been included in the priority review procedure in order to encourage the research and development of drugs for treating rare diseases. In the next step, we will continue to speed up the review and approval of drugs for the treatment of rare diseases and improve the supply guarantee level of drugs for rare diseases.

　　Data map: The doctor is taking medicine for the patient. China News Service reporter Yang Huafeng photo

　　The World Health Organization defines rare diseases as 0.65&permil of the total population; To 1 & permit; At present, there are about 6000 to 8000 kinds of rare diseases recognized internationally. According to incomplete statistics, there are more than 20 million patients with rare diseases in China.

　　National Medical Products Administration pointed out that in order to encourage the research and development of drugs for the treatment of rare diseases, the Opinions on Solving the Backlog of Drug Registration Applications was revised and implemented in December 2017, giving priority to the review and approval of drug registration applications in 18 situations, including drugs for the treatment of rare diseases. As of May 2019, National Medical Products Administration has included 69 applications for registration of drugs for the treatment of rare diseases in the priority review process, greatly accelerating the process of product development and listing.

　　In order to speed up the listing of overseas new drugs urgently needed for clinical use in China, National Medical Products Administration, together with relevant departments, jointly issued the Announcement on Relevant Matters Concerning the Review and Approval of Overseas New Drugs Needed for Clinical Use, which established a special channel for the review and approval of overseas new drugs urgently needed for clinical use, and concluded the treatment of rare drugs included in the special channel within three months.

　　According to the news from National Medical Products Administration, two lists of overseas new drugs urgently needed in clinic have been released, including 37 drugs for treating rare diseases. Up to now, there are selepag tablets for the treatment of idiopathic pulmonary hypertension, Elosulfatase-&alpha for the treatment of mucopolysaccharide storage disease; , Fengomode Hydrochloride Capsules for the treatment of multiple sclerosis, Eloizumab for the treatment of hypercholesterolemia, Icazumab for the treatment of paroxysmal nocturnal hemoglobinuria, Terfluramide tablets for the treatment of multiple sclerosis, Nosinasone Sodium Injection for the treatment of spinal muscular atrophy and Edaravone Injection for the treatment of amyotrophic lateral sclerosis were approved for import through this special channel. (End)