Cracking the medicine is difficult! The national medical insurance drug list has included more than 40 drugs for rare diseases

Beijing, 18 Dec (Xinhua) -- In order to solve the problem of medication for patients with rare diseases, relevant departments are taking action. The reporter learned from the 2021 China Rare Disease Conference held in Beijing on the 18th that as of now, more than 60 rare disease drugs in China have been approved for listing, of which more than 40 have been included in the national medical insurance drug directory, involving 25 diseases.

Since its establishment in 2018, the National Medical Security Bureau has dynamically adjusted the list of medical insurance drugs once a year, and drugs for rare diseases are also transferred. At the same time, through the negotiation and access of rare disease drugs, the price of rare disease drugs has been greatly reduced. Li Tao, deputy director of the National Medical Security Bureau, introduced at the meeting that in 2021, a total of 7 rare disease drugs were successfully negotiated, and the average price reduction reached 65%.

According to the estimates of relevant institutions, there are about 20 million patients with various rare diseases in China, and more than 200,000 new patients are added every year. In the face of an increasingly large group of rare disease patients, the pace of medication protection must continue to accelerate.

In recent years, China has given priority to the review and approval of drugs for the treatment of rare diseases. The State Drug Administration will include innovative drugs and improved new drugs with obvious clinical value in the prevention and treatment of rare diseases into the priority review and approval procedure.

Chen Shifei, deputy director of the State Drug Administration, introduced that the State Food and Drug Administration, together with the National Health Commission, jointly issued an announcement on matters related to the review and approval of new drugs that are urgently needed for clinical use, and selected and released three batches of lists of new drugs that are urgently needed abroad for clinical use, encouraging enterprises to declare. Of the 81 drugs selected and released in the three batches, more than half of the drugs for the treatment of rare diseases. At present, 26 rare disease drugs have been approved for listing through the special channel of urgently needed overseas new drugs.

"Among all drug listing applications, the time limit for review and approval of rare disease drugs is the shortest." Chen Shifei said that in 2020, the newly revised measures for the administration of drug registration clearly stated that innovative drugs and improved new drugs for the prevention and treatment of rare diseases with obvious clinical value will be included in the priority review and approval procedures, and the rare disease drugs that have been listed overseas and are not listed in China will be reviewed within 70 days.

According to reports, in 2021, the State Food and Drug Administration newly approved 10 rare disease drugs such as risprolane oral solution for the treatment of rare diseases such as spinal muscular atrophy. Chen Shifei said that these drugs fill the gap in the treatment of related rare diseases in China, and bring hope for more rare disease patients to delay the development of their diseases and improve their quality of life. (End)