Xinhua News Agency, Beijing, December 18 (Reporters Tian Xiaohang and Li Heng) Relevant departments are taking action in order to solve the difficult problem of patients with rare diseases.
The reporter learned from the 2021 China Rare Diseases Conference held in Beijing on the 18th that up to now, more than 60 rare disease drugs have been approved for marketing in China, of which more than 40 have been included in the National Medical Insurance Drug List, involving 25 diseases.
Since its establishment in 2018, the National Medical Security Administration has dynamically adjusted the list of medical insurance drugs once a year, and drugs for rare diseases are also included.
At the same time, through the negotiation of access to rare disease drugs, the price of rare disease drugs has been drastically reduced.
Li Tao, deputy director of the National Medical Security Administration, introduced at the meeting that a total of 7 drugs for rare diseases have been successfully negotiated in 2021, with an average price drop of 65%.
According to estimates by relevant agencies, there are currently about 20 million patients with rare diseases in my country, and more than 200,000 new patients are added every year.
In the face of an increasingly large group of patients with rare diseases, the pace of medication protection must continue to accelerate.
In recent years, my country has implemented priority 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 for the prevention and treatment of rare diseases into the priority review and approval procedures.
According to Chen Shifei, deputy director of the National Medical Products Administration, the National Medical Products Administration and the National Health Commission jointly issued an announcement on matters related to the review and approval of clinically urgently needed overseas new drugs. Three batches of clinically urgently needed overseas new drugs were selected and released, and enterprises were encouraged to apply.
Among the 81 drugs selected and released in three batches, more than half of rare disease treatment drugs.
At present, 26 drugs for rare diseases have been approved for marketing through special channels for clinically urgently needed overseas new drugs.
"Among all drug marketing applications, rare disease drugs have the shortest time limit for review and approval." Chen Shifei said that in 2020, the newly revised drug registration management measures clarified the inclusion of innovative drugs and improved new drugs with obvious clinical value for the prevention and treatment of rare diseases. Priority review and approval procedures, for clinically urgently needed rare disease drugs that have been marketed overseas and have not been marketed in China, the review shall be completed within 70 days.
According to reports, in 2021, the State Food and Drug Administration has newly approved 10 rare disease drugs including risporan oral solution powder for the treatment of rare diseases such as spinal muscular atrophy.
Chen Shifei said that these drugs have filled the gap in the treatment of related rare diseases in China, and brought hope for more rare disease patients to delay disease development and improve the quality of life.