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Intractable Rare Dis Res. 2019 Aug;8(3):165-171. doi: 10.5582/irdr.2019.01090.

Marketing of drugs for rare diseases is speeding up in China: Looking at the example of drugs for mucopolysaccharidosis.

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Department of Health Policy Research, Shanghai Health Development Research Center, Shanghai Medical Information Center, Shanghai, China.
Department of Learning, Informatics, Management, and Ethics, Karolinska Institute, Solna, Sweden.
School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, China.


In May 2019, China National Medical Products Administration approved the marketing of an elosulfase alfa injection (brand name: Vimizim) from BioMarin Pharmaceutical for the treatment of patients with mucopolysaccharidosis (MPS) type IVA. This is the first drug to treat MPS in China, and it has ended the "dearth of medicines" to treat MPS in China, a situation that has persisted for many years. One can reasonably say that the drug has benefited from the continuous reform of the drug review and approval system in China and the increasing attention paid to rare diseases. At present, China has implemented a series of preferential policies for the review and approval of drugs for rare diseases, mainly including priority review and approval, accelerated review and approval, special review and approval (mainly simplified review and approval), data protection, and communication. Moreover, China now has a specific reference for the review and approval of drugs for rare diseases with the creation of China's First List of Rare Diseases and the publication of two batches of the List of Overseas New Drugs Urgently Needed in Clinical Settings. Drug review and approval has been significantly accelerated, as has marketing. The two batches of lists of new drugs, issued in November 2018 and May 2019, include 43 drugs for rare diseases (58.1% of all drugs in the lists), 37 of which were included in China's First List of Rare Diseases. The lists also include three other drugs for MPS. As of July 1, 2019, four drugs for rare diseases from the first batch of new drugs have been approved for marketing. In order to further improve the review and approval of drugs for rare diseases in China, a special department should be established for the evaluation of drugs for rare diseases, research on and management of drugs in the post-approval phase should be enhanced, international cooperation in research on use of drugs to treat rare diseases should be enhanced, and the incentive policy for marketing drugs for rare diseases should be improved.


China; Rare disease; drug review and approval; drugs for rare diseases; mucopolysaccharidosis

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