Five Chinese government agencies, including the State Drug Administration and the National Health Commission, published a national list of 121 qualified rare diseases to support diagnosis and treatment of such conditions in a move that could spur R&D and regulatory submissions for rare disease therapies.
This is the first time China has recognized rare diseases on a national level, James Xue told BioCentury. Xue is founder, chairman and CEO of Canbridge Life Sciences Ltd. (Beijing, China) and previously served as first China general manager of rare diseases pioneer Genzyme Corp., which is now part of Sanofi (Euronext:SAN; NYSE:SNY).
He said a publicly available list will help promote R&D, improve review and approval times, and could make it easier for companies to gain reimbursement for their drugs in China.
While China has historically shown a lack of focus on rare diseases, the list is one signal that the area is increasingly becoming a priority for the country.
However, China still has a way to go towards encouraging development of rare disease treatments. For instance, Xue said there is no definition or criteria for what constitutes a rare disease in the country and no national reimbursement policy for Orphan drugs.
Xue said current policies are geared more towards rare disease drugs already approved in western markets. There is "no policy on newly developed drugs in China that encourages companies to take risks and includes exclusivities like pricing reimbursement, or IP,” he said.
Of the 121 diseases on the list, which was released on May 22, 110 already have a drug approved by U.S. FDA, Xue said. The list may encourage sponsors of those drugs to pursue approval in China. It also could facilitate a shorter review timeline and allow them to reference the list to signal an unmet medical need in China while seeking reimbursement, according to Xue.
Additionally, Xue said any company that wants to submit an IND to study a rare disease treatment in China is likely to have receptive discussions with regulators, if the targeted disease is on the list.
Furthermore, he believes the list will be a useful guidance for initiating price reimbursement discussions. He expects guidance will be released to set terms for coverage for rare disease therapies "relative to what other countries price drugs."
Over the past year, the State Drug Administration (formerly China FDA) has published a series of regulations intended to bring many of the agency's policies closer in line with western standards.
Draft regulations released in May 2017 set 10 years of exclusivity for innovative drugs for rare or pediatric diseases. In December, SDA issued draft guidelines on a conditional approval pathway for life-threatening rare diseases and guidance to implement a priority review pathway that includes rare diseases. Guidance to encourage development of generics also includes drugs for rare diseases (see BioCentury Extra, Dec. 29, 2017).
Joining SDA and the National Health Commission in compiling the list were the Ministry of Science and Technology (MOST), the Ministry of Industry and Information Technology (MIIT) and the State Administration of Traditional Chinese Medicine of China.