China Orphan Drug Update 2017

China currently has virtually no specific policies or legislation regarding orphan drugs despite having 16.8 million patients with rare diseases, according to the Chinese Center for Disease Control and Prevention. Furthermore, many orphan drugs approved in western countries are unavailable in China. Currently, only 37.8% of orphan drugs approved in the US are available in China. Even if the orphan drug is available, it is often unaffordable under the current healthcare system. The only preferential treatment orphan drugs currently receive in China is priority review during registration. Fortunately, relief is on its way as China has begun to pave a path towards a national rare disease policy.

On May 11, 2017, the China Food and Drug Administration (CFDA) published proposed groundbreaking orphan drug policies for public comment. The CFDA proposes that drugs and devices that treat designated rare diseases may apply for a clinical trial waiver. Orphan drugs and devices that have already been approved overseas may be granted a conditional approval without any domestic clinical studies. Follow-up studies as directed by the CFDA must be completed in China after approval.

On May 19, 2017, Chinese officials announced that they are currently compiling a draft list of rare diseases that may be released by the end of the year. Li Dingguo, chairman of the Shanghai Rare Disease Prevention and Treatment Fund, said China’s draft list covers more than 100 diseases. This list will serve as a basis for orphan drug policymaking.