Edition of 28 November 2017

Contributed by national rare diseases registry system of China (NRDRS)

Rare diseases are very important in a country like China due to its huge population: the number of people living with a rare disease is high because of the number of rare diseases and the national population being the highest in the world. China has thus been facing the greatest challenge in managing patients with rare diseases. However, notable delays exist in China’s orphan drug market comparing to US, EU, and Japan due to the lack of clear definition of rare diseases or orphan drugs.  
On October 8, 2017, the General Office of the CPC Central Committee and the General Office of the State Council of the People’s Republic of China jointly issued “Opinions on Promulgating the Reform of Review and Approval System for Drugs and Medical Devices to Encourage Innovation (the Opinion)”. The Opinion set up regulatory guidelines for several critical issues in R&D of drugs and medical devices, including the reform of clinical trial management, the acceleration of review and approval process, the augmentation of drug innovation and generics development, the promotion in product lifecycle management, technical support, and organizational implementation.
In particular, the Opinion addresses specific regulations for rare diseases in Section 2, Article 10:
“The National Commission of Health and Family Planning or the authorized societies will publish the catalogue of rare diseases and rare diseases registries will be established. Registration of drugs or medical devices for rare diseases may be exempted from clinical trials conditionally. Drugs and medical devices that have been approved for marketing overseas can be approved in China with conditions.  Risk management plans should be set up to conduct research in consonance with specific regulations.”
In addition to representing a milestone in China’s efforts to reform its drug and medical device approval system, the Opinion grants noteworthy support for rare diseases and orphan drugs. However, the Opinion is not a formal law or regulation. Relevant laws and regulations will need to be amended to fully implement the reform.