OBJECTIVES

: The availability of orphan drugs (ODs) in China has long been limited, due to slower development of OD and substantial delay in approval compared with other countries. Since the “Directory of rare diseases (First round)” was released in May 2018, a range of encouraging regulation initiatives were sequentially established in China. This study aims to describe the policy incentives provided to ODs in China.

METHODS

: Governmental websites including the National Health Commission (NHC), National Medical Products Administration (NMPA) and Centre for Drug Evaluation (CDE) were browsed to collect the most up-to-date information related to ODs policies published after 2018.

RESULTS

: Despite the lack of clear definition of rare diseases, a total of 121 rare diseases were designated in February 2018. In November 2018, a special pathway for overseas new urgently needed ODs was established. It stipulated that CDE would complete the technique review within 3 months (6 months for non-ODs) from the reception of dossiers, and NMPA must deliver the decisions within 10 days (instead of 20 days for drugs not urgently needed) after receiving the review outcomes from CDE. Additionally, the value added tax for 21 ODs and 4 active pharmaceutical ingredients was reduced from 16% to 3% upon import since February 2019. Furthermore, the concept of “breakthrough therapy” was introduced in November 2019, aiming at enhancing the interaction between NMPA and developers, and facilitating approval of innovative therapies with potential benefits based on the preliminary clinical evidence supporting clinical benefits.

CONCLUSIONS

: China is devoting substantial efforts to foster the development and approval of ODs. Such initiatives promote the faster availability of ODs in China that will allow China to catch up with other developed countries.