A meeting on rare disease drug R&D, under the theme "Care Comes First," was held on February 28 at the Center for Drug Evaluation of the National Medical Products Administration (NMPA). Huang Guo, Deputy Commissioner of the NMPA, attended the event and delivered a speech.

It was noted at the meeting that in recent years, the NMPA has continuously strengthened supports for rare disease medications. By implementing a combination of policies, the NMPA has established and streamlined three pathways of encouraging innovation, accelerating domestic approval, and facilitating temporary imports. As a result, both the number and speed of rare disease drug approvals in China have significantly increased. In 2025, 48 rare disease drugs were approved for market, enabling more patients to simultaneously access international advanced treatments and medicines.

It was emphasized that addressing challenges in the rare disease field requires greater institutional innovation and resource integration. The NMPA will continue to strengthen policy support and improve review and approval procedure. At the same time, it will foster multi-party collaboration, build consensus and synergy, and promote the application of new technologies and products.

Representatives from relevant government departments, related NMPA departments and affiliated institutions, as well as representatives from academia, industry, and patient communities in the rare disease field, attended the meeting.

(From NMPA website-2026-02-28)