Sino-Singapore Health丨National Food and Drug Administration- Promote the research and development of rare disease drugs through multiple channels

On September 13, the State Council Information Office held a press conference centered on “Promoting High-Quality Development.” During this event, we had the opportunity to speak with Huang Guo, the Deputy Director of the National Medical Products Administration (NMPA), who shared important updates regarding the approval process for rare disease medications in China.

Huang noted that there has been a remarkable increase in both the quantity and speed of drug approvals for rare diseases in recent years. Since 2018, a total of 134 rare disease drugs have gained market approval, with 37 of those approved just between January and August 2024.

When asked about the challenges regarding medication accessibility for patients with rare diseases—a topic that has garnered significant public interest—Huang stressed the need for a nationwide commitment to addressing these concerns. He elaborated on the NMPA’s initiatives aimed at fostering innovation and facilitating the research and development of rare disease medications, highlighting three primary pathways:

Firstly, the self-innovation pathway encourages domestic drug development for rare diseases that currently lack effective treatments. To streamline the approval process, the NMPA has set up accelerated approval channels for breakthrough therapies, allowing for conditional approvals and priority reviews specifically for rare disease submissions. This includes enhancing communication throughout the research process, permitting rolling submissions, determining alternative clinical endpoints, and reducing review timelines.

Secondly, the accelerated import pathway aims to expedite access to international medications that are in research or production phases. The NMPA is actively supporting multinational pharmaceutical companies in conducting research, filing applications, and launching products in China at the same time. New measures include an implied consent system for clinical trials and the acceptance of overseas study data. Additionally, the NMPA is inviting public feedback to improve the review and approval processes for rare disease drugs already available in other markets, potentially exempting them from clinical trials or offering priority reviews.

Thirdly, the temporary import pathway has been instituted to meet urgent clinical needs under special circumstances. Since 2022, in collaboration with the National Health Commission, the NMPA has introduced a work plan for the temporary importation of critically needed medications. This effort has successfully secured essential drugs such as chlorambucil and saquinavir for patients suffering from rare diseases.

Huang also pointed out the enhanced collaboration with local governments and relevant departments to create more avenues for clinical use of rare disease medications. Initiatives include supporting medical institutions in the Hainan Boao Lecheng International Medical Tourism Pilot Zone and the Guangdong-Hong Kong-Macau Greater Bay Area to import urgently needed drugs, as well as establishing a rare disease drug guarantee pilot zone in the Beijing Tianzhu Comprehensive Bonded Zone.

In summary, Huang concluded that through these comprehensive strategies, China has significantly improved the number and speed of rare disease drug approvals in recent years, with a total of 134 drugs approved since 2018 and 37 in just the first eight months of 2024.