During a recent press conference in Beijing, Huang Guo, the Deputy Director of the National Medical Products Administration (NMPA), discussed significant strides made in the availability of medications for rare diseases in China. “We’ve seen a ‘double improvement’ in both the quantity and speed of drug approvals over the past few years,” Huang noted. Since 2018, over 130 drugs for rare diseases have received approval, with 37 of those occurring just between January and August of 2024.

Addressing concerns about access to these critical medications for rare disease patients, Huang pointed out that the NMPA has been actively implementing a range of incentive policies to bolster the research and development of these essential drugs.

Huang also stressed the need to foster domestic innovation to tackle the treatment gaps for certain rare diseases. He elaborated on the establishment of expedited approval pathways, such as breakthrough therapy designations, conditional approvals, and priority reviews specifically for rare disease medications. “These measures enhance the efficiency of drug development and approval,” he explained, by providing guidance throughout the research process, enabling rolling submissions of documents, and establishing reasonable alternative clinical endpoints.

Furthermore, Huang addressed the efforts being made to accelerate the introduction of international rare disease medications into the Chinese market. He highlighted that the administration is supporting multinational pharmaceutical companies to synchronize their research, applications, and marketing strategies in China. This support includes initiatives like implementing a system for implicit approval of clinical trials and recognizing overseas clinical data. “We’re also encouraging the importation of already approved rare disease drugs from abroad,” he added, noting that public consultations are currently underway to optimize the approval process.

When it comes to emergency imports, Huang reiterated the administration’s commitment to addressing urgent clinical needs under special circumstances. In collaboration with the National Health Commission, the NMPA established a temporary import scheme in 2022 for urgently needed medications, which has been effective in meeting critical demands over the past two years.

In conclusion, Huang underscored the importance of developing and maintaining these essential pathways to ensure access to medications for rare diseases. He expressed the administration’s dedication to collaborating with health and insurance sectors to enhance policy support and foster innovative research, ultimately improving access to vital drugs for patients with rare diseases.