National Medical Products Administration : Multiple ways to promote the development and marketing of drugs for rare diseases

On September 13, China‘s State Information Office held a press conference on the theme of “Promoting High-Quality Development”. At the meeting, the State Drug Administration, deputy director of Huang Guo said that in recent years, China‘s rare disease drugs listed in the number and speed to achieve a “double increase”. 2018 so far, there have been 134 rare disease medicines have been approved for listing, 2024 January-August, has been approved for listing up to 37.

In response to the issue of medication for patients with rare diseases, which has attracted much attention in the past two years, Huang Guo said at the meeting that the State Drug Administration will continue to launch a series of incentives to encourage the research and development of medicines for rare diseases to be listed.

In terms of encouraging independent innovation channel, by encouraging new drug research and development in China, to solve the problem that some rare diseases have no drugs available. The State Drug Administration has set up breakthrough therapeutic drugs, conditional approval, and priority review and approval procedures to accelerate the listing channels for rare disease drug declarations. Technically, the efficiency of R&D and listing of drugs for rare diseases has been enhanced by strengthening communication and guidance during the R&D process, allowing rolling submission of information, reasonably determining clinical endpoint substitution, and shortening the review timeframe.

In terms of speeding up the introduction channel, Huang Guo said that more international drugs for rare diseases that are under research or in production can enter the country more quickly. On the one hand, multinational pharmaceutical companies are supported to synchronize R&D, declaration and listing in China. To this end, a series of initiatives have been taken, such as establishing an implied license system for clinical trials and accepting overseas clinical trial data. On the other hand, to encourage the importation of overseas listed drugs for rare diseases, China is currently soliciting opinions from the public on the Announcement of the State Drug Administration on Matters Relating to Further Optimizing the Review and Approval of Overseas Listed Drugs in Urgent Need for Clinical Purposes, which encourages the declaration of the importation of those drugs that meet the requirements by taking such measures as exempting them from clinical trials, incorporating them into the priority review and approval, as well as shortening the timeframe for inspection, and reducing the batches of inspections and the number of samples. .

In addition, it has also strengthened the synergy and linkage with relevant places and departments to open up more channels and think of more ways for the clinical use of drugs for rare diseases, including: supporting the importation of clinically urgently needed drugs for medical institutions in Boao Lecheng International Medical Tourism Pilot Zone in Hainan and Guangdong-Hong Kong-Macao Greater Bay Area; and supporting the establishment of an advanced zone for the protection of drugs for rare diseases in the Tianzhu Comprehensive Bonded Zone in Beijing.

The State Drug Administration will build and develop these important notices to protect drugs for rare diseases, work closely with health departments, health insurance and other departments, increase policy support, and encourage enterprises to innovate in research and development, so that more rare diseases can have drugs available.