(Yicai) March 9 -- The president of Peking Union Medical College Hospital has urged lawmakers to speed up the drafting and adoption of legislation on the prevention and treatment of rare diseases so as to tighten inter-agency policy coordination, further motivate drugmakers, and build a comprehensive prevention and treatment system.

“A Rare Disease Prevention and Treatment Law will help establish an incentive policy system that covers the entire process of research and development, evaluation, production, and usage," Zhang Shuyang, who is also chairwoman of the National Health Commission's Rare Disease Diagnosis and Treatment Committee and a deputy to the National People's Congress, said in a joint proposal at the annual meeting of China's parliament, which opened on March 5.

The treatment of rare diseases and the development of related drugs are not only critical livelihood issues but also a strategic driver of innovation in the biopharmaceutical sector and the cultivation of new quality productive forces, Zhang told Yicai.

Rare diseases is a collective term for illnesses with extremely low incidence rates -- about 10,867 rare diseases have been identified worldwide -- with patients often facing either a lack of available treatments or a situation where medicines exist overseas but are not available in China.

For drugmakers, high research and development costs and small patient populations often dampen incentives to develop new therapies or introduce drugs from abroad, especially in the absence of clear policy support and efficient regulatory approval channels.

Due to its large population, China has about 20 million rare disease patients, with more than 200,000 new patients added each year, Zhang pointed out.

Since 2018, the NHC and other government departments have issued two batches of rare disease lists, and the number of related drugs approved through priority review for market entry has increased annually, improving access to treatment. Still, many professionals at innovative drugmakers, as well as patients and clinical experts, believe systematic solutions and institutional safeguards are lacking for drugs targeting diseases not included in the official lists, and even for the production of some that are already covered by medical insurance.

Beyond encouraging rare disease drugs R&D, the new law would also strengthen comprehensive protection for patients. It could help align China’s rare disease prevention and treatment policies with international standards and support the sharing of China’s experience in this field globally.

The proposal also suggests that a platform created by legislation should actively participate in global rare disease governance. Drawing on the experience of developed regions such as the United States and Europe, it further recommends setting up an authority responsible for orphan drug certification and management, thereby building a complete legal and regulatory framework for such medicines.

The NPC is one of China's two most important annual political gatherings and runs through March 12, while the Chinese People's Political Consultative Conference, the top political advisory body, opened on March 4 and ends its session on March 11.

Editors: Tang Shihua, Martin Kadiev