Not long ago, at Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine, 14-year-old Gaucher disease patient Xiao Ai began treatment with a new domestically developed drug. "Having access to an affordable medicine brings much more peace of mind," said Xiao Ai's mother, who felt a sense of relief she hadn't experienced in a long time.

For rare disease medications, which have long been reliant on imports and carried high price tags, more "Made in China" options are now emerging.

"True pharmaceutical innovation must ultimately demonstrate its value in patient accessibility and the dignity of life," said Xue Qun, Founder, Chairman, and CEO of the drug R&D company CanoCan. He added that the future will involve a continued focus on unmet clinical needs, ensuring that more new and effective medicines benefit patients.

Rare diseases serve as a measure of a society's compassion. The journey of this new drug—from laboratory R&D to regulatory approval, and now to inclusion in this year's national commercial health insurance negotiations—epitomizes the accelerated development of domestic innovative medicines in recent years.

The proposals for the "15th Five-Year Plan" include "support for the development of innovative drugs and medical devices." Industry insiders indicate that this signals further intensified policy support from multiple departments. Efforts will span dimensions such as regulatory review and approval, payment security, and industrial support, all aimed at "paving the way and building bridges" for local innovative pharmaceutical companies.

Making Rare Disease Drugs Less 'Rare'

To address the long-standing challenges faced by rare disease patients—"difficulty in diagnosis, difficulty in accessing treatment, and high drug prices"—relevant authorities have established green channels for drug R&D and regulatory review. Additionally, through national医保目录 negotiations and dynamic adjustment mechanisms, multiple rare disease drugs have been accelerated to market and made more affordable.

"The level of support from the national level for innovative drugs, especially clinically urgent rare disease medicines, is unprecedented," said Li Linkang, Executive Chairman of the China Rare Diseases Alliance.

Propelling Innovative Drugs into the "Fast Lane"

In 2024, the average review time for marketing applications of innovative drugs in China was 225 working days. Among these, innovative drugs granted priority review averaged only 162 working days, reflecting a significant acceleration in regulatory review and approval speeds.

Data shows that since the start of the "14th Five-Year Plan," over 110 domestically developed innovative drugs have been approved for marketing in China, with the market scale reaching RMB 100 billion. The proportion of new drugs under research in China now accounts for over 20% of the global total, elevating the country to the second position worldwide in new drug R&D.

A representative from the China Pharmaceutical Innovation and Research Association noted that in recent years, "Chinese drugs" have gradually shifted from imitation and follow-on to original innovation, with a marked improvement in R&D capabilities and internationalization levels.

Multiple government departments have introduced measures to support the development of innovative drugs, employing novel technology platforms to enhance R&D efficiency, and utilizing real-world study data to provide more robust evidence for drug evaluation and reimbursement decisions… In the future, innovative drugs will receive further support throughout their "full lifecycle."

With the deepening integration of policies, industry, and technology, more domestically developed innovative drugs are expected to reach millions of households. The vision of "accessible medical care and affordable medicines" is poised to become a reality at an accelerating pace.

Reference： https://finance.eastmoney.com/a/202512033580725064.html