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Background <p>Over the past approximately 40 years, Chinese drug regulations have undergone many major reforms to accelerate the approval of drugs and keep pace with the scientific innovation of drugs in the world, especially developed countries. In 2018 and 2023 China’s National Health Commission in collaboration with other departments released China’s “First Batch of Rare Diseases Catalogue” and “Second Batch of Rare Disease Catalogue”. However, there is currently less relevant research on the overview and speed of the approval of rare disease drugs in China.</p> Methods <p>This study used cross-sectional analysis of rare disease drugs approved in China and the USA from 1983 to 2022 through official drug search databases and systematically analyzed and compared rare disease drugs approved in the USA and China, including the number, approval time, related laws and regulations, and expedited programs.</p> Results <p>Between 1983 and 2022, the USA Food and Drug Administration (FDA) approved a total of 693 orphan drugs (covering 1228 formulations/specifications). Among these, 201 drugs (425 formulations/specifications) were approved by China’s National Medical Products Administration (NMPA), accounting for 29.0% (201/693) of the approved drugs and 34.6%(425/1228) of the approved formulations/specifications. The number of China’s rare diseases drugs is increasing year by year, and the approval speed has gradually accelerated. The median approval lag time was 32.3 years (IQR: 22.9–33.6) from 1983 to 1987. In contrast, the shortest median lag time occurred between 2018 and 2022, at approximately 1.4 years (IQR: 0.5–2.6)—a reduction of 94.2% that reflects the success of China’s ongoing drug regulatory reforms. The special procedures for drug approval have a great promoting effect on rare diseases drugs’ approval.</p> Conclusion <p>This research provides evidence of breakthroughs in the review and approval of rare disease drugs in China and demonstrates the tremendous boost to rare disease drugs from China’s ongoing restructuring and reform of the drug regulatory ecosystem, as well as a stimulus for future rare disease drugs development in China.</p>

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Trends, lag and characteristics of rare disease drug approval in the USA and China, 1983–2022

  • 期刊论文

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  • 发表日期：

    2025年12月22日

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  • Shaohong Wang,
  • Xin Liu,
  • Yuzhen Zou,
  • Yan Tang,
  • Wei Zuo,
  • Rong Jiang,
  • Junmei Shang,
  • Xin Tian,
  • Qingyang Liu,
  • Tingting Xu,
  • Bo Zhang,
  • Shuyang Zhang

摘要

Background

Over the past approximately 40 years, Chinese drug regulations have undergone many major reforms to accelerate the approval of drugs and keep pace with the scientific innovation of drugs in the world, especially developed countries. In 2018 and 2023 China’s National Health Commission in collaboration with other departments released China’s “First Batch of Rare Diseases Catalogue” and “Second Batch of Rare Disease Catalogue”. However, there is currently less relevant research on the overview and speed of the approval of rare disease drugs in China.

Methods

This study used cross-sectional analysis of rare disease drugs approved in China and the USA from 1983 to 2022 through official drug search databases and systematically analyzed and compared rare disease drugs approved in the USA and China, including the number, approval time, related laws and regulations, and expedited programs.

Results

Between 1983 and 2022, the USA Food and Drug Administration (FDA) approved a total of 693 orphan drugs (covering 1228 formulations/specifications). Among these, 201 drugs (425 formulations/specifications) were approved by China’s National Medical Products Administration (NMPA), accounting for 29.0% (201/693) of the approved drugs and 34.6%(425/1228) of the approved formulations/specifications. The number of China’s rare diseases drugs is increasing year by year, and the approval speed has gradually accelerated. The median approval lag time was 32.3 years (IQR: 22.9–33.6) from 1983 to 1987. In contrast, the shortest median lag time occurred between 2018 and 2022, at approximately 1.4 years (IQR: 0.5–2.6)—a reduction of 94.2% that reflects the success of China’s ongoing drug regulatory reforms. The special procedures for drug approval have a great promoting effect on rare diseases drugs’ approval.

Conclusion

This research provides evidence of breakthroughs in the review and approval of rare disease drugs in China and demonstrates the tremendous boost to rare disease drugs from China’s ongoing restructuring and reform of the drug regulatory ecosystem, as well as a stimulus for future rare disease drugs development in China.