Trends, lag and characteristics of rare disease drug approval in the USA and China, 1983–2022
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.
Authors
- Tingting Xu (ORCID: https://orcid.org/0000-0002-5662-4694)
- Q P Liu (ORCID: https://orcid.org/0000-0002-5304-3173)
- Junmei Shang (ORCID: https://orcid.org/0000-0001-9363-8502)
- Xin Tian (ORCID: https://orcid.org/0000-0003-2593-4208)
- Bo Zhang (ORCID: https://orcid.org/0000-0003-3389-5018)
- Shuyang Zhang
- Xin Liu
- Shaohong Wang
- Wei Zuo
- Rong Jiang
- Yuzhen Zou
- Yan Tang
Institutions
- China Pharmaceutical University (CN)
- Chinese Academy of Medical Sciences & Peking Union Medical College (CN)
- Peking Union Medical College Hospital (CN)
- National Engineering Research Center for Information Technology in Agriculture (CN)
- National Quality Forum (US)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-12-22
- DOI
- https://doi.org/10.1186/s13023-025-04175-4
- Primary Topic
- Health Systems, Economic Evaluations, Quality of Life
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Peking Union Medical College Hospital