ORIGINAL RESEARCH article
Front. Pharmacol.
Sec. Drugs Outcomes Research and Policies
Volume 16 - 2025 | doi: 10.3389/fphar.2025.1595497
Drug lag and associated factors of orphan drugs approved by the U.S. in China
Provisionally accepted
- 1Hainan University, Haikou, China
- 2Shenyang Pharmaceutical University, Shenyang, Liaoning Province, China
- 3Tsinghua University, Beijing, Beijing, China
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Background: A pronounced disparity exists in the accessibility of orphan drugs between China and developed countries, such as the United States. Identifying and analyzing the critical determinants that contribute to this gap is essential for enhancing the availability of orphan drugs and promoting equitable access for patients.We included all new orphan drugs approved by the US Food and Drug Administration (FDA) between 2013 and 2023 and collected their approval information in China and the United States. Major factors of interest included accelerated review pathway, locations where pivotal clinical trials were conducted, therapeutic category, and other factors affecting the delay in drug launch. They were analyzed using multinomial logistic regression, analysis of covariance, and the Mann-Whitney U test.The FDA approved a total of 242 new orphan drugs between 2013 and 2023. Among these, 119 (49.2%) of these drugs had been approved in China as of January 1, 2025, with a median lag time of 1,004 days (2.75 years). Among them, 47 drugs (41.2%) were included in the China's List of Rare Diseases. Multinomial logistic regression analysis revealed that the conduct of pivotal trials supporting FDA approval in mainland China was associated with whether such drugs were launched in China (odds ratio = 10.53, 95% confidence interval 3.67-40.79; P < 0.001). Furthermore, the Mann-Whitney U test indicated that such characteristics as the inclusion of indications in China's List of Rare Diseases, the granting of breakthrough therapy designation by the National Medical Products Administration (NMPA), and the inclusion of pivotal clinical trials in mainland China were all associated with the shortening of drug lag time.Discussion: Our findings suggest that orphan drug approval delays in China have improved significantly, but still face significant challenges. China's involvement in the global collaborative development of drugs not only helps shorten the relative lag time for drugs to obtain approval in China but also avoids repeated trials and significantly improves R&D efficiency. We recommend that pharmaceutical companies include Chinese patients in the drug development stage so that they can enjoy cutting-edge
Keywords: orphan drug, Launch delay, Clinical Trial, Drug access, Expedited Program
Received: 18 Mar 2025; Accepted: 31 Jul 2025.
Copyright: © 2025 Mo, He, Guo and YANG. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Yue YANG, Tsinghua University, Beijing, 100084, Beijing, China
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.