Mo Qihui, He Jiaqi, Guo Qixiang, Yang Yue
School of Biomedical Engineering, Hainan University, Sanya, China.
School of Business Administration, Shenyang Pharmaceutical University, Shenyang, China.
Front Pharmacol. 2025 Aug 29;16:1595497. doi: 10.3389/fphar.2025.1595497. eCollection 2025.
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 1 January 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.
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 innovative therapies more quickly.
中国与美国等发达国家在孤儿药可及性方面存在显著差距。识别和分析造成这一差距的关键决定因素对于提高孤儿药的可及性和促进患者公平获取至关重要。
我们纳入了2013年至2023年期间美国食品药品监督管理局（FDA）批准的所有新型孤儿药，并收集了它们在中国和美国的获批信息。主要关注因素包括加速审评途径、关键临床试验开展地点、治疗类别以及其他影响药物上市延迟的因素。使用多项逻辑回归、协方差分析和曼-惠特尼U检验对这些因素进行分析。
2013年至2023年期间，FDA共批准了242种新型孤儿药。截至2025年1月1日，其中119种（49.2%）已在中国获批，中位滞后时间为1004天（2.75年）。其中，47种药物（41.2%）被列入中国罕见病目录。多项逻辑回归分析显示，在中国境内开展支持FDA批准的关键试验与这些药物是否在中国上市相关（比值比=10.53，95%置信区间3.67-40.79；P<0.001）。此外，曼-惠特尼U检验表明，诸如被列入中国罕见病目录、获得国家药品监督管理局（NMPA）突破性治疗认定以及在中国境内开展关键临床试验等特征均与缩短药物滞后时间相关。
我们的研究结果表明，中国孤儿药获批延迟情况虽有显著改善，但仍面临重大挑战。中国参与全球药物合作研发不仅有助于缩短药物在中国获批的相对滞后时间，还能避免重复试验并显著提高研发效率。我们建议制药公司在药物研发阶段纳入中国患者，以便他们能更快地享受到前沿创新疗法。
