Peng Feifei, Zheng Hang
School of Pharmaceutical Sciences, Chongqing Medical University, #1 Yixueyuan Road, Yuzhong District, Chongqing, 400016, China.
Ther Innov Regul Sci. 2023 Nov;57(6):1314-1321. doi: 10.1007/s43441-023-00572-8. Epub 2023 Aug 31.
In order to meet the unmet needs of rare disease patients in China, importing orphan drugs is an important way. The objectives of this study were to investigate the marketing trend of orphan drugs approved by the US Food and Drug Administration (FDA) and imported by China, to examine the orphan drug lag between China and the United States.
This study analyzes the orphan drugs approved by FDA and imported by China from January 2010 to December 2021. The approval lag for orphan drugs between China and the US was calculated and analyzed by approval time. Factors potentially affecting the approval lag, such as target disease, ATC classification, formulation, corporation name, drug type, and whether the indications belong to the first batch of rare diseases catalogue were investigated.
The number of FDA-approved orphan drugs imported by China is increasing year by year, and the approval lag of these drugs is gradually decreasing, especially in the classification of Non-L, Injections, Non-United States, and biological product. Compared with 2010-2015, the approval lag of total drugs in the study was significantly improved in 2016-2021 (1977 days) compared with 2010-2015 (3928 days).
China's groundbreaking regulatory reforms of drugs since 2015 had made significant progress in reducing orphan drug lags, but there is still considerable room for progress. We should more actively promote the approval of rare disease drugs in China, establish a better approval mechanism, and enable Chinese patients with rare diseases to receive drug treatment in a timely manner.
为满足中国罕见病患者未被满足的需求，进口孤儿药是一条重要途径。本研究的目的是调查美国食品药品监督管理局（FDA）批准并由中国进口的孤儿药的市场趋势，以考察中美之间孤儿药的滞后情况。
本研究分析了2010年1月至2021年12月期间FDA批准并由中国进口的孤儿药。根据批准时间计算并分析了中美之间孤儿药的批准滞后时间。研究了可能影响批准滞后的因素，如目标疾病、解剖学治疗学化学分类系统（ATC）分类、剂型、公司名称、药物类型以及适应症是否属于首批罕见病目录。
中国进口的FDA批准的孤儿药数量逐年增加，这些药物的批准滞后时间逐渐缩短，尤其是在非L类、注射剂、非美国公司生产以及生物制品类别中。与2010 - 2015年相比，2016 - 2021年研究中所有药物的批准滞后时间有显著改善（从2010 - 2015年的3928天降至2016 - 2021年的1977天）。
2015年以来中国具有开创性的药品监管改革在减少孤儿药滞后方面取得了显著进展，但仍有相当大的进步空间。我们应更积极地推动中国罕见病药物的批准，建立更好的批准机制，使中国罕见病患者能够及时获得药物治疗。
