School of Pharmacy, Chongqing Medical University, Yuanjiagang Campus, Shiyou Road Street, Yuzhong District, Chongqing, 68485161, China.
Orphanet J Rare Dis. 2022 Jul 27;17(1):294. doi: 10.1186/s13023-022-02440-4.
Rare diseases have been increasingly recognized as unmet medical and health needs worldwide; a growing demand for the development of orphan drugs emerges subsequently. Therefore, it is of great interest for both the Chinese regulatory agency and pharmaceutical companies to keep tract on the clinical development of orphan drugs in China.
This study aims to reveal the current situation and trend of the clinical development of orphan drugs in China, based on the data collected from the Chinese official platform, dating from January 1, 2013 to December 31, 2021.
A total of 331 clinical trials for orphan drugs were extracted from the platform, covering 31 rare diseases and 124 drugs. Increases were seen in the annual number of clinical trials and drugs being tested, with a sharp increase after 2018. About the disease types of the 331 trials, Parkinson disease (young-onset, early-onset) (86, 26%), hemophilia (70, 21%), homozygote hypercholesterolemia (60, 18%) were the most common. Furthermore, it was also observed that the largest number of clinical trial units for rare disease in east China (90, 41%) and the smallest number located in northwest China (18, 6%) and northeast China (18, 6%).
The growth trends illustrate the progress in clinical trial and drug development of rare diseases from 2013 to 2021. However, promoting orphan drugs development still is an important issue in China; at the same time, further efforts should be made for meet the unmet needs of disease types and balance the uneven distribution of medical resources for clinical trial on rare diseases.
罕见病已日益被全球公认为未被满足的医学和健康需求；随之而来的是对孤儿药开发的需求不断增长。因此，中国监管机构和制药公司都有兴趣跟踪中国孤儿药的临床开发情况。
本研究旨在基于中国官方平台自 2013 年 1 月 1 日至 2021 年 12 月 31 日收集的数据，揭示中国孤儿药临床开发的现状和趋势。
从该平台共提取了 331 项孤儿药临床试验，涵盖 31 种罕见病和 124 种药物。每年的临床试验和受试药物数量均有所增加，2018 年后增幅明显。在 331 项试验的疾病类型中，帕金森病（早发型、青年型）（86，26%）、血友病（70，21%）、纯合子高胆固醇血症（60，18%）最为常见。此外，还观察到华东地区（90，41%）罕见病临床试验单位数量最多，而西北地区（18，6%）和东北地区（18，6%）最少。
增长趋势说明了 2013 年至 2021 年罕见病临床试验和药物开发的进展。然而，促进孤儿药的发展仍然是中国的一个重要问题；同时，应进一步努力满足疾病类型的未满足需求，并平衡罕见病临床试验中医疗资源分布不均的问题。
