中国新药杂志

基于ClinicalTrials.gov的中国罕见病目录临床试验注册现状分析

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郑建洪 ¹ , 邱春凤 ² , 张真 ³

中国新药杂志 | 新药述评与论坛 2023,32(16): 1600-1607

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中国新药杂志 | 新药述评与论坛 2023, 32(16): 1600-1607

基于ClinicalTrials.gov的中国罕见病目录临床试验注册现状分析

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郑建洪¹, 邱春凤², 张真³

作者信息

¹厦门大学附属第一医院药剂科，厦门 361001

²厦门大学附属第一医院制剂科，厦门 361001

³厦门大学附属第一医院药物临床试验机构，厦门 361001

郑建洪，男，学士，主要从事医院药学及药物临床试验相关工作。E-mail: 373721370@qq.com。

通讯作者:

张真，女，博士，主要从事药物临床试验管理工作。E-mail: zhangzhen_xdfy@163.com。

Analysis of the status of clinical trials registered on ClinicalTrials.gov on diseases included in the list of rare diseases in China

Jian-hong ZHENG¹, Chun-feng QIU², Zhen ZHANG³

Affiliations

¹Department of Pharmacy, the First Affiliated Hospital of Xiamen University, Xiamen 361001, China

²Department of Preparation, the First Affiliated Hospital of Xiamen University, Xiamen 361001, China

³Drug Clinical Trial Institution, the First Affiliated Hospital of Xiamen University, Xiamen 361001, China

出版时间: 2023-08-30

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摘要

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目的：

了解中国罕见病目录涉及罕见病临床试验在ClinicalTrials.gov的注册情况，分析其注册特点。

方法：

检索建库以来至2022年4月5日在ClinicalTrials.gov注册平台发布的关于中国罕见病目录涉及所有罕见病临床试验，收集临床试验相关条目信息，运用WPS Office进行数据合并统计分析。

结果：

检索到罕见病相关临床试验12 904项，总体呈逐年增长趋势；临床试验分期缺失较多；样本量以0~100例为主；试验类型以干预性研究（9 042项，70.1%）和观察性研究（3 760项，29.1%）为主；罕见病临床试验状态以已完成（6 467项，50.1%）、招募中（2 388项，18.5%）和未知状态（1 207项，9.4%）3种状态为主；药物治疗方式的数量最多（5 992项，46.3%）；全球参与临床试验的国家/地区以欧美发达国家为主，单/多中心罕见病临床试验数量前3名均为欧美国家，我国开展的临床试验较少（460项，3.6%），但单中心临床试验占比最高（260项，56.5%）；研究机构以大学院校和医疗机构为主，我国以医疗机构为主（358项，77.8%）；申办机构以欧美国家的机构为主；罕见病临床试验设计以非随机、开放标签、平行分配的治疗性研究为主；临床试验结局指标以安全性及有效性相关指标为主。

结论：

我国罕见病临床试验的发展正处于上升阶段，但与欧美国家存在较大差距，应尽快建立系统的研发激励机制并出台相关法律法规，以促进罕见病临床试验的高质量开展。

关键词

罕见病 / 孤儿药 / 临床试验 / ClinicalTrials.gov / 注册特点 / 分析

Abstract

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Objective:

To investigate the status of clinical trials registered on ClinicalTrials.gov on diseases in the list of rare diseases in China and analyze the registration characteristics.

Methods:

All clinical trials involved in the list of rare diseases in China were searched on ClinicalTrials.gov from inception to April 5th, 2022. The relevant information of clinical trial registration was collected, and WPS Office was used for data analysis.

Results:

A total of 12 904 rare disease-related clinical trials were screened out, and the overall trend was increasing year by year. The information of clinical trial stages was missing a lot, and the sample size was mainly 0~100 cases. The types of trials were mainly interventional studies (9 042 trials, 70.1%) and observational studies (3 760 trials, 29.1%). There were three main statuses of trials: completed (6 467 trials, 50.1%), recruiting (2 388 trials, 18.5%), and unknown status (1 207 trials, 9.4%). The number of drug therapy trials was the largest (5 992 trials, 46.3%). The countries/regions participating in clinical trials were mainly developed countries in Europe and the United States. All of the top three single/multi-center rare disease-related clinical trials were conducted in European and American countries. There were a small percentage of clinical trials conducted in China (460 trials, 3.6%), but the proportion of single center-clinical trials was the highest (260 trials, 56.5%). The research institutions were mainly universities and medical institutions, and in China they were mainly medical institutions (358 trials, 77.8%). The sponsoring institutions were mainly European and American countries. The design of rare disease-related clinical trials was mainly non-randomized, open label and parallel allocation of therapeutic regimens. The outcome indicators were mainly related to safety and efficacy.

Conclusion:

The development of clinical trials of rare diseases in China is on the rise, but there is a big gap with European and American countries. We should establish a systematic research and development incentive mechanism and introduce relevant laws and regulations as soon as possible to promote the high-quality development of clinical trials of rare diseases.

Key words

rare diseases / orphan drug / clinical trials / ClinicalTrials.gov / registration features / analyze

引用本文

郑建洪, 邱春凤, 张真. 基于ClinicalTrials.gov的中国罕见病目录临床试验注册现状分析. 中国新药杂志, 2023 , 32 (16) : 1600 -1607 .

Jian-hong ZHENG, Chun-feng QIU, Zhen ZHANG. Analysis of the status of clinical trials registered on ClinicalTrials.gov on diseases included in the list of rare diseases in China[J]. Chinese Journal of New Drugs, 2023 , 32 (16) : 1600 -1607 .

参考文献

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2023年第32卷第16期

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首发时间：2026-03-05
出版时间：2023-08-30

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录用日期：2022-09-01

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作者信息

¹厦门大学附属第一医院药剂科，厦门 361001

²厦门大学附属第一医院制剂科，厦门 361001

³厦门大学附属第一医院药物临床试验机构，厦门 361001

通讯作者:

张真，女，博士，主要从事药物临床试验管理工作。E-mail: zhangzhen_xdfy@163.com。

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2种不同金属材料的力学参数

科 Family	属数 Number of genus	种数 Number of species	占总种数比例 Percentage of total species (%)	属 Genus	种数 Number of species	占总种数比例 Percentage of total species (%)
鹅膏菌科Amanitaceae	2	11	5.26	鹅膏菌属 Amanita	10	4.78
小菇科 Mycenaceae	2	12	5.74	丝盖伞属 Inocybe	5	2.39
多孔菌科 Polyporaceae	8	14	6.70	蜡蘑属 Laccaria	5	2.39
红菇科 Russulaceae	3	23	11.00	小皮伞属 Marasmius	6	2.87
				小菇属 Mycena	11	5.26
				光柄菇属 Pluteus	5	2.39
				红菇属 Russula	17	8.13
				栓菌属 Trametes	5	2.39

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