Knowledge and Attitudes of Clinical Trials among Patients with Rare Diseases and the Guardians in China

作者全名："Xiang, Ziling; Jiang, Hui; Jiang, Wengao; Wang, Yali; Zheng, Hang"

作者地址："[Xiang, Ziling; Jiang, Wengao; Zheng, Hang] Chongqing Med Univ, Sch Pharm, Yuanjiagang Campus,Shiyou Rd St, Chongqing, Peoples R China; [Jiang, Hui] Fujian Med Univ, Zhangzhou Hosp, Zhangzhou, Peoples R China; [Wang, Yali] Guangzhou First Peoples Hosp, Dept Neurol, Guangzhou, Peoples R China"

通信作者："Zheng, H (通讯作者)，Chongqing Med Univ, Sch Pharm, Yuanjiagang Campus,Shiyou Rd St, Chongqing, Peoples R China."

来源：THERAPEUTIC INNOVATION & REGULATORY SCIENCE

ESI学科分类：PHARMACOLOGY & TOXICOLOGY

WOS号：WOS:001067714900001

JCR分区：Q4

影响因子：1.5

年份：2024

卷号：58

期号：1

开始页：53

结束页：62

文献类型：Article

关键词：Clinical trial; Rare disease; Knowledge; Attitude

摘要："BackgroundConducting of clinical trials for rare diseases faces multiple challenges. Patients' cognition and attitude toward clinical trials are crucial, which may affect their participation and compliance, and affect the schedule of clinical trials eventually.Objective and MethodThis study aims to explore the knowledge and attitudes of clinical trials of patients with rare diseases or patients' guardians. An anonymous cross-sectional survey was conducted from November 1, 2021, to November 30, 2021. A total of 1131 valid questionnaires were included. Among them, 417 were filled in by the patients themselves, and 714 were answered by the patients' guardians.ResultsThe average score of clinical trial knowledge of the patients (8.25) was lower than that of the guardians (8.85). The willingness of the patients to participate in clinical trials was high (4.28), and the willingness of the patients' guardians was also high for patients to participate in clinical trials (4.35). The main promoting factors of clinical trial participation were the possibility of curing the disease. The main hindering factors of participation in clinical trials were lack of access to clinical trial information and concern about the safety and effectiveness of the trial drug.ConclusionsIn conclusion, most respondents had some basic knowledge of clinical trials and high willingness to participate in clinical trials. But there were some cognitive deficiencies about clinical trials and many hindering factors to participate in clinical trials. Clinical trials of rare diseases should be patient-centered and truly meet the unmet clinical, psychological, and social needs of patients with rare diseases."

基金机构："The authors would like to thank the contribution of the rare disease organizations (CORD Center for Rare Diseases, etc.) for the questionnaire collection of this research and colleagues (Rufang Huang, Mingshu Ma, Feifei Peng, Chengqiao Shi, Ying Wang, Lixi"

基金资助正文："The authors would like to thank the contribution of the rare disease organizations (CORD Center for Rare Diseases, etc.) for the questionnaire collection of this research and colleagues (Rufang Huang, Mingshu Ma, Feifei Peng, Chengqiao Shi, Ying Wang, Lixin Wang, etc.) for the questionnaire design, questionnaire collection, and questionnaire analysis."