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

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Abstract

Background

Conducting 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 Method

This 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.

Results

The 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.

Conclusions

In 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.

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Acknowledgements

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.

Funding

This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.

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Authors and Affiliations

  1. School of Pharmacy, Chongqing Medical University, Yuanjiagang Campus, Chongqing Medical University, Shiyou Road Street, Yuzhong District, Chongqing, China

    Ziling Xiang, Wengao Jiang & Hang Zheng

  2. Zhangzhou Hospital affiliated to Fujian Medical University, Zhangzhou, China

    Hui Jiang

  3. Department of Neurology, Guangzhou First People’s Hospital, Guangzhou, China

    Yali Wang

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  1. Ziling Xiang
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  2. Hui Jiang
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Contributions

All authors designed the research and carried out the data collection. Ziling Xiang analyzed the data. Ziling Xiang and Hang Zheng wrote the manuscript. All authors have approved the final manuscript.

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Correspondence to Hang Zheng.

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Xiang, Z., Jiang, H., Jiang, W. et al. Knowledge and Attitudes of Clinical Trials among Patients with Rare Diseases and the Guardians in China. Ther Innov Regul Sci 58, 53–62 (2024). https://doi.org/10.1007/s43441-023-00571-9

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