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离散选择实验在罕见病用药价值评估及偏好测量中的应用
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| 篇名： | 离散选择实验在罕见病用药价值评估及偏好测量中的应用 |
| TITLE： | Application of discrete choice experiment in value assessment and preference measurement for orphan medicinal product |
| 摘要： | 目的 系统梳理离散选择实验（DCE）在罕见病用药价值评估及偏好测量中的应用现状，为该方法在我国的规范应用提供参考。方法系统检索中国知网、万方数据、维普网、中国生物医学文献服务系统、PubMed、WebofScience、Medline、Embase等中英文数据库，纳入采用DCE法对罕见病用药进行价值或偏好评估的原始研究。采用ISPOR联合分析清单和DIRECT清单分别评价纳入研究的方法学质量和报告完整性，并对调查对象、属性设置及属性相对重要性等进行归纳分析。结果共纳入8篇符合标准的文献，所有文献的报告质量和方法学质量均较高。调查对象包括公众、患者/照护者、决策者及利益相关者。DCE属性数量为4～13个，中位数为7.5个，经主题综合法归纳为“疾病相关“”治疗相关”和“经济费用相关”3个维度、14个二级准则。其中，治疗效果（13次）、疾病严重性（9次）、安全性（7次）、未满足需求（6次）和治疗费用（5次）为最常纳入的二级准则。相对重要性排序显示，治疗效果在多数研究中居首位，其次为医保筹资。结论当前DCE在罕见病用药价值评估中已初步形成较为一致的核心属性框架和选择偏好，未来应进一步推动DCE运用，为罕见病用药多准则决策分析的属性与准则选择提供依据。 |
| ABSTRACT： | OBJECTIVE To systematically review the current application of discrete choice experiment （DCE） in the value assessment and preference measurement of orphan medicinal product （OMP）， and to provide a reference for the standardized use of this methodology in China. METHODS The systematic search was conducted across Chinese and English databases including CNKI， Wanfang Data， VIP， CBM， PubMed， Web of Science， Medline， and Embase. Original studies that employed DCE to evaluate the value or preferences related to OMP were included. The methodological quality and reporting completeness of the included studies were assessed using the ISPOR Conjoint Analysis Checklist and the DIRECT Checklist， respectively. Respondent populations， attribute setting， and the relative importance of attributes were summarized and analyzed. RESULTS Eight eligible studies were included； all studies demonstrated high-quality reporting and methodological rigor. Respondents comprised the general public， patients/caregivers， policymakers， and other stakeholders. The number of DCE attributes ranged from 4 to 13 （median＝7.5）. Through thematic synthesis， these attributes were categorized into three dimensions， namely “disease-related” “treatment-related” and “economic/financial-related” along with 14 secondary criteria. The most frequently included secondary criteria were treatment efficacy （13 occurrences）， disease severity （9 occurrences）， safety （7 occurrences）， unmet medical need （6 occurrences）， and treatment cost （5 occurrences）. Rankings of relative importance identified treatment efficacy as the most valued criterion across most studies， followed by health insurance financing. CONCLUSIONS DCE applications in the value assessment of OMP have begun to converge on a relatively consistent core attribute framework and selection preference. Future research should further promote the use of DCE to inform attribute and criterion selection in multi-criteria decision analysis frameworks for OMP. |
| 期刊： | 2026年第37卷第07期 |
| 作者： | 支腾；唐弦；骆彦州；胡明 |
| AUTHORS： | ZHI Teng，TANG Xian，LUO Yanzhou，HU Ming |
| 关键字： | 罕见病用药；离散选择实验；价值评估；社会偏好；系统评价；多准则决策分析 |
| KEYWORDS： | orphan medicinal product； discrete choice experiment； value assessment； societal preference； systematic review； |
| 阅读数： | 9 次 |
| 本月下载数： | 1 次 |
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