患者偏好信息在美国医疗产品上市前决策中的应用及启示

任晓悦; 颜建周; 丁瑞琳; 邵蓉

doi:10.6039/j.issn.1001-0408.2024.19.01

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患者偏好信息在美国医疗产品上市前决策中的应用及启示

药事管理 | 更新时间：2024-10-11

- 患者偏好信息在美国医疗产品上市前决策中的应用及启示
- Application of patient preference information in premarketing decision of medical products in the United States and its implications
- 中国药房 2024年35卷第19期页码：2321-2327
- 作者机构：
  
  中国药科大学国家药物政策与医药产业经济研究中心，南京　211198
- 作者简介：
  
  硕士研究生。研究方向：医药政策与法规。E-mail：18852079330@163.com
  教授，博士生导师，博士。研究方向：医药政策与法规。电话：025-86185193。E-mail：shaorong118@163.com
- 基金信息：
  
  教育部人文社会科学研究一般项目(22YJAZH126)
- DOI：10.6039/j.issn.1001-0408.2024.19.01
  中图分类号： R95
- 纸质出版日期：2024-10-15，
  
  收稿日期：2024-05-18，
  
  修回日期：2024-08-26，
- 稿件说明：
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任晓悦,颜建周,丁瑞琳等.患者偏好信息在美国医疗产品上市前决策中的应用及启示 ^Δ[J].中国药房,2024,35(19):2321-2327.

REN Xiaoyue,YAN Jianzhou,DING Ruilin,et al.Application of patient preference information in premarketing decision of medical products in the United States and its implications[J].ZHONGGUO YAOFANG,2024,35(19):2321-2327.
任晓悦,颜建周,丁瑞琳等.患者偏好信息在美国医疗产品上市前决策中的应用及启示 ^Δ[J].中国药房,2024,35(19):2321-2327. DOI： 10.6039/j.issn.1001-0408.2024.19.01.

REN Xiaoyue,YAN Jianzhou,DING Ruilin,et al.Application of patient preference information in premarketing decision of medical products in the United States and its implications[J].ZHONGGUO YAOFANG,2024,35(19):2321-2327. DOI： 10.6039/j.issn.1001-0408.2024.19.01.

摘要

目的

通过研究患者偏好信息在美国医疗产品上市前决策中的应用，为我国患者偏好信息的应用提供借鉴。

方法

采用文献研究法探究美国患者偏好信息收集与应用的概况、法律依据和参与主体，详细分析患者偏好信息在医疗产品上市前决策中的应用情况，分析我国患者偏好信息的应用现状与挑战，并提出建议。

结果与结论

美国通过开展多项患者参与项目以及立法推动多主体进行患者偏好信息的收集和应用，将患者偏好信息用于为医疗产品上市前决策提供参考：在研发环节为医疗产品研发和设计提供信息、辅助临床试验设计；在上市审批环节作为FDA上市审批决策的证据之一，用于识别获益大于风险的患者群体，并纳入医疗产品说明书。我国患者偏好信息的应用缺乏更高层级法律文件的指导，尚无针对性的指南文件。建议我国借鉴美国经验，在更高层级的法律文件中明确鼓励患者偏好的研究与应用；针对患者偏好信息的收集与应用制定专门的指导文件；根据我国国情确定监管决策中患者偏好信息纳入的权重和形式。

Abstract

OBJECTIVE

To provide reference for the application of patient preference information in China by studying the application of patient preference information in the premarketing decision-making of medical products in the United States.

METHODS

The literature research method was used to explore the general situation， legal basis， and participants of the collection and application of patient preference information in the United States， analyze the application of patient preference information in premarketing decision-making of medical products in detail， and analyze the application status and challenges of patient preference information in China， so as to put forward suggestions.

RESULTS & CONCLUSIONS

United States has promoted the collection and application of patient preference information through several patient participation projects and legislation. The patient preference information is used to support premarketing decision-making of medical products： providing information for medical product development and design， and assisting clinical trial design in the research and development process； helping to support FDA’s marketing approval decisions， identifying patient groups whose benefits outweigh risks， and included in medical product descriptions in the marketing approval process. The application of patient preference information in China lacks the guidance of higher-level legal documents， and there are no targeted guidance documents. It is suggested that China should learn from the experience of the United States and clearly encourage the research and application of patient preference in higher-level legal documents； develop specific guidance documents for the collection and application of patient preference information； determine the weight and form of patient preference information to be considered in regulatory decision-making according to national conditions.

关键词

患者偏好药品医疗器械上市前决策

Keywords

drugmedical devicepremarketing decision

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