Value， methods and challenges of applying patient experience data in real-world study of drugs

ZHANG Lele; ZHU Keying; WANG Duanning; HE Yuying; DING Zuoqi

doi:10.6039/j.issn.1001-0408.2024.23.02

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Value， methods and challenges of applying patient experience data in real-world study of drugs

更新时间：2024-12-09

- Value， methods and challenges of applying patient experience data in real-world study of drugs
- ZHONGGUO YAOFANG Vol. 35, Issue 23, Pages: 2844-2850(2024)
- 作者机构：
  
  中国药科大学国际医药商学院，南京　211198
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2024.23.02
  CLC： R95
- Published：15 December 2024，
  
  Received：11 June 2024，
  
  Revised：22 October 2024，
- 稿件说明：
移动端阅览
张乐乐,朱可盈,王端宁等.患者体验数据应用于药物真实世界研究的价值、方法与挑战 ^Δ[J].中国药房,2024,35(23):2844-2850.

ZHANG Lele,ZHU Keying,WANG Duanning,et al.Value， methods and challenges of applying patient experience data in real-world study of drugs[J].ZHONGGUO YAOFANG,2024,35(23):2844-2850.
张乐乐,朱可盈,王端宁等.患者体验数据应用于药物真实世界研究的价值、方法与挑战 ^Δ[J].中国药房,2024,35(23):2844-2850. DOI： 10.6039/j.issn.1001-0408.2024.23.02.

ZHANG Lele,ZHU Keying,WANG Duanning,et al.Value， methods and challenges of applying patient experience data in real-world study of drugs[J].ZHONGGUO YAOFANG,2024,35(23):2844-2850. DOI： 10.6039/j.issn.1001-0408.2024.23.02.

摘要

“以患者为中心”的药品监管理念下，在真实世界证据中纳入患者维度的评价愈显重要。患者体验数据可以补充和解释已有数据、生成直接来自患者的证据，实现药物研发中的患者参与。数据类型包括患者报告结局、自由文本类数据等，可通过自主收集或数据库获取。应用场景涉及新药注册、安全性评价、新增适应证等。在我国，将患者体验数据应用于真实世界研究主要面临以下挑战：缺乏收集高质量数据的条件、标准、动力，数据类型单一，数据安全与数据自由难以平衡等。建议我国出台专项指南、建立测量工具认证程序、拓展数据收集途径、探索数据源整合方法、优化知情同意机制、建立证据协同机制，以推动“以患者为中心”理念在药物真实世界研究中的实践应用。

Abstract

Under the “patient-centered” drug regulation concept， the inclusion of patient dimensions in real-world evidence becomes increasingly important. Patient experience data can complement and interpret existing data， generate evidence directly from patients， and achieve patient participation in drug development. Data types include patient-reported outcomes and free-text data， which can be collected autonomously or obtained from databases. Application scenarios involve new drug registration， safety evaluation， and additional indications. In China， applying patient experience data to real-world study mainly faces the following challenges： lack of conditions， standards， and motivation to collect high-quality data， a single type of data， and the difficulty of balancing data security with freedom， etc. It is recommended to issue special guidelines， establish a measurement tool certification process， expand data collection channels， explore data source integration methods， optimize the informed consent mechanism， and establish an evidence synergy mechanism to promote the practical application of the “patient-centered” concept in real-world study of drugs.

关键词

患者体验数据真实世界研究监管决策患者报告结局

Keywords

real-world studyregulatory decision-makingpatient-reported outcome

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