基于患者报告结局的症状监测在药物警戒中的应用前景及方法学考量

中国医药导刊 ›› 2023, Vol. 25 ›› Issue (12): 1194-1199.

基于患者报告结局的症状监测在药物警戒中的应用前景及方法学考量

石丘玲1，王旭2

1.重庆医科大学超声医学工程国家重点实验室，重庆 400016；
2.伊普德峰（北京）健康科技有限公司，北京 100094

收稿日期:2024-01-04 修回日期:2024-01-09 出版日期:2023-12-28 发布日期:2024-02-18
基金资助:
科技部国家重点研发计划“政府间国际科技创新合作”重点专项项目（项目编号：2022YFE0133100；项目名称：智能化肺癌术后症状“监测-预警-干预”关键技术研发)

Prospects and Methodological Considerations of PRO-Based Symptom Monitoring for Pharmacovigilance

1.State Key Laboratory of Ultrasound in Medicine and Engineering， Chongqing Medical University， Chongqing 400016，China；
2.ePRO Vision (Beijing) Health Technology Co.， Ltd.， Beijing 100094， China

Received:2024-01-04 Revised:2024-01-09 Online:2023-12-28 Published:2024-02-18

摘要/Abstract

摘要： 患者是药品不良反应的直接承受者，患者报告结局（PRO）是对患者经历的直接评估。在“以患者为中心”的医疗新模式下，患者在医疗行为过程中的深度参与可为不良反应的监测提供其自身经历的信息。本研究依据作者团队在国内外PRO临床应用及方法学研究的基础，探讨了PRO在药物警戒三大核心领域——监测、评估及预防中的潜在应用前景及可能遇到的方法学障碍。①作为患者主观感受的评估指标，PRO是药品不良反应相关症状监测的关键技术。在方法学方面，主要障碍体现在监测工具的选择及监测时间点制定。采用简短且特异的量表，结合低负担的监测方案，将有助于提高不良反应监测的实时性和精准度。②在解读监测结果时，需兼顾统计学显著差异及临床显著意义。制定PRO评分阈值及最小临床意义变化值是确保监测结果有效解读的方法学基础。③研究讨论了电子化PRO“监测-预警-干预”模式在药物警戒领域的应用潜力，以及电子化平台辅助PRO在药物警戒应用中的代表性和依从性问题。针对这些问题，本研究提出了基于临床医学、人工智能及数据科学等多学科交叉技术创新的解决思路，供业内人士交流参考，旨在共同促进“以患者为中心”的药物警戒实践及科研的发展。

关键词: 药物警戒, 患者报告结局, 不良事件, 症状监测, 方法学考量

Abstract: Experiencing adverse events induced by pharmaceutical products， patients are one of the key contributors to pharmacovigilance (PV). Patients-reported outcome (PRO) is an approach that directly measures the severity， frequency or interference of symptoms from patients， without interpretation from others， thereby presenting an innovative way for PV. This study explores the potential utilization of PRO in the three core areas of PV-monitoring， evaluation， and prevention， as well as the potential methodological obstacles that may be encountered. Firstly， with the ability to measure subjective feelings of patients， PRO has been recognised as the major approach for monitoring symptoms related to adverse events. Methodologically， the main obstacles are reflected in the selection of monitoring tools and the establishment of assessment timepoints. The use of short and specific scales， combined with low-burden schedules， will ensure timely and accurate identification of adverse events. Secondly， when interpreting PRO scores， both statistical and clinical significances need to be considered. Establishing PRO score thresholds and minimum clinically significant changes is the methodological basis for clinically meaningful interpretation of monitoring results. Finally， the article discusses the potential application of the electronic PRO “monitoring-alert-intervention” in the field of PV， as well as the representativeness and compliance issues of the current electronic approaches. In response to these issues， this study proposes solutions based on interdisciplinary technological innovations such as clinical medicine， artificial intelligence， and data science. The aim is to highlight the potential of PRO in PV， with the overarching goal of enhancing patients' quality of life and ultimately prolonging their survival.

Key words: Pharmacovigilance, Patient-reported outcomes, Adverse events, Symptom monitoring, Methodological considerations

中图分类号:

石丘玲, 王旭. 基于患者报告结局的症状监测在药物警戒中的应用前景及方法学考量[J]. 中国医药导刊, 2023, 25(12): 1194-1199.

SHI Qiuling, WANG Xu. Prospects and Methodological Considerations of PRO-Based Symptom Monitoring for Pharmacovigilance[J]. CHINESE JOURNAL OF MEDICINAL GUIDE, 2023, 25(12): 1194-1199.

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