真实世界证据在药品上市后安全性评价的应用前景

Application Prospect of Real World Evidence in Post-Market Safety Evaluation of Drugs

随着真实世界研究方法学的不断丰富和数据源的数量、质量等研究基础不断提升,真实世界证据在药品安全性评价上具备更加广阔的应用前景。基于新药快速上市对安全检测的挑战,在上市后安全性评价的优势驱动下,在上市后开展真实世界研究进行安全性评价的情形将日渐增多。本文简述了FDA、EMA、PMDA等监管机构和ICH在使用真实世界数据进行安全性评价方面的主要进展,并指出真实世界安全性评价未来主要研究方向至少包括研究框架及方法学的丰富完善、登记系统等数据源的丰富和回归患者为中心,在日常诊疗中重视真实世界研究三个重要方向。

With the continuous enrichment of methodology and the improvement of the quantity and quality of data sources,real world evidence has broad application prospects in drug safety evaluation.The challenges of safety evaluation brought by rapid approval of new drugs and the advantages of real world research will drive the increasing use of real world studies for safety evaluation after market approval.This article briefly describes the major progress made by regulatory agencies such as the FDA,EMA,PMDA,and ICH in utilizing real world data for safety evaluation.Furthermore,it points out that the main future research directions for real world safety evaluation include the enrichment and improvement of research framework and methodology,the enrichment of registry systems and other data sources,and the focus on patient-centeredness and the importance of real world studies in daily medical practice.