Department of Non-Communicable Disease Epidemiology, London School of Hygiene and Tropical Medicine, London, England, UK.
Department of Non-Communicable Disease Epidemiology, London School of Hygiene and Tropical Medicine, London, England, UK.
Value Health. 2021 Sep;24(9):1241-1244. doi: 10.1016/j.jval.2021.03.020. Epub 2021 Jun 26.
The value of real-world evidence (RWE) in medicines regulation and health technology assessment has been increasingly emphasized. Nevertheless, although RWE is increasingly used, there has been limited systematic evidence of its value. A recent study that examined the role and impact of RWE in regulatory assessments conducted through the European Medicines Agency provided such evidence. Results of the study demonstrated RWE was important to decision making, particularly for certain questions such as the quantification of adverse events, the evaluation of risk minimization measures, and the assessment of product usage. The study suggested, however, that in many of the assessments further RWE would have been valuable and concluded that RWE has, as yet, played a limited role in hypothesis generation and in the assessment of medication effectiveness. This study had been possible only because of the transparency of the European Medicines Agency decision making. Ensuring transparency of RWE evidence collection, study design and conduct, and of decision making based on this evidence will facilitate further development of the uses and value of RWE. Keywords: benefit-risk assessment; medicines regulation; real-world evidence; regulatory decision making.
真实世界证据（RWE）在药物监管和卫生技术评估中的价值已日益受到重视。然而，尽管 RWE 的应用日益广泛，但关于其价值的系统证据有限。最近一项研究考察了 RWE 在欧洲药品管理局开展的监管评估中的作用和影响，为这方面提供了证据。该研究结果表明，RWE 对决策很重要，特别是对于某些问题，如不良事件的量化、风险最小化措施的评估以及产品使用情况的评估。然而，该研究表明，在许多评估中，进一步的 RWE 将是有价值的，并得出结论认为，RWE 在假设生成和药物疗效评估方面的作用有限。这项研究之所以成为可能，仅仅是因为欧洲药品管理局决策的透明度。确保 RWE 证据收集、研究设计和实施以及基于该证据的决策的透明度，将有助于进一步发展 RWE 的用途和价值。关键词：风险获益评估；药物监管；真实世界证据；监管决策。
