Lumanity Inc, Sheffield, England, UK.
Lumanity Inc, Sheffield, England, UK.
Value Health. 2023 Apr;26(4S):11-19. doi: 10.1016/j.jval.2023.01.010. Epub 2023 Jan 24.
In the past decade, there have been increasing calls for greater use of real-world evidence (RWE) and data (RWD), with the explicit goal of enabling faster provision of effective medicines to patients in need. The push for decision makers to accept RWE is especially noticeable in the pursuit of regulatory approval, but RWE, particularly when used to estimate the relative effectiveness of interventions, is not always readily accepted by agencies responsible for reimbursement and pricing of new pharmaceuticals and, to a varying degree, is not accepted across jurisdictions. This lack of trust hampers the use of RWE despite a very large and growing literature base on the principles of how RWE should be used. In this article, we suggest an important part of the explanation of why this situation has arisen and make suggestions for its alleviation. Given that problems commonly arise that are particular to the question being asked and the data sources being used, general guidance on the principles of how to use RWD cannot cover all eventualities. Therefore, we are suggesting the creation of an archive, or repository, to record uses of RWD in support of decisions by funding bodies or their advisors. This article introduces a proposed, structured classification of decision types using RWE, around which evidence can be assembled in a curated source (RWD/RWE taxonomy) and thus facilitate judgments on when evidence is "good enough." This article is part of a series in a special issue of this journal that looks at the barriers to optimal use of RWE in health technology assessment and how to overcome them. We begin significantly to populate our "taxonomy" with examples in an accompanying article. We also propose recommendations for international standards of evaluating the acceptability of RWD governance practices.
在过去的十年中,人们越来越呼吁更多地使用真实世界证据(RWE)和数据(RWD),其明确目标是使有需要的患者能够更快地获得有效的药物。决策者尤其希望接受 RWE,这在追求监管批准方面尤为明显,但 RWE(尤其是用于估计干预措施的相对有效性时)并不总是被负责新药品报销和定价的机构所接受,而且在不同程度上也不被所有司法管辖区所接受。尽管关于如何使用 RWE 的原则已经有大量且不断增长的文献基础,但由于缺乏信任,RWE 的使用受到了阻碍。在本文中,我们提出了造成这种情况的一个重要原因,并提出了缓解这种情况的建议。鉴于通常会出现特定于所提出问题和所使用数据源的问题,关于如何使用 RWD 的一般指导原则无法涵盖所有情况。因此,我们建议创建一个档案库或存储库,以记录 RWD 在支持资助机构或其顾问做出决策方面的使用情况。本文提出了一种使用 RWE 进行决策的分类建议,围绕该分类建议可以在经过策划的来源(RWD/RWE 分类法)中收集证据,从而便于判断证据是否“足够好”。本文是该期刊特刊中一系列文章的一部分,这些文章探讨了在健康技术评估中充分利用 RWE 的障碍以及如何克服这些障碍。我们在一篇相关文章中开始显著地用实例来填充我们的“分类法”。我们还提出了评估 RWD 治理实践可接受性的国际标准建议。
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