Department of Applied Health Research, University College London, London, England, UK.
Putnam PHMR, London, England, UK.
Value Health. 2024 May;27(5):623-632. doi: 10.1016/j.jval.2024.01.020. Epub 2024 Feb 17.
Evidence about the comparative effects of new treatments is typically collected in randomized controlled trials (RCTs). In some instances, RCTs are not possible, or their value is limited by an inability to capture treatment effects over the longer term or in all relevant population subgroups. In these cases, nonrandomized studies (NRS) using real-world data (RWD) are increasingly used to complement trial evidence on treatment effects for health technology assessment (HTA). However, there have been concerns over a lack of acceptability of this evidence by HTA agencies. This article aims to identify the barriers to the acceptance of NRS and steps that may facilitate increases in the acceptability of NRS in the future.
Opinions of the authorship team based on their experience in real-world evidence research in academic, HTA, and industry settings, supported by a critical assessment of existing studies.
Barriers were identified that are applicable to key stakeholder groups, including HTA agencies (eg, the lack of comprehensive methodological guidelines for using RWD), evidence generators (eg, avoidable deviations from best practices), and external stakeholders (eg, data controllers providing timely access to high-quality RWD). Future steps that may facilitate future acceptability of NRS include improvements in the quality, integration, and accessibility of RWD, wider use of demonstration projects to highlight the value and applicability of nonrandomized designs, living, and more detailed HTA guidelines, and improvements in HTA infrastructure relating to RWD.
NRS can represent a crucial source of evidence on treatment effects for use in HTA when RCT evidence is limited.
新治疗方法的比较效果证据通常在随机对照试验 (RCT) 中收集。在某些情况下，RCT 不可行，或者由于无法在更长时间或所有相关人群亚组中捕捉治疗效果，其价值受到限制。在这些情况下，越来越多地使用使用真实世界数据 (RWD) 的非随机研究 (NRS) 来补充 RCT 对治疗效果的证据，以进行卫生技术评估 (HTA)。然而，人们担心 HTA 机构对这种证据的接受程度不足。本文旨在确定接受 NRS 的障碍，并确定未来可能增加 NRS 可接受性的步骤。
根据作者在学术、HTA 和行业环境中进行真实世界证据研究的经验得出的观点，辅以对现有研究的批判性评估。
确定了适用于关键利益相关者群体的障碍，包括 HTA 机构（例如，缺乏使用 RWD 的综合方法学指南）、证据生成者（例如，可避免偏离最佳实践）和外部利益相关者（例如，及时提供高质量 RWD 的数据控制者）。未来可能促进 NRS 未来可接受性的步骤包括提高 RWD 的质量、整合和可及性，更广泛地使用示范项目来突出非随机设计的价值和适用性，制定更具活力和更详细的 HTA 指南，以及改善与 RWD 相关的 HTA 基础设施。
当 RCT 证据有限时，NRS 可以成为治疗效果的重要证据来源，用于 HTA。
