RAND Corporation, Arlington, VA, USA.
RAND Corporation, Santa Monica, CA, USA.
Value Health. 2021 Dec;24(12):1792-1798. doi: 10.1016/j.jval.2021.07.003. Epub 2021 Aug 18.
To enhance the generalizability of the evidence it reviews, the US Food and Drug Administration (FDA) has encouraged manufacturers to expand the submission of real-world evidence (RWE). The extent to which this evidence, which is generated outside of research settings, can support decision making remains unclear. We described the current use of RWE for medical devices, assessed manufacturers' challenges in generating and using it for regulatory and coverage decisions, and identified opportunities to expand its use.
We conducted 27 solo and group interviews with FDA officials and representatives of device manufacturers, payers, and health technology assessment organizations. All interviews used a semistructured protocol and were transcribed to allow thematic analysis.
Accessing and linking real-world data sources, identifying unique devices, capturing longitudinal data, limited staff expertise, and uncertain return on investment have hampered efforts to use real-world data. Many companies in our sample were conducting research using real-world data, but none had submitted RWE as the primary evidence supporting a premarket approval. FDA guidance was helpful, but regulatory requirements remained ambiguous and examples of successful regulatory decisions based on RWE were limited. Payers mainly used RWE to supplement experimental evidence in coverage decisions, evaluated both types of evidence in similar ways, and had concerns about the rigor of RWE.
Technical challenges may slow efforts to generate and use RWE in the near term. Additional regulatory guidance and examples, greater use of rigorous study designs and analytic methods, and continued stakeholder engagement could accelerate the use of RWE.
为了增强其审查证据的普遍性，美国食品和药物管理局（FDA）鼓励制造商扩大真实世界证据（RWE）的提交。目前尚不清楚这种在研究环境之外产生的证据在多大程度上能够支持决策。我们描述了医疗器械中 RWE 的当前使用情况，评估了制造商在为监管和覆盖范围决策生成和使用 RWE 方面面临的挑战，并确定了扩大其使用的机会。
我们对 FDA 官员和医疗器械制造商、支付方和卫生技术评估组织的代表进行了 27 次单独和小组访谈。所有访谈均使用半结构化方案，并进行转录以允许进行主题分析。
访问和链接真实世界数据源、识别独特的设备、捕获纵向数据、有限的员工专业知识以及不确定的投资回报，这些都阻碍了使用真实世界数据的努力。我们样本中的许多公司都在使用真实世界数据进行研究，但没有一家公司将 RWE 作为支持上市前批准的主要证据提交。FDA 指南很有帮助，但监管要求仍然模棱两可，并且基于 RWE 的成功监管决策的例子有限。支付方主要将 RWE 用于补充覆盖决策中的实验证据，以类似的方式评估这两种类型的证据，并对 RWE 的严谨性表示关注。
技术挑战可能会在短期内减缓生成和使用 RWE 的努力。更多的监管指导和示例、更多地使用严格的研究设计和分析方法以及持续的利益相关者参与都可以加速 RWE 的使用。
