Center for Food and Drug Inspection, National Medical Products Administration, Beijing, China.
Duke University School of Medicine, Durham, North Carolina, USA.
Contemp Clin Trials. 2021 Oct;109:106521. doi: 10.1016/j.cct.2021.106521. Epub 2021 Jul 31.
The 21st Century Cures Act passed by the United States (US) Congress in December 2016 requires the US Food and Drug Administration (FDA) shall establish a program to evaluate the potential use of real-world evidence (RWE) which is generated from real-world data (RWD) to (i) support approval of new indication for a drug approved under section 505 (c) and (ii) satisfy post-approval study requirements. RWE offers the opportunities to develop robust evidence using high-quality data and sophisticated methods for producing causal-effect estimates regardless randomization is feasible. In this article, we have demonstrated that the assessment of treatment effect (RWE) based on RWD could be biased due to the potential selection and information biases of RWD. Although fit-for-purpose RWE may meet regulatory standards under certain assumptions, it is not the same as substantial evidence (current regulatory standard in support of approval of regulatory submission). In practice, it is then suggested that when there are gaps between fit-for-purpose RWE and substantial evidence, we should make efforts to fill these gaps based on a comprehensive evaluation of the treatment effect. We also review two RWE examples to show some potential use of RWE in clinical studies.
2016 年 12 月，美国国会通过的《21 世纪治愈法案》要求美国食品和药物管理局（FDA）建立一个项目，以评估从真实世界数据（RWD）中产生的真实世界证据（RWE）的潜在用途，（i）支持根据第 505(c)节批准的药物的新适应症，以及（ii）满足批准后研究要求。RWE 提供了使用高质量数据和复杂方法产生因果效应估计值的机会，而无需考虑随机化是否可行。在本文中，我们已经证明，基于 RWD 的治疗效果评估（RWE）可能会因 RWD 的潜在选择和信息偏倚而产生偏差。尽管在某些假设下，适合目的的 RWE 可能符合监管标准，但它与实质性证据（目前支持监管提交批准的监管标准）不同。在实践中，当适合目的的 RWE 与实质性证据之间存在差距时，我们应该努力根据对治疗效果的综合评估来填补这些差距。我们还审查了两个 RWE 示例，以展示 RWE 在临床研究中的一些潜在用途。
