The Impact of RWE Data on Regulatory Decision-Making

In the pharmaceutical and medical device industry — where being first-to-market can make or break a new product or treatment's financial viability — continuous process improvement is essential.

Clinical trial sponsors are always interested in ways to maximize cost-effectiveness and speed the process of commercialization. Real world evidence (RWE），使用传统的数据在临床试验和学术界的领域之外，可以帮助，提供潜在的效率，同时保持关键主题安全。

In many ways, RWE can be complementary to clinical trials. RWE can be useful for ongoing surveillance and especially in the assessment of long-term clinical outcomes for implants and therapeutic products. What makes this even more interesting is the increasing adoption of technology and widespread use of social media. Many people share their experiences in many types of online platforms, and the potential to collect data, in an ethical way, directly from patients, could provide more insights on the utility and safety of the product.

定义RWE

Real world evidence, in its broadest form, is evidence that is derived from collecting and analyzing Real world data (RWD). RWD are observational data obtained outside of randomized controlled trials (RCTs) usually generated from routine clinical practice. These data may be collected prospective or retrospectively. RWD sources may include medical charts, electronic patient records, reimbursement figures, billing databases and, more recently, clinical registries and patient reported outcomes.

美国，加拿大和欧洲的REWE倡议现状

U.S.: The Food and Drug Administration (FDA) published a framework for its RWE program in December 2018. In this document, the FDA firmly defines the current use of RWD in evidence generation and the framework for evaluating RWD/RWE for regulatory decision-making. Section 505F(b) of the Federal Food, Drug and Cosmetic Act (FD&C) defines RWE as "data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials" (21 U.S.C. 355g(b)).

在开发RWE计划时，FDA认为，区分RWD的来源和从该数据得出的证据有助于。在监管决策的背景下，评估RWE不仅取决于对用于产生证据的方法的评估，还取决于依据RWD的可靠性和相关性的方法。FDA已经驾驶了这个计划，它将继续评估和探索优化RWE效用的方法和方法。

加拿大：卫生加拿大（HC）一直在积极努力定义RWE在监管决策中的作用。2018年，HC开始与多个利益相关者咨询，以确定RWE在受监管产品开发生命周期中发挥的作用。其中一些讨论包括加强监管决策，风险沟通，映射RWE来源，潜在的新法规和基于风险的框架。HC预计在2022年秋季2020年秋季2020年和药品中完成医疗设备RWE项目。

Europe:The role of RWE has mainly focused on pricing and reimbursement in the past. Over the last several years there have been multiple discussions on what role, if any, RWE should have in clinical evidence, patient safety and outcomes and ultimately in licensing of products, particularly in pharmaceuticals.

The London School of Economics (LSE) published an Expert民意调查从2017年3月起，他们从35种定性和定量问题收集了关于使用RWE许可和覆盖建议，RWE所有权和RWE的未来的答案。来自各个国家的40名受访者（主要是欧洲）和职业。

在我看来，我发现最有趣的结论是接受孤儿和肿瘤的较低证据水平，RWE被认为在临床和报销中具有更多价值而不是监管决策，大多数受访者认为RWE是一个补充RCT而不是更换。LSE创造了一个潜在的rwe路线图这就是调试和访问，临床证据和患者和结果。这项工作由Roche提供资金，并通过LSE公开访问。

挑战需要考虑

RWE仍然是明显的重要性many areas of healthcare but in particular, drug reimbursement and health technology assessment. With this in mind, we need to assess where the data is coming from in order to feel assured that the data is representative of the actuals needs of the research methodology. We do not want to run the risk of using data that may lead to flawed conclusions; this will not only be detrimental for the evidence, but it risks public mistrust and patient safety concerns. There are many challenges to consider, I would like to highlight a few that may be impactful on the use of RWE on regulatory decision making:

• 全球RWE的定义和用途继续变化;事实上，目前没有达成共识。对于有助于对监管决策进行有用的RWE，将需要协调和标准化。
• Multiple stakeholders must address data quality and availability for RWD/RWE; this should include transparency of data sources and how this data is communicated to the general public.
• 数据库和注册机构在一段时间内只会真正有用，我们必须确保在使用此类资源时不会跳到结论。

Conclusion

毫无疑问，RWD / RWE在提供医疗保健时已经雕刻了空间，特别是在我们目前住的数字世界中。RWE可能发挥更大的作用，当然是对RCT对监管决策的补充，然而，这取决于监管机构的作用和期望。RWD / RWE现在正在用于患者的一小部分，例如在肿瘤和稀有疾病中。通过更强大的数据（通过透明数据源）和精致的方法和分析，RWE可以获得监管机构和公众的更多支持。