Real-world Evidence Data now also expanded in the Field of Medical Devices

Real-world evidence (RWE) is an approach that has gained importance in both the EU and the US in recent years. What does it mean? Real-world evidence helps when clinical trials cannot take into account the entire patient population for a particular disease. This applies, for example, to multimorbid patients or patients who have not participated in any clinical trials due to their age. Real-world evidence is clinical knowledge about the use and potential benefits or risks of a drug, obtained from the analysis of real-world data (RWD - please also see FDA's final Guidance on Real-World Data and Real World Evidence. This concept is now to be extended to medical devices in the USA. What is planned?

In a statement, the US Food and Drug Administration (FDA) indicated that a restriction on the use of real-world evidence (RWE) in the review of drug and medical device applications would be lifted. In new guidance for certain types of medical device applications, the agency states that it will accept RWE without requiring identifiable individual patient data from real-world data sources to always be submitted in an application for approval. 

In the past, the FDA had insisted that all RWE submitted to the agency contain private and confidential information at the individual patient level. This approach made it impractical to use most large databases containing valuable data. However, both sponsors and data scientists saw opportunities to gain meaningful insights from some big data sources even without private, individual information.

The FDA is now complying with this. In future, FDA reviewers will examine the validity of the submitted RWE on a case-by-case basis. This applies, for example, to access to extensive databases such as cancer and cystic fibrosis registries, which contain important insights into how treatments work in practice. In future, in addition to the National Cancer Institute's national cancer registry, databases from hospitals, insurance claims databases and electronic health record networks will also be available for use. The FDA hopes that this data will provide insights into the outcomes of patients in different population groups and under real-world treatments that cannot be captured by traditional clinical trials.

Further information can be found on the FDA website.