Estimands in RWD/RWE

The team that is formed would have representation from the Implementation of Estimands (ICH E9 (R1)) using Data Standards project team with Estimands expertise, along with representation from various teams under the RWE working group. It seems appropriate that this would be a new team under the RWE working group, with knowledge contribution from the Estimands implementation team, since the focus of this project is on best practices of RWD Estimands, not on their implementation using data standards at this time.

There is currently very little guidance on how to construct Estimands for RWD/RWE, including how it is similar or different from randomised clinical trials (RCTs). From my knowledge, only one FDA guidance related to RWD/RWE mentions Estimands: Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products Guidance for Industry – Feb 2023 (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-design-and-conduct-externally-controlled-trials-drug-and-biological-products). This is a good start but expanding these principles with more guidance and examples provides an opportunity.

It would be helpful to many stakeholders across the Pharma and biotech industries to discuss and make available best practices for RWD Estimands, including how they are similar or different from Estimands in RCTs. It is expected to borrow expertise from the Implementation of Estimands (ICH E9 (R1)) using Data Standards project team that has a white paper currently in public review, along with other RWE working group project team expertise as well.