Estimands for RWD/RWE

The team formed would have representation from the PHUSE Implementation of Estimands (ICH E9 (R1)) using Data Standards project team, and the EIWG, both with estimands expertise, along with representation from 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 at this time on best practices of RWD estimands, not on their implementation using data standards.

There is currently very little guidance or discussion 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 – February 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 PHUSE Implementation of Estimands (ICH E9 (R1)) using Data Standards project team, which has a white paper nearing publication, along with other PHUSE RWE Working Group project team expertise and the Estimands Implementation Working Group (EIWG) subteam on HTA and RWE. The EIWG subteam on HTA and RWE started about 18 months ago, and has a cross-functional membership, including regulators, statisticians, epidemiologists and health economists.