Estimands for RWD/RWE

The focus of this project is on best practices of RWD estimands, not on their implementation using data standards. The team formed has representation from the PHUSE Implementation of Estimands (ICH E9 (R1)) using Data Standards project team, which has a White Paper nearing publication, and the Estimands Implementation Working Group (EIWG) sub team on HTA and RWE, both with estimands expertise, along with representation from teams under the RWE Working Group. The EIWG sub team on HTA and RWE started in late 2022, and has a cross-functional membership, including regulators, statisticians, epidemiologists and health economists.

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). As of Q1 2024, 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.