Estimands for RWD/RWE
Project Description |
This project focuses on establishing best practices for defining estimands in real-world data (RWD) and real-world evidence (RWE), rather than their implementation using data standards. The team brings together expertise from across PHUSE initiatives, including the Implementation of Estimands (ICH E9 (R1)) project, the Estimands Implementation Working Group (EIWG) HTA and the RWE sub-team, and contributors from the RWE Working Group, representing regulators, statisticians, epidemiologists and health economists. Currently, there is limited guidance on constructing estimands for RWD/RWE and how these differ from or align with those used in randomised clinical trials (RCTs). Existing regulatory guidance, such as the FDA’s February 2023 publication on externally controlled trials, provides an initial foundation but lacks detailed practical direction. This project aims to address these gaps by developing clear, practical guidance and examples, supporting consistent and appropriate use of estimands across RWD/RWE studies and benefiting stakeholders across the pharmaceutical and biotech industries. |
Project Leads | |
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Ksenia Titorenko, ICON | |
Matt Baldwin, Volunteer | |
Paramita Chakraborty, IQVIA | |
Nicola Newton, PHUSE Project Coordinator |
Webinar Series |
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Q2-2025: Q2-2025 Q1-2026: Q2-2026: Q3-2026: Webinar 5 |
Click above for the latest project status. |