– Written by Matt Baldwin
PHUSE’s webinar series At the Intersection of Estimands and Target Trial Emulation (TTE) for RWE welcomed a record-breaking number of attendees.
The first two webinars, on 22 April and 5 June, delivered practical insights into how estimands and target trial emulation frameworks are reshaping the design and interpretation of real-world evidence (RWE) studies. Featuring experts from regulatory agencies, industry and academia, the sessions brought much-needed clarity to one of the most complex areas in modern clinical research.
Webinar 1: An Introduction to the Estimands and Target Trial Emulation Frameworks
Date: 22 April 2025
Why It Was So Popular:
- Framework Fundamentals: Dr Rachele Hendricks-Sturrup (Duke-Margolis Institute) opened the series with a comparative overview of the Estimands (ICH E9(R1) ) framework, the Target Trial Emulation (TTE) framework and the Causal Roadmap. Publication Reference: Real-World Evidence to Support Causal Inference: Methodological Considerations for Non-Interventional Studies (June 2024)
- Methodical Guidance: Dr Lauren Eyler Dang (NIAID ) detailed how the Causal Roadmap supports assumption testing and pre-specification. Publication Reference: A Causal Roadmap for Generating High-Quality Real-World Evidence (September 2023)
- Regulatory Relevance: Dr Juan José Abellán (EMA) introduced their reflection paper and an EMA-funded research project to implement the TTE and Estimands frameworks, including 10 use cases of regulatory interest. Publication Reference: Reflection paper on use of real-world data in noninterventional studies to generate real-world evidence for regulatory purposes (March 2025)
Additional Resources:
Webinar 2: Estimands in Real-World Evidence Studies
Date: 5 June 2025
Why Attendance Grew:
- FDA Clarity: Dr Hana Lee (FDA ) explained how the agency distinguishes real-world data from real-world evidence – and why transparent protocols, fit-for-purpose data and patient-level submissions are vital.
- Estimands in Action: Dr Hongwei Wang (AbbVie ) highlighted why estimands are even more critical in real-world settings – where ICEs, heterogeneous populations and uncontrolled conditions demand rigorous frameworks.
- Publication Reference: Estimands in Real-World Evidence Studies (November 2023)
- Real-World Example: A retrospective RWE study paired with a Phase II trial supported FDA decision-making. Estimands helped navigate challenges around endpoint comparability and confounder adjustment.
Panel Takeaways:
- Ben Ackerman (Johnson & Johnson ) stressed the need to iterate between theoretical estimands and real-world data realities.
- Birol Emir (Pfizer ) emphasised the need for cross-functional education around estimands.
Additional Resources:
- Webinar Recording
- Presentation Slides
- Q&A – coming soon
Still Wondering What the Hype Is About?
These webinars are more than academic – they offer real strategies for conducting credible, reproducible and regulatory-ready RWE studies. Whether you’re in industry, academia or regulatory science, this series is essential to your work.
With more sessions to come, now’s the time to catch up. Recordings, slides, Q&A documents and future events are available via the PHUSE Working Group Events calendar page.
Don’t just hear about the hype – join us to see why this series is setting a new standard across the PHUSE Community.