f you’ve ever wondered whether single-arm studies are “good enough” for regulators or HTA bodies, this episode will challenge your assumptions. Anja Schiel, one of Europe’s leading voices at the regulator–HTA interface, explains why comparisons matter, where single-arm designs break down, and what smarter alternatives exist.

You’ll walk away with:

✔ A clearer understanding of the limits of single-arm trials

✔ Practical strategies for choosing comparators that strengthen your evidence package

✔ Insight into adaptive and hybrid designs that balance efficiency with rigor

✔ A regulator/HTA insider’s perspective on what decision-makers really need from statisticians

00:01:36 – Meet Anja Schiel and learn how she bridges regulation and HTA

00:02:28 – Why connecting regulators and HTA bodies early matters for drug development

00:06:07 – Where single-arm studies often fall short in real-world decision-making

00:08:01 – What accelerated approvals have taught us about assumptions in trial design

00:10:17 – Why comparison—not just randomization—is at the heart of sound evidence

00:12:12 – The pitfalls of relying on literature-based or naïve comparisons

00:16:54 – How regulators and HTA approach evidence differently

00:17:00 – What “concurrent control” really means and why it’s crucial

00:20:33 – Strategic thinking when selecting comparators for long-term value

00:26:00 – The role of adaptive and hybrid designs in modern trials

00:33:59 – Ethical considerations when trial designs fall short

00:36:26 – Why rare diseases demand smarter collaboration and evidence planning

00:40:25 – Communicating study objectives and estimands clearly for all stakeholders

00:44:55 – Final takeaways: how statisticians can lead the push for better designs

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