This chapter explores the significant challenges and biases associated with single arm studies, particularly in oncology drug approval and health technology assessments (HTA). It critiques the reliance on these studies due to issues of internal validity and the difficulties in making reliable comparisons without control groups. The discussion emphasizes the necessity for robust comparative studies to ensure accurate assessments of treatment efficacy and the complexities involved in clinical trial design.
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Interview with Anja Schiel
Why You Should Listen:
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
Episode Highlights:
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
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