The Effective Statistician - in association with PSI Top 8: The Single Arm Studies and What are the Alternatives?
10 snips
Sep 15, 2025 Anja Schiel, a vice chair at UNETA 21 and expert at the Norwegian Medicines Agency, delves into the complexities of single-arm studies in drug approval. She discusses the critical need for robust comparisons in evidence evaluation and the limitations of single-arm designs. Anja also highlights the importance of concurrent controls and shares practical strategies for choosing effective comparators. With insights into adaptive trial designs, she sheds light on statistical communication between regulators and HTA bodies, emphasizing the need for clear evidence strategies.
AI Snips
Chapters
Transcript
Episode notes
Limits Of Single‑Arm Trials
- Single-arm trials often fail because they lack a credible comparison and rest on untestable assumptions.
- Without a control you cannot assess selection bias or quantify the impact of trial conduct on outcomes.
Comparison Is The Core
- Comparison, not just randomization, is the core need for making inferential conclusions.
- Randomization prevents selection bias but the essential value is having a contemporaneous control to assess effects.
Pick Comparators Strategically
- Choose comparators strategically to strengthen later indirect comparisons and HTA submissions.
- Run a pretrial network exercise to pick a comparator with rich existing evidence.
