Evidence in the Wild

03

Feb

5 min read

What FDA’s Recent Rare Disease Approvals Teach Us About Single‑Arm Trial Design

Between late 2024 and late 2025, FDA approved six rare-disease therapies supported primarily by single-arm trials. None of these sponsors ran randomized controlled trials. All received traditional or accelerated approval. What separated success from rejection wasn’t luck or regulatory leniency. It was understanding what evidence compensates for the absence

29

Jan

4 min read

I Asked an LLM to Design My Clinical Trial

I asked an LLM a question that junior biostatisticians ask senior biostatisticians all the time: Should I use a Bayesian borrowing design for my Phase 2 single-arm oncology trial? Here's what I got back. The LLM Response Should I use a Bayesian borrowing design for my Phase 2

27

Jan

7 min read

Qalsody: The Probability of Harm

What FDA Actually Decided When the Trial Failed

Evidence in the Wild

Latest Articles

What FDA’s Recent Rare Disease Approvals Teach Us About Single‑Arm Trial Design

I Asked an LLM to Design My Clinical Trial

Qalsody: The Probability of Harm

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