EPISODE · Sep 27, 2026 · 20 MIN
Going for Gold: A Plan for Implementation
from Emma•ism · host Emma Søndergaard Jensen
How should we enact the revision to the rare disease medicine evidence hierarchy that I proposed in my LSE dissertation? This podcast explains how. Loosely, there should be 4 review steps: 1. Confirm the Study is for a Rare Disease 2. What is the Mechanism Plausibility Rating? 3. Future Plans for or Record of a Test of the Intervention (N-of-1 or Adaptive Bayesian) 4. Registry-Enabled Confirmatory Phase
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Going for Gold: A Plan for Implementation
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